Molecular Profile Detail

Profile Name KIT L576P
Gene Variant Detail

KIT L576P (gain of function)

Relevant Treatment Approaches KIT Inhibitor

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown non-small cell lung carcinoma no benefit KIT Inhibitor Carboplatin + Paclitaxel + Motesanib Phase III Actionable In a Phase III trial, Motesanib (AMG 706) in combination with Taxol (paclitaxel) and Paraplatin (carboplatin) did not improve progression-free survival significantly compared to placebo (6.1 vs 5.6 months) in patient with non-squamous non-small cell lung cancer (PMID: 28902534). 28902534
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Motesanib + Carboplatin Phase III Actionable In a Phase III study, motesanib plus Paraplatin (carboplatin) or Taxol (paclitaxel) improved overall survival, progression free survival and objective response rate for a subset of Asian patients with advanced nonsquamous NSCLC (PMID: 24419239). 24419239
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Motesanib + Paclitaxel Phase III Actionable In a Phase III study, motesanib plus Paraplatin (carboplatin) or Taxol (paclitaxel) improved overall survival, progression free survival and objective response rate for a subset of Asian patients with advanced nonsquamous NSCLC (PMID: 24419239). 24419239
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Amuvatinib + Paclitaxel + Carboplatin Phase I Actionable In a Phase I clinical trial, Amuvatinib (MP470) in combination with chemotherapeutic agents, demonstrated safety and some efficacy in patients with advanced solid tumors (PMID: 24849582). 24849582
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor RXDX-105 Phase I Actionable In a Phase I clinical trial, RXDX-105 (CEP-32496) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including 2 patients with heavily pretreated non-small cell lung cancer that achieved stable disease for greater that 6 months (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr C188). detail...
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor RXDX-105 Phase I Actionable In a Phase I clinical trial, RXDX-105 (CEP-32496) was tolerable and demonstrated preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2574)). detail...
Unknown unknown angiosarcoma not applicable KIT Inhibitor Carotuximab + Pazopanib Phase Ib/II Actionable In a Phase I/II trial, TRC105 and Votrient (pazopanib) combination therapy resulted complete response in 40% (2/5) of angiosarcoma patients (J Clin Oncol 34, 2016 (suppl; abstr 11016)). detail...
Unknown unknown sarcoma not applicable KIT Inhibitor Carotuximab + Pazopanib Phase Ib/II Actionable In a Phase I/II trial, TRC105 and Votrient (pazopanib) combination therapy resulted in a median progression free survival of 3.95 months and ongoing complete response in 4% (3/81) of soft tissue sarcoma patients (J Clin Oncol 34, 2016 (suppl; abstr 11016)). detail...
Unknown unknown clear cell renal cell carcinoma no benefit KIT Inhibitor everolimus + pazopanib Phase II Actionable In a Phase II trial, alternating treatment with Votrient (pazopanib) and Afinitor (everolimus) did not improve 1-year progression free survival rate (45% vs 32%) or time to progression/death (7.4 vs 9.4 months) compared to continuous Votrient (pazopanib) treatment in patients with renal clear cell carcinoma (PMID: 27918762). 27918762
Unknown unknown melanoma not applicable KIT Inhibitor Gemcitabine + Pazopanib Phase I Actionable In a Phase I study, Votrient (pazopanib) administered with Gemzar (gemcitabine) was shown to be tolerated with one partial response (metastatic melanoma) and prolonged disease stabilization in three patients (metastatic melanoma, cholangiocarcinoma, and colorectal carcinoma) (PMID: 23064954). 23064954
Unknown unknown Advanced Solid Tumor sensitive KIT Inhibitor Gemcitabine + Pazopanib Phase I Actionable In a Phase I study, Votrient (pazopanib) administered with Gemzar (gemcitabine) was shown to be tolerated in patients with advanced solid tumors, and resulted in one partial response (metastatic melanoma) and prolonged disease stabilization in three patients (metastatic melanoma, cholangiocarcinoma, and colorectal carcinoma) (PMID: 23064954). 23064954
Unknown unknown uterine corpus sarcoma not applicable KIT Inhibitor Pazopanib Clinical Study Actionable In a retrospective clinical study, Votrient (pazopanib) treatment resulted in objective response in 29% (10/35) and stable disease in 31% (11/35) of patients with uterine sarcoma, with median progression-free survival of 5.8 months and overall survival of 20.0 months (PMID: 29185261). 29185261
Unknown unknown breast cancer not applicable KIT Inhibitor Pazopanib Phase II Actionable In a Phase II trial, Votrient (pazopanib) displayed safety and some efficacy in breast cancer patients (PMID: 20682606). 20682606
Unknown unknown sarcoma not applicable KIT Inhibitor Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 15.4 weeks and median survival of 11.2 months in patients with soft tissue sarcoma (PMID: 26970174). 26970174
Unknown unknown sarcoma not applicable KIT Inhibitor Pazopanib FDA approved Actionable In a Phase III trial that supported FDA approval, Votrient (pazopanib) improved progression free survival in patients with advanced soft tissue sarcoma (PMID: 22595799). detail... 22595799
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Pazopanib Phase I Actionable In a Phase I trial, Votrient (pazopanib) demonstrated safety and efficacy in a variety of pediatric solid tumors (PMID: 23857966). 23857966
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Pazopanib Phase I Actionable In a Phase I study, Votrient (pazopanib) in combination with Taxol (paclitaxel) and Paraplatin (carboplatin) demonstrated efficacy in solid tumors (PMID: 22679111). 22679111
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Pazopanib FDA approved Actionable In a Phase III trial that supported FDA approval, Votrient (pazopanib) improved progression free survival in patients with advanced renal cell carcinoma (PMID: 20100962). detail... 20100962
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Pazopanib Phase III Actionable In a Phase III trial, adjuvant Votrient (pazopanib) therapy post nephrectomy did not improve disease-free survival (HR=0.862, p=0.165) compared to placebo in patients with renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 4507)). detail...
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor Pazopanib Phase II Actionable In a Phase II trial, treatment with Votrient (pazopanib) plus best supportive care (BSC) resulted in improved progression-free survival (45% at 4 months) compared to BSC alone (15% at 4 months) in patients with Gleevec (imatinib) and Sutent (sunitinib)-resistant gastrointestinal stromal tumors (J Clin Oncol 33, 2015 (suppl; abstr 10506)). detail...
Unknown unknown gastrointestinal neuroendocrine tumor not applicable KIT Inhibitor Pazopanib Clinical Study Actionable In a clinical study, Votrient (pazopanib) treatment in patients with gastroenteropancreatic neuroendocrine tumors resulted in an overall response rate of 24% (19/124), stable disease in 39.5% (49/124), a progression free survival of 36% at six months, and a median overall survival of 10.2 months (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 446P). detail...
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Pazopanib Phase II Actionable In a Phase II trial, Votrient (pazopanib) treatment resulted in 86% (30/35) of patients with non-small cell lung cancer experiencing a decrease in tumor size, including one patient with a greater than 50% reduction, and resulted in a partial response based on RECIST criteria in three patients (PMID: 20516450). 20516450
Unknown unknown fibrous histiocytoma not applicable KIT Inhibitor Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 15.3 weeks and median survival of 9.5 months in patients with undifferentiated pleomorphic sarcoma/malignant fibrous histiocytoma (PMID: 26970174). 26970174
Unknown unknown liposarcoma not applicable KIT Inhibitor Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 8 weeks and median survival of 7.3 months in patients with liposarcoma (PMID: 26970174). 26970174
Unknown unknown synovial sarcoma not applicable KIT Inhibitor Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 16.4 weeks and median survival of 10.6 months in patients with synovial sarcoma (PMID: 26970174). 26970174
Unknown unknown germ cell cancer not applicable KIT Inhibitor Pazopanib Phase II Actionable In a Phase II trial, Votrient (pazopanib) treatment resulted in partial responses in 4.7% (2/43), stable disease in 44.2% (19/43), and a 3-month progression free survival probability of 12.8% in patients with refractory germ cell cancer (PMID: 28383677). 28383677
Unknown unknown clear cell renal cell carcinoma not applicable KIT Inhibitor Pazopanib Phase III Actionable In a Phase III trial, Votrient (pazopanib) did not result in a significantly improved disease free survival compared to placebo in patients with renal clear cell carcinoma (PMID: 28902533). 28902533
Unknown unknown clear cell renal cell carcinoma not applicable KIT Inhibitor Pazopanib Phase II Actionable In a Phase II trial, 84% (84/100) of patients with renal clear cell cancer demonstrated a clinical benefit, which included a median tumor reduction of 14.4%, when treated with Votrient (pazopanib) prior to a planned nephrectomy (PMID: 27254750). 27254750
Unknown unknown leiomyosarcoma not applicable KIT Inhibitor Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 18.6 weeks and median survival of 20.1 months in patients with leiomyosarcoma (PMID: 26970174). 26970174
Unknown unknown uterus leiomyosarcoma not applicable KIT Inhibitor Pazopanib Clinical Study Actionable In a retrospective clinical study, 33% (9/27) of patients with uterine leiomyosarcoma responded to Votrient (pazopanib) treatment (PMID: 29185261). 29185261
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Pazopanib + Abexinostat Phase Ib/II Actionable In a Phase Ib/II trial, the combination of Abexinostat (PCI-24781) and Votrient (pazopanib) in advanced solid tumor patients resulted in a clinical benefit rate of 37% (16/43), a median response duration of 9.1 months, and 8 patients of 43 achieved stable disease or durable response for greater than 12 months (PMID: 28221861). 28221861
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Pazopanib + Abexinostat Phase Ib/II Actionable In a Phase Ib/II trial, 27% (6/22) of renal cell carcinoma patients demonstrated a response when treated with a combination of Abexinostat (PCI-24781) and Votrient (pazopanib) (PMID: 28221861). 28221861
Unknown unknown clear cell renal cell carcinoma not applicable KIT Inhibitor Pazopanib + Abexinostat Phase Ib/II Actionable In a Phase Ib/II trial, 31% (5/16) of renal clear cell carcinoma patients demonstrated a response to the combination of Abexinostat (PCI-24781) and Votrient (pazopanib) (PMID: 28221861). 28221861
Unknown unknown papillary renal cell carcinoma not applicable KIT Inhibitor Pazopanib + Abexinostat Phase Ib/II Actionable In a Phase Ib/II trial, 17% (1/6) of patients with papillary renal cell carcinoma demonstrated a response to the combination of Abexinostat (PCI-24781) and Votrient (pazopanib) (PMID: 28221861). 28221861
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Pazopanib + Carboplatin + Paclitaxel Phase I Actionable In a Phase I study, Votrient (pazopanib) in combination with Taxol (paclitaxel) and Paraplatin (carboplatin) demonstrated efficacy in solid tumors (PMID: 22679111). 22679111
Unknown unknown head and neck squamous cell carcinoma not applicable KIT Inhibitor Pazopanib + Cetuximab Phase Ib/II Actionable In a Phase Ib clinical trial, combined therapy, Votrient (pazopanib) and Erbitux (cetuximab), was well tolerated in head and neck squamous cell carcinoma patients with metastatic or resistant disease, and resulted in a 6% (2/31) complete response rate, a 29% (9/31) partial response rate, a median time to progression of 5.5 months, and a median overall survival of 9.5 months (PMID: 30001987; NCT01716416). 30001987
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Pazopanib + Paclitaxel Phase I Actionable In a Phase I trial, the combination of Votrient (pazopanib) and Taxol (paclitaxel) demonstrated safety and resulted in partial response in 36% (10/28) and stable disease in 36% (10/28) of patients with advanced solid tumors (PMID: 25504632). 25504632
Unknown unknown transitional cell carcinoma not applicable KIT Inhibitor Pazopanib + Paclitaxel Phase II Actionable In a Phase II trial, patients with urothelial carcinoma treated with the combination of Taxol (paclitaxel) and Votrient (pazopanib) demonstrated an overall response rate of 54% (15/28), in which 11% (3/28) experienced a complete response and 43% (12/28) experienced stable disease (PMID: 27068017). 27068017
Unknown unknown renal cell carcinoma no benefit KIT Inhibitor Pazopanib + Pembrolizumab Phase I Actionable In a Phase I trial, Votrient (pazopanib) and Keytruda (pembrolizumab) combination treatment resulted in significant hepatotoxicity in patients with advanced renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 4506)). detail...
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Pazopanib + Trametinib Preclinical - Cell line xenograft Actionable In a preclinical study, combination of Mekinist (trametinib) and Votrient (pazopanib) effectively inhibited tumor angiogenesis and growth in cell line xenograft models of renal cell carcinoma (PMID: 26487278). 26487278
Unknown unknown sarcoma no benefit KIT Inhibitor Pazopanib + Trametinib Phase Ib/II Actionable In a Phase Ib/II trial, the combination of Votrient (pazopanib) and Mekinist (trametinib) demonstrated safety and resulted in partial response in 8% (2/25) and stable disease in 48% (12/25) of patients with advanced soft tissue sarcomas, but did not improve the 4 month progression-free survival rate over Votrient (pazopanib) alone (PMID: 28377484). 28377484
Unknown unknown glioblastoma multiforme not applicable KIT Inhibitor Pexidartinib Phase II Actionable In a Phase II trial, PLX3397 in combination with radiation therapy and Temodar (temozolomide) demonstrated safety and improved efficacy over standard therapy, resulted in a median progression free survival of 9.7 months and an estimated overall survival of 25.1 months in newly diagnosed glioblastoma multiforme patients (Neuro Oncol (2016) 18 (suppl 6): vi6.). detail...
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Pexidartinib + PLX9486 Phase I Actionable In a Phase I trial, PLX9486 and Pexidartinib (PLX3397) combination therapy demonstrated safety and preliminary efficacy, resulted in a partial response rate of 8.3% (1/12) and progression-free survival not yet reached in patients with advanced solid tumors, 11 of these patients had gastrointestinal stromal tumor that progressed on Gleevec (imatinib mesylate), and most harbored KIT exon 11 and exon 17 mutations (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 11509-11509; NCT02401815). detail...
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Derazantinib Phase I Actionable In a Phase I trial, Derazantinib (ARQ 087) treatment resulted in partial response in 4.5% (3/67) and stable disease in 38.8% (26/67) of patients with advanced solid tumors (PMID: 28972963; NCT01752920). 28972963
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Avelumab + Axitinib Phase III Actionable In a Phase III trial (JAVELIN Renal 101), Inlyta (axitinib) plus Bavencio (avelumab) treatment resulted in a median progression-free survival of 13.8 mo. vs. 8.4 mo. with Sutent (sunitinib), and an objective response rate of 51.4% vs. 25.7% with Sutent (sunitinib) in patients with advanced renal cell carcinoma, and at median follow-up 14.3% (63) of patients treated with Inlyta (axitinib) plus Bavencio (avelumab) had died vs. 16.9% (75) with Sutent (sunitinib) (PMID: 30779531; NCT02684006). 30779531
Unknown unknown neuroendocrine tumor not applicable KIT Inhibitor Axitinib Phase II Actionable In a Phase II clinical trial, treatment with Inlyta (axitinib) resulted in a median overall progression-free survival (PFS) of 26.7 months, a 12-month PFS rate of 74.5%, and median overall survival of 45.3 months, and led to partial response in 3% (1/30) and stable disease in 70% (21/30) of patients with neuroendocrine tumors, with some tumor shrinkage in 68% (15/22) of patients (PMID: 27080472). 27080472
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Axitinib Phase I Actionable In a Phase I trial, Inlyta (axitinib), in combination with chemotherapeutics, demonstrated antitumor activity in 42% (17/42) of patients with advanced solid tumors (PMID: 22996612). 22996612
Unknown unknown brain glioblastoma multiforme not applicable KIT Inhibitor Axitinib Preclinical Actionable In a preclinical study, Inlyta (axitinib) demonstrated efficacy in glioblastoma cells, including those resistant to Bevacizumab (J Clin Oncol (Meeting Abstracts) May 2013 vol. 31 no. 15_suppl 2077). detail...
Unknown unknown nasopharynx carcinoma not applicable KIT Inhibitor Axitinib Clinical Study Actionable In a clinical trial, treatment with Inlyta (axitinib) resulted in a 3-month clinical benefit rate (CBR) of 78.4% (29/37; 1 confirmed partial response (PR), 6 unconfirmed PR, 22 stable disease for greater than 3 months) and 6-month CBR of 43.2%, a median progression-free survival of 5.0 months, and median overall survival of 10.4 months in patients with nasopharyngeal carcinoma (PMID: 29301831). 29301831
Unknown unknown nasopharynx carcinoma not applicable KIT Inhibitor Axitinib Preclinical - Cell line xenograft Actionable In a preclinical study, Inlyta (axitinib) inhibited growth of nasopharyngeal carcinoma cell lines in culture, and inhibited tumor growth, decreased microvessel density, and increased tumor necrosis in a nasopharyngeal carcinoma xenograft model (PMID: 29301831). 29301831
Unknown unknown breast cancer not applicable KIT Inhibitor Axitinib Preclinical - Cell line xenograft Actionable In a preclinical study, Inlyta (axitinib) disrupted tumor microvasculature and inhibited tumor growth in breast cancer cell line xenograft models (PMID: 17371720). 17371720
Unknown unknown melanoma not applicable KIT Inhibitor Axitinib Phase II Actionable In a Phase II study in patients with metastatic melanoma, Inlyta (axitinib) was well tolerated and demonstrated antitumor activity (PMID: 21976544). 21976544
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Axitinib Phase II Actionable In a Phase II clinical trial, Inlyta (axitinib) was well-tolerated and demonstrated activity in patients with advanced non-small cell lung cancer, with a disease control rate of 41% (13/32), median progression-free survival of 4.9 months, and median overall survival of 14.8 months (PMID: 19597027). 19597027
Unknown unknown thyroid cancer not applicable KIT Inhibitor Axitinib Phase II Actionable In a Phase II study, Inlyta (axitinib) demonstrated antitumor activity in patients with advanced refractory thyroid cancer, resulting in a 30% response rate with another 38% of patients having stable disease (PMID: 20694064). 20694064
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Axitinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Inlyta (axitinib) as second-line therapy resulted in an improved progression-free survival of 8.3 months compared to 5.7 months with Nexavar (sorafenib) (HR 0.656, 95% CI 0.552-0.779; p<0.0001) in patients with metastatic renal cell carcinoma (PMID: 23598172). 23598172
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Axitinib Phase II Actionable In a Phase II clinical trial, treatment with Inlyta (axtinib) as first-line therapy resulted a prolonged median overall survival of 42.7 months compared to 30.4 months with placebo in patients with metastatic renal cell carcinoma (PMID: 27236772). 27236772
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Axitinib Clinical Study Actionable In a meta-analysis, Inlyta (axitinib) improved progression-free survival in patients with metastatic renal cell carcinoma (PMID: 24037486). 24037486
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Axitinib + Bevacizumab Phase I Actionable In a Phase I trial, Inlyta (axitinib) in combination with Avastin (bevacizumab) demonstrated safety and efficacy in patients with advanced solid tumors including metastatic colorectal cancer (PMID: 19940012). 19940012
Unknown unknown gastrointestinal system cancer not applicable KIT Inhibitor Axitinib + FOLFIRI Phase I Actionable In a Phase I trial, Inlyta (axitinib), in combination with FOLFIRI, demonstrated safety and some efficacy in patients with gastrointestinal tumors (PMID: 24423921). 24423921
Unknown unknown gastrointestinal system cancer not applicable KIT Inhibitor Axitinib + FOLFOX Phase I Actionable In a Phase I trial, Inlyta (axitinib), in combination with FOLFOX, demonstrated safety and some efficacy in patients with gastrointestinal tumors (PMID: 24423921). 24423921
Unknown unknown pancreatic cancer not applicable KIT Inhibitor Axitinib + Gemcitabine Phase I Actionable In a Phase I study, Inlyta (axitinib) in combination with Gemzar (gemcitabine), exhibited safety and antitumor activity in advanced pancreatic cancer patients (PMID: 21670972). 21670972
Unknown unknown ovarian cancer not applicable KIT Inhibitor Axitinib + Paclitaxel + Carboplatin Phase I Actionable In a Phase I clinical trial, Inlyta (axitinib) in combination with paclitaxel and carboplatin, demonstrated safety and efficacy in patients with advanced solid tumors including ovarian cancers (PMID: 22990652). 22990652
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Axitinib + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Inlyta (axitinib) and radiotherapy resulted in improved efficacy compared to either agent alone, and decreased pneumonitis in non-small cell lung cancer cell line xenograft models (PMID: 24862536). 24862536
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Axitinib + X4P-001 Phase I Actionable In a Phase I trial, X4P-001 in combination with Axitinib, displayed safety and efficacy in patients with renal cell carcinoma (AACR; Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B201, NCT02667886). detail...
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Carotuximab + Axitinib Phase I Actionable In a Phase I trial, treatment the combination of Inlyta (axitinib) and Carotuximab (TRC-105) demonstrated preliminary activity in patients with renal cell carcinoma, resulting in partial response in 5 and stable disease in 10 of 17 evaluable patients, and a median progression-free survival of 11.3 months (PMID: 30190302; NCT01806064). 30190302
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Dalantercept + Axitinib Phase II Actionable In a Phase II trial, the combination of Dalantercept (ACE-041) and Inlyta (axitinib) resulted in an objective response rate of 25% (7/28) and a median PFS of 8.3 months in renal cell carcinoma patients (PMID: 28031424). 28031424
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Everolimus + Lenvatinib Phase Ib/II Actionable In a Phase Ib clinical trial, the combination of Lenvima (lenvatinib) and Afinitor (everolimus) demonstrated safety, and resulted in partial response in 30% (6/20) of patients with metastatic renal cell carcinoma (PMID: 24190702). 24190702
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Everolimus + Lenvatinib FDA approved Actionable In a Phase II clinical trial that supported FDA approval, treatment with the combination of Lenvima (lenvatinib) and Afinitor (everolimus) resulted in a prolonged median progression-free survival of 14.6 months, compared to 5.5 months for Afinitor (everolimus) alone in patients with advanced renal cell carcinoma (PMID: 26482279). 26482279
Unknown unknown sarcoma not applicable KIT Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) demonstrated safety and anti-tumor activity in patients with advanced solid tumors, including partial responses in patients with soft-tissue sarcoma (PMID: 22516948). 22516948
Unknown unknown thyroid medullary carcinoma not applicable KIT Inhibitor Lenvatinib Phase II Actionable In a Phase II trial, advanced medullary thyroid cancer patients experienced an objective response rate of 36% (21/59, all partial responses) and median progression free survival was 9 months when treated with Lenvima (lenvatinib) (PMID: 26311725). 26311725
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors (PMID: 22516948). 22516948
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) treatment resulted in partial response in 11.7% (9/77) and stable disease in 51.9% (40/77) of patients with advanced solid tumors (PMID: 26169970). 26169970
Unknown unknown colon cancer not applicable KIT Inhibitor Lenvatinib Preclinical Actionable In a preclinical study, Lenvima (lenvatinib) induced apoptosis and inhibited proliferation of colorectal cancer cells in culture (PMID: 24255582). 24255582
Unknown unknown papillary thyroid carcinoma not applicable KIT Inhibitor Lenvatinib Clinical Study Actionable In a clinical study, Lenvima (lenvatinib) treatment resulted in prolonged response in a papillary thyroid carcinoma patient whose disease progressed despite 3 prior therapies including Nexavar (sorafenib), Sutent (sunitinib), and Votrient (pazopanib) (PMID: 29167691). 29167691
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) was demonstrated to be well tolerated and displayed anti-tumor activity in patients with melanoma and renal cell carcinoma (PMID: 22516948). 22516948
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Lenvatinib Phase I Actionable In a Phase I clinical trial, Lenvima (lenvatinib) demonstrated anti-tumor activity in patients with several advanced solid tumor types, including patients with non-small cell lung cancer (PMID: 26169970). 26169970
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Lenvatinib Phase II Actionable In a Phase II trial, Lenvima (lenvatinib) treatment resulted in partial response in 37% (17/46) and stable disease in 41% (19/46) of patients with advanced hepatocellular carcinoma, and a median overall survival of 18.7 months (PMID: 27704266). 27704266
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Lenvatinib FDA approved Actionable In a Phase III trial (REFLECT) that supported FDA approval, Lenvima (lenvatinib) demonstrated activity comparable to Nexavar (sorafenib), resulted in a median survival time of 13.6 months in patients with unresectable hepatocellular carcinoma (PMID: 29433850; NCT01761266). 29433850
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) resulted in safety and preliminary efficacy in patients with hepatocellular carcinoma, demonstrating a partial response in 15% (3/20) of patients and tumor regression in 70% (14/20) (PMID: 26500236). 26500236
Unknown unknown melanoma not applicable KIT Inhibitor Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) was demonstrated to be well tolerated and displayed anti-tumor activity in patients with melanoma and renal cell carcinoma (PMID: 22516948). 22516948
Unknown unknown thyroid cancer not applicable KIT Inhibitor Lenvatinib Phase II Actionable In a Phase II clinical trial, Lenvima (lenvatinib) demonstrated partial response in 36% (21/59) of patients, partial response or stable disease in 80% (47/59), and median progression free survival of 9 months in patients with advanced medullary thyroid cancer (PMID: 26311725). 26311725
Unknown unknown thyroid cancer not applicable KIT Inhibitor Lenvatinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Lenvima (lenvatinib) improved progression free survival and response rates in patients with radioiodine-refractory thyroid cancer (PMID: 25671254). detail... 25671254
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Lenvatinib + Carboplatin + Paclitaxel Phase I Actionable In a Phase I trial of patients with advanced or metastatic NSCLC, treatment with Lenvima (lenvatinib) in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) was tolerated and demonstrated anti-tumor activity (PMID: 23860537). 23860537
Unknown unknown pancreatic ductal adenocarcinoma not applicable KIT Inhibitor Gemcitabine + Masitinib Phase I Actionable In a Phase I trial, the median OS did not differ (7.7 mo vs 7.1 mo) between pancreatic ductal adenocarcinoma patients treated with the combination of Gemzar (gemcitabine) plus Masitinib (AB1010) versus those treated with Gemzar (gemcitabine) plus placebo (PMID: 25858497). 25858497
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor XL999 Phase II Actionable In six Phase II clinical trials, XL999 demonstrated anti-tumor activity in patients with advanced solid tumors, but also resulted in significant cardiotoxicity, which improved after discontinuation of the drug (J Clin Oncol, 25:18s, 2007 (Suppl; abstr 3591)). detail...
Unknown unknown glioblastoma multiforme not applicable KIT Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human glioblastoma multiforme cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown breast carcinoma not applicable KIT Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human breast carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in a variety of human advanced solid tumor cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor MGCD516 Phase I Actionable In a Phase I trial, MGCD516 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2575)). detail...
Unknown unknown lung carcinoma not applicable KIT Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human lung carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown colorectal cancer not applicable KIT Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human colorectal carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown stomach carcinoma not applicable KIT Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human stomach carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown liposarcoma not applicable KIT Inhibitor MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 blocked cell proliferation of a human liposarcoma cell line in culture and repressed tumor growth in xenograft models (PMID: 26675259). 26675259
Unknown unknown stomach cancer not applicable KIT Inhibitor Chiauranib Preclinical - Cell line xenograft Actionable In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a gastric cancer cell line xenograft model (PMID: 28004478). 28004478
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Chiauranib Preclinical - Cell line xenograft Actionable In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a hepatocellular carcinoma cell line xenograft model (PMID: 28004478). 28004478
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Chiauranib Preclinical - Cell line xenograft Actionable In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a non-small cell lung cancer cell line xenograft model (PMID: 28004478). 28004478
Unknown unknown colon cancer not applicable KIT Inhibitor Chiauranib Preclinical - Cell line xenograft Actionable In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a colon cancer cell line xenograft model (PMID: 28004478). 28004478
Unknown unknown clear cell renal cell carcinoma no benefit KIT Inhibitor Bevacizumab + Sorafenib Phase III Actionable In a Phase II clinical trial, treatment with the combination of Nexavar (sorafenib) and Avastin (bevacizumab) did not result in a significant improvement in progression-free survival compared to treatment with Avastin (bevacizumab) as a single agent (9.2 months vs 7.4 months) in patients with renal clear cell carcinoma (PMID: 26077237). 26077237
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Bevacizumab + Sorafenib Phase I Actionable In a Phase I trial, the treatment combination of Avastin (bevacizumab) and Nexavar (sorafenib) in patients with advanced solid tumors resulted in stable disease in 25% (29/115) of patients and a partial response in 5% (6/115) of patients (PMID: 25363205). 25363205
Unknown unknown invasive bladder transitional cell carcinoma not applicable KIT Inhibitor Cisplatin + Gemcitabine + Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) in combination with Platinol (cisplatin) and Gemzar (gemcitabine) resulted in pathologic complete response in 42.2% (19/45) of patients with muscle-invasive urothelial bladder cancer (J Clin Oncol 35, 2017 (suppl 6S; abstract 345)). detail...
Unknown unknown colon cancer not applicable KIT Inhibitor CVX-060 + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of CVX-060 and Nexavar (sorafenib) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown hepatocellular carcinoma no benefit KIT Inhibitor Erlotinib + Sorafenib Phase III Actionable In a Phase III trial, the combination treatment of Nexavar (sorafenib) with Tarceva (erlotinib) compared to Nexavar (sorafenib) plus placebo did not demonstrate an improved overall survival and time to progression in patients with hepatocellular carcinoma (PMID: 25547503). 25547503
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Fingolimod + Sorafenib Preclinical Actionable In a preclinical study, Gilenya (fingolimod) worked synergistically with Nexavar (sorafenib) to inhibit growth and induce apoptosis in hepatocellular carcinoma cell lines in culture (PMID: 26516583). 26516583
Unknown unknown colorectal cancer not applicable KIT Inhibitor Fluorouracil + Quinacrine + Sorafenib Preclinical - Cell culture Actionable In a preclinical study, Acrichine (quinacrine) and Adrucil (5-fluorouracil) synergistically enhanced the cytotoxicity of Nexavar (sorafenib) in human colorectal cancer cell lines in culture (PMID: 21725213). 21725213
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor GW5074 + Sorafenib Preclinical Actionable In a preclinical study, the combination of GW5074 and Nexavar (sorafenib) induced cell death in several tumor cell lines, and phosphorylation of DAPK at amino acid S308 correlated positively with response to therapy (PMID: 26100670). 26100670
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor GW5074 + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of GW5074 and Nexavar (sorafenib) worked synergistically to induce cell death in renal cell carcinoma cells in culture and inhibited tumor growth in xenograft and spontaneous mouse models of renal cell carcinoma (PMID: 26100670). 26100670
Unknown unknown hepatocellular carcinoma no benefit KIT Inhibitor Lenalidomide + Sorafenib Phase I Actionable In a Phase I clinical trial, the combination of Revlimid (lenalidomide) and Nexavar (sorafenib) was not well-tolerated and did not demonstrate clinical activity in patients with hepatocellular carcinoma, and the study was terminated early due to toxicity (PMID: 27256874). 27256874
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Metformin + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, addition of Glucophage (metformin) sensitized hepatocellular carcinoma cells to Nexavar (sorafenib) induced apoptosis in culture, resulted in suppression of postoperative intrahepatic recurrence and lung metastasis in cell line xenograft models (PMID: 26957312). 26957312
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Pemetrexed + Sorafenib Phase I Actionable In a Phase I clinical trial, the combination of Alimta (pemetrexed) and Nexavar (sorafenib) demonstrated safety and some preliminary efficacy in patients with advanced solid tumors, with an objective response rate of 15% (5/33) and stable disease in 45% (15/33) of patients (PMID: 27213589). 27213589
Unknown unknown triple-receptor negative breast cancer not applicable KIT Inhibitor Pemetrexed + Sorafenib Phase I Actionable In a Phase I clinical trial in patients with advanced solid tumors, the combination of Alimta (pemetrexed) and Nexavar (sorafenib) demonstrated safety and preliminary efficacy in patients with triple-receptor breast cancer (TNBC), with 60% (3/5) of TNBC patients demonstrating an objective response and 100% (5/5) of patients achieving stable disease or better (PMID: 27213589). 27213589
Unknown unknown breast cancer not applicable KIT Inhibitor Pemetrexed + Sorafenib Phase I Actionable In a Phase I clinical trial in patients with advanced solid tumors, the combination of Alimta (pemetrexed) and Nexavar (sorafenib) demonstrated safety and preliminary efficacy in patients with breast cancer, with 58% (7/12) of breast cancer patients achieving objective response or stable disease (PMID: 27213589). 27213589
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor PX-866 + Sorafenib Preclinical Actionable In a preclinical study, treatment with the combination of Stivarga (regorafenib) PK-866 resulted in increased cell death in a variety of solid tumor cell lines in culture (PMID: 23877009). 23877009
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Refametinib + Sorafenib Phase I Actionable In a Phase I trial, 43.8% (7/16) of hepatocellular carcinoma patients treated with a combination of Refametinib (BAY86-9766) and Nexavar (sorafenib) demonstrated stable disease (PMID: 26644411). 26644411
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Refametinib + Sorafenib Phase I Actionable In a Phase I trial, the combination treatment of Refametinib (BAY86-9766) and Nexavar (sorafenib) in patients with advanced solid tumors resulted in a disease control rate of 65.8% (25/38), specifically, 2.6% (1/38) experienced a partial response and 63.2% (24/38) demonstrated stable disease (PMID: 26644411). 26644411
Unknown unknown colorectal cancer not applicable KIT Inhibitor Refametinib + Sorafenib Phase I Actionable In a Phase I trial, a patient with colorectal cancer demonstrated a durable partial response for 358 days when treated with the combination of Refametinib (BAY86-9766) and Nexavar (sorafenib) (PMID: 26644411). 26644411
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Resminostat + Sorafenib Phase Ib/II Actionable In a Phase I/II trial, the combination of Resminostat (4SC-201) and Nexavar (sorafenib) demonstrated increased efficacy compared to Resminostat (4SC-201) alone in advanced hepatocellular carcinoma patients, resulting in an improved progression-free survival rate of 62.5% vs. 12.5%, a median time to progression of 4.1 vs. 1.8 months, and an overall survival of 8.0 vs. 6.5 months (PMID: 26952006). 26952006
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Selumetinib + Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) and Selumetinib (AZD6244) combination treatment resulted in partial response in 15% (4/27) and stable disease in 48% (13/27) of hepatocellular carcinoma patients (PMID: 27681866). 27681866
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor SF1126 + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of SF1126 and Nexavar (sorafenib) was synergistic or additive in inhibiting proliferation of hepatocellular carcinoma cell lines in culture, and demonstrated increased efficacy in hepatocellular carcinoma cell line xenograft models compared to SF1126 alone (PMID: 23355037). 23355037
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) resulted in an improved median progression-free survival of 167 days in patients with renal cell carcinoma (PMID: 17189398). detail... 17189398
Unknown unknown endometrial carcinoma not applicable KIT Inhibitor Sorafenib Preclinical Actionable In preclinical studies, Nexavar (sorafenib) promoted apoptosis of endometrial carcinoma cells (PMID: 23463670). 23463670
Unknown unknown desmoid tumor not applicable KIT Inhibitor Sorafenib Phase III Actionable In a Phase III trial, Nexavar (sorafenib) treatment resulted in an increased 2-year progression-free survival compared to placebo (81% vs 36%), an objective response rate of 33% (16/49; 1 complete response and 15 partial responses (PR)) compared in 20% (7/25; all PR) with placebo, and a median time to objective response of 9.6 months, compared to 13.3 months with placebo, in patients with refractory desmoid tumors (PMID: 30575484; NCT02066181). 30575484
Unknown unknown thyroid cancer not applicable KIT Inhibitor Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) improved median progression free survival to 10.8 months in metastatic differentiated thyroid cancer patients (PMID: 24768112). detail... 24768112
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) improved median progression free survival to 10.7 months in patients with unresectable hepatocellular carcinoma (PMID: 19144678). 19144678 detail...
Unknown unknown non-small cell lung carcinoma no benefit KIT Inhibitor Sorafenib Phase III Actionable In a Phase III trial, Nexavar (sorafenib) treatment in non-small cell lung carcinoma patients did not reach its primary endpoint, resulting in an overall survival similar to that when treated with placebo, however, did meet its secondary endpoint, demonstrating a greater progression free survival and time to progression when compared to placebo (PMID: 26743856). 26743856
Unknown unknown Her2-receptor negative breast cancer not applicable KIT Inhibitor Sorafenib Clinical Study Actionable In a meta-analysis of 844 ERBB2 (HER2)-negative breast cancer patients, Nexavar (sorafenib) increased progression-free survival time, but not overall survival or objective response rate (PMID: 24940450). 24940450
Unknown unknown thyroid carcinoma not applicable KIT Inhibitor Sorafenib Clinical Study Actionable In a clinical case report, Nexavar (sorafenib) therapy based on in vitro efficacy testing using patient-derived tumor cells resulted in 43 months of disease-free in a patient with anaplastic thyroid carcinoma (PMID: 27379749). 27379749
Unknown unknown thyroid medullary carcinoma not applicable KIT Inhibitor Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) treatment resulted in partial response in 6.3% (1/16) and stable disease in 87.5% (14/16) of patients with sporadic medullary thyroid carcinoma, with a median progression free survival of 17.9 months (PMID: 20368568). 20368568
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) treatment resulted in partial response in 13% (4/31), stable disease in 52% (16/31), a median progression-free survival of 4.9 months and an overall survivals of 9.7 months in gastrointestinal stromal tumor patients who failed prior tyrosine kinase inhibitors (PMID: 22270258). 22270258
Unknown unknown colon cancer not applicable KIT Inhibitor Sorafenib + Fluorouracil Phase I Actionable In a Phase I trial, Nexavar (sorafenib) in combination with Adrucil (fluorouracil) displayed safety and efficacy in advanced solid tumors, including colon cancer (PMID: 22232731). 22232731
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Sorafenib + Fluorouracil Phase I Actionable In a Phase I trial, Nexavar (sorafenib) in combination with Adrucil (fluorouracil) displayed safety and efficacy in advanced solid tumors, including colon cancer (PMID: 22232731). 22232731
Unknown unknown clear cell renal cell carcinoma no benefit KIT Inhibitor Sorafenib + Temsirolimus Phase II Actionable In a Phase II clinical trial, treatment with the combination of Nexavar (sorafenib) and Torisel (temsirolimus) did not prolong progression-free survival compared to treatment with Avastin (bevacizumab) monotherapy (7.4 months vs 7.5 months) in patients with renal clear cell carcinoma (PMID: 26077237). 26077237
Unknown unknown head and neck squamous cell carcinoma not applicable KIT Inhibitor Cetuximab + Dasatinib Phase II Actionable In a Phase II trial, Sprycel (dasatinib) and Erbitux (cetuximab) combination treatment resulted in stable disease in 46% (6/13) and disease progression in 54% (7/13) of patients with cetuximab-resistant head and neck squamous cell carcinoma, with low serum IL6 level correlated with stable disease (PMID: 28559019). 28559019
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Crizotinib + Dasatinib Phase I Actionable In a Phase I trial, Xalkori (crizotinib) therapy, in combination with Sprycel (dasatinib), in patients with advanced solid tumors resulted in limited efficacy, including one patient with a partial response and three patients with stable disease for at least six months or more (PMID: 29047029). 29047029
Unknown unknown rhabdomyosarcoma not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in rhabdomyosarcoma patients (n=13) was suspended due to lack of drug activity (PMID: 26710211). 26710211
Unknown unknown malignant pleural solitary fibrous tumor not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II trial, patients with solitary fibrous tumors demonstrated a median progression free survival of 2 months and five patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown sarcoma not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II clinical trial, 19% (8/42) of patients with undifferentiated pleomorphic sarcoma demonstrated clinical benefit when treated with Sprycel (dasatinib), however the 6 month progression free survival rate in patients was only 12% (6/42) (PMID: 26710211). 26710211
Unknown unknown bone Ewing's sarcoma not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in Ewing sarcoma patients (n=17) was suspended due to lack of drug activity (PMID: 26710211). 26710211
Unknown unknown malignant peripheral nerve sheath tumor not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in patients with malignant peripheral nerve sheath tumors (n=14) was suspended due to lack of drug activity (PMID: 26710211). 26710211
Unknown unknown chordoma not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II trial, patients with chordoma demonstrated a median progression free survival of 6.3 months and six patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown alveolar soft part sarcoma not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II trial, patients with alveolar soft part sarcoma demonstrated a median progression free survival of 11 months and one patient demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor Dasatinib Phase 0 Actionable In a clinical trial, Sprycel (dasatinib) treatment resulted in a 6-month progression-free survival (PFS) rate of 29% (n=48), median PFS of 2.9 months, median overall survival of 19 months, and partial response in 25% (12/48) of patients with Gleevec (imatinib)-resistant gastrointestinal stromal tumor (PMID: 29710216). 29710216
Unknown unknown leiomyosarcoma not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II clinical trial, 13% (6/42) of patients with leiomyosarcoma demonstrated clinical benefit with a median progression free survival of 2.2 months when treated with Sprycel (dasatinib) but were below levels considered to result from drug activity (PMID: 26710211). 26710211
Unknown unknown chronic myeloid leukemia not applicable KIT Inhibitor Dasatinib Clinical Study Actionable In a meta-analysis, Sprycel (dasatinib) treatment was associated with increased rate of major molecular response compared with Gleevec (imatinib) (Odds Ratio (OR): 2.17 [1.66-2.83]), but not improved overall survival (OR: 0.42 [0.14-1.31]), and was associated with increased risk of vascular occlusive events (OR: 3.86 [1.33-11.18]) in patients with chronic myeloid leukemia (PMID: 26847662). 26847662
Unknown unknown liposarcoma not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in liposarcoma patients (n=11) was suspended due to lack of drug activity (PMID: 26710211). 26710211
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II trial, Sprycel (dasatinib) treatment in non-small cell lung carcinoma patients showed some clinical efficacy, resulting in one patient with a partial response, and 12 patients with stable disease, demonstrating a disease control rate of 43% (13/30)(PMID: 20855820). 20855820
Unknown unknown chondrosarcoma not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II trial, patients with chondrosarcoma demonstrated a median progression free survival of 5.5 months and six patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown osteosarcoma not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in osteosarcoma patients (n=46) was suspended due to lack of drug activity (PMID: 26710211). 26710211
Unknown unknown epithelioid sarcoma not applicable KIT Inhibitor Dasatinib Phase II Actionable In a Phase II trial, patients with epithelioid sarcoma demonstrated a median progression free survival of 7.9 months and two patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown chronic myeloid leukemia not applicable KIT Inhibitor Dasatinib + GSK343 Preclinical - Patient cell culture Actionable In a preclinical study, the combination of Sprycel (dasatinib) and GSK343 resulted in increased apoptosis and reduced viability of human primary chronic myeloid leukemia cells in culture, compared to Sprycel (dasatinib) alone (PMID: 27630125). 27630125
Unknown unknown brain glioma not applicable KIT Inhibitor Vandetanib + Dasatinib Phase I Actionable In a Phase I trial, Caprelsa (vandetanib), in combination with dasatinib, demonstrated safety in pediatric patients with intrinsic pontine glioma (PMID: 23536435). 23536435
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Cabozantinib Phase II Actionable In a Phase II clinical trial, Cometriq (cabozantinib) showed safety and anti-tumor activity in several advanced solid tumor types (J Clin Oncol 29: 2011 (suppl; abstr 3010)). detail...
Unknown unknown pancreatic adenocarcinoma not applicable KIT Inhibitor Cabozantinib Preclinical Actionable In a preclinical trial, Cometriq (cabozantinib) promoted apoptosis of pancreactic ductal adenocarcinoma cells (PMID: 23661005). 23661005
Unknown unknown pancreatic endocrine carcinoma not applicable KIT Inhibitor Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) inhibited pancreatic neuroendocrine tumor growth and invasion in transgenic mouse models (PMID: 22585997). 22585997
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Cabozantinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Cabometyx (cabozantinib) resulted in a median progression-free survival of 7.4 months in patients with renal cell carcinoma, compared to 3.8 months with Afinitor (everolimus), and an objective response rate of 22% (17/76) versus 3% (2/77) with Afinitor (everolimus) (PMID: 26406150). 26406150
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cabometyx (cabozantinib) treatment demonstrated improved median progression-free survival (8.2 vs 5.6 months) and overall response rate (46% vs 18%) over Sutent (sunitinib) in untreated patients with metastatic renal cell carcinoma (ESMO 2016 Congress in Copenhagen, Abstract LBA30_PR). detail...
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Cabozantinib FDA approved Actionable In a Phase III trial, final results extending those that supported FDA approval demonstrated Cabometyx (cabozantinib) improved median overall survival compared to Afinitor (everolimus) (21.4 m vs. 16.5 m) and progression-free survival (7.4 m vs. 3.9 m), and led to a 17% (57/330) objective response rate vs. 3% (11/328) with Afinitor (everolimus) in renal cell carcinoma patients (PMID: 27279544). 27279544
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Cabozantinib FDA approved Actionable In a Phase III trial (CELESTIAL) that supported FDA approval, Cabometyx (cabozantinib) significantly improved overall survival (10.2 vs 8.0 months, HR=0.76, p=0.005) and progression-free survival (5.2 vs 1.9 months, HR=0.44, p<0.001) compared to placebo in patients with previously treated advanced hepatocellular carcinoma (PMID: 29972759; NCT01908426). 29972759
Unknown unknown breast cancer not applicable KIT Inhibitor Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) suppressed metastasis, angiogenesis, and tumor growth in mouse models of breast cancer (PMID: 21926191). 21926191
Unknown unknown transitional cell carcinoma not applicable KIT Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in complete response in 2% (1/41), partial response in 17% (7/41), stable disease in 44% (18/41) of urothelial carcinoma patients, with a median progression-free survival of 3.7 months and median overall survival of 8.2 months (J Clin Oncol 34, 2016 (suppl; abstr 4534)). detail...
Unknown unknown uveal melanoma not applicable KIT Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in sttable disease in 61% (14/23) of patients with metastatic uveal melanoma, with a median progression free survival of 4.8 months and an overall survival of 12.6 months (PMID: 28103611). 28103611
Unknown unknown colorectal cancer not applicable KIT Inhibitor Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) showed anti-tumor activity in human colorectal cancer explants (PMID: 25242168). 25242168
Unknown unknown cholangiocarcinoma not applicable KIT Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in a median progression free survival of 1.8 months, and a median overall survival of 5.2 months in patients with advanced cholangiocarcinoma, but also induced grade 3/4 adverse events in 89% (17/19) of the patients (PMID: 28192597). 28192597
Unknown unknown multiple myeloma no benefit KIT Inhibitor Cabozantinib Phase I Actionable In a Phase Ib trial, Cometriq (Cabometyx, cabozantinib) treatment did not demonstrate significant efficacy, resulting in a minimal response in 9% (1/11), stable disease in 73% (8/11), and progression in 18% (2/11) of patients with relapsed and/or refractory multiple myeloma (PMID: 27020089; NCT01866293). 27020089
Unknown unknown thyroid medullary carcinoma not applicable KIT Inhibitor Cabozantinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Cometriq (cabozantinib) resulted in improved progression free survival in patients with metastatic medullary thyroid cancer (PMID: 23319867). detail... 23319867
Unknown unknown ovarian cancer not applicable KIT Inhibitor Cabozantinib Phase II Actionable In a Phase II clinical trial, Cometriq (cabozantinib ) demonstrated safety and efficacy in patients with ovarian cancers (J Clin Oncol 29: 2011 (suppl; abstr 5008)). detail...
Unknown unknown brain glioma not applicable KIT Inhibitor Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) decreased cell proliferation and induced apoptosis in mouse models of glioma (PMID: 21926191). 21926191
Unknown unknown melanoma not applicable KIT Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in partial response in 5% (4/77) and sttable disease in 39% (30/77) of patients with metastatic melanoma, with a median overall progression free survival of 3.8 months (PMID: 28103611). 28103611
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Cabozantinib Phase Ib/II Actionable In a Phase Ib/II trial, Cometriq (cabozantinib) treatment resulted in partial response in 6.7% (1/15) of patients with non-small cell lung carcinoma that had progressed during treatment with Tarceva (erlotinib), with a median progression free survival of 1.9 months (PMID: 28352985). 28352985
Unknown unknown clear cell renal cell carcinoma not applicable KIT Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in improved progression free survival (8.2 v 5.6 months) and objective response rate (46% vs 18%) compared to Sutent (sunitinib) in patients with untreated clear cell metastatic renal cell carcinoma, with a 34% reduction in rate of progression or death (HR=0.66, p=0.012) (PMID: 28199818). 28199818
Unknown unknown gastrointestinal system cancer not applicable KIT Inhibitor Cabozantinib Preclinical - Pdx Actionable In a preclinical study, Cometriq (cabozantinib) demonstrated efficacy in colorectal cancer patient-derived tumor explant models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1). detail...
Unknown unknown renal carcinoma not applicable KIT Inhibitor Cabozantinib Phase II Actionable In a Phase II trial, renal cancer patients treated with Cabozantinib demonstrated a 28 % objective response rate, a 62 % stable disease rate, and a median progression free survival of 14.7 months (PMID: 23292795). 23292795
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Cabozantinib + CT-707 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Cometriq (Cabometyx, cabozantinib) and CT-707 resulted in synergism in hepatocellular carcinoma cells, demonstrating increased apoptosis and inhibition of colony formation in culture and decreased tumor weight in xenograft models (PMID: 27638856). 27638856
Unknown unknown non-small cell lung carcinoma conflicting KIT Inhibitor Cabozantinib + Erlotinib Phase Ib/II Actionable In a Phase Ib/II trial, Cometriq (cabozantinib) and Tarceva (erlotinib) combination treatment resulted in no response (0/13) in patients with non-small cell lung carcinoma that had progressed during treatment with Tarceva (erlotinib) in Phase II, despite an objective response rate of 8.2% (5/61) in Phase I (PMID: 28352985). 28352985
Unknown unknown triple-receptor negative breast cancer not applicable KIT Inhibitor Cabozantinib + Navitoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Navitoclax (ABT-263) and Cometriq (cabozantinib) resulted in a synergistic effect and inhibited the growth of triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown prostate cancer not applicable KIT Inhibitor Cabozantinib + unspecified CTLA4 antibody + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, combination of myeloid-derived suppressor cell-targeting with Cometriq (cabozantinib) and immune checkpoint blockade with anti-CTLA4 and anti-PD-1 antibodies resulted in synergistic inhibition of tumor growth and metastasis in transgenic mouse models of metastatic castration-resistant prostate cancer (PMID: 28321130). 28321130
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor OSI-930 Phase I Actionable In a Phase I trial, 58% (11/19) of patients with a gastrointestinal stromal tumor demonstrated stable disease based on RECIST criteria when treated with OSI-930 (PMID: 23403628). 23403628
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor AGS-003 + Sunitinib Phase II Actionable In a Phase II clinical trial, treatment with the combination of AGS-003 and Sutent (sunitinib) resulted in clinical benefit in 62% (13/21) of patients with advanced renal cell carcinoma, with 9 partial responses and 4 patients achieving stable disease, a median overall survival of 30.2 months, and median progression-free survival if 11.2 months (PMID: 25901286). 25901286
Unknown unknown colon cancer not applicable KIT Inhibitor CVX-060 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of CVX-060 and Sutent (suntinib) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown kidney cancer not applicable KIT Inhibitor CVX-060 + Sunitinib Preclinical Actionable In a preclinical study, the combination of CVX-060 and Sutent (sunitinib) demonstrated a trend improved overall survival compared to single agent Sutent (sunitinib) in mouse models of unresected and resected renal cancer, however, also demonstrated increased toxicity (PMID: 27651308). 27651308
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Everolimus + Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) as first line therapy followed by second line therapy, Afinitor (everolimus), resulted in a greater overall survival (29.5 mo vs 22.4 mo) compared to the reverse treatment of the two therapies in patients with metastatic renal cell carcinoma (PMID: 28327953). 28327953
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Ixabepilone + Sunitinib Phase I Actionable In a Phase I trial, Ixabepilone and Sutent (sunitinib) combination therapy resulted in partial response in 15% (4/27) and stable disease in 48% (13/27) of patients with advanced solid tumors (PMID: 26864210). 26864210
Unknown unknown glioblastoma multiforme not applicable KIT Inhibitor PRX177561 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PRX177561 and Sutent (sunitinib) decreased tumor growth and improved time-to-progression, disease-free survival, and overall survival over either agent alone in glioblastoma cell line xenograft models (PMID: 28057017). 28057017
Unknown unknown brain glioblastoma multiforme not applicable KIT Inhibitor RIST Clinical Study Actionable In two clinical case studies, RIST (rapamycin, irinotecan, sunitinib, temozolomide) resulted in anti-tumor activity in patients with glioblastoma (PMID: 25123598). 25123598
Unknown unknown thyroid cancer not applicable KIT Inhibitor SL327 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of SL327 and Sutent (sunitinib) worked additively to decrease viability, induce apoptosis, and decrease migration of Taxotere (docetaxel)-resistant anaplastic thyroid cancer cell lines in culture, and to inhibit tumor growth in xenograft models (PMID: 28178630) 28178630
Unknown unknown malignant ependymoma not applicable KIT Inhibitor Sunitinib Phase II Actionable In a Phase II clinical trial, Sutent (sunitinib) was well-tolerated in young patients with ependymoma, but did not demonstrate sufficient anti-tumor activity as a single agent, with no patients achieving a sustained objective response (PMID: 27109549). 27109549
Unknown unknown pancreatic endocrine carcinoma not applicable KIT Inhibitor Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, Sutent (sunitinib) demonstrated safety and improved progression free survival in patients with pancreatic neuroendocrine tumors (PMID: 21306237). 21306237 detail...
Unknown unknown lung small cell carcinoma not applicable KIT Inhibitor Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) treatment in small cell lung cancer patients resulted in a partial response of 11% (1/9) and stable disease in 30% (3/9) (PMID: 26716400). 26716400
Unknown unknown glioblastoma multiforme no benefit KIT Inhibitor Sunitinib Phase II Actionable In multiple Phase II clinical trials, Sutent (sunitinib) failed to demonstrate any benefit in patients with glioblastoma with or without concurrent bevacizumab treatment (PMID: 24424564, PMID: 23086433). 23086433 24424564
Unknown unknown colon cancer not applicable KIT Inhibitor Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, Sutent (sunitinib) induced apoptosis in colon cancer cells in culture and in cell line xenograft models (PMID: 22912816). 22912816
Unknown unknown ovarian cancer not applicable KIT Inhibitor Sunitinib Phase Ib/II Actionable In a Phase II trial, Sutent (sunitinib) treatment in ovarian cancer patients resulted in an increased PFS and a response rate of 16.7% (6/36) in those that received Sutent (sunitinib) continuously and a a response rate of 5.4% (2/37) in those that received the drug non-continuously (PMID: 22377563). 22377563 24070205
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Sutent (sunitinib) improved median progression free survival to 27.3 weeks in GIST patients (PMID: 17332278). detail... 17332278
Unknown unknown malignant glioma not applicable KIT Inhibitor Sunitinib Preclinical Actionable In a preclinical study, Sutent (sunitinb) induced cell death and decreased proliferation of glioma cells in culture (PMID: 25458015). 25458015
Unknown unknown malignant glioma not applicable KIT Inhibitor Sunitinib Phase II Actionable In a Phase II clinical trial, Sutent (sunitinib) was well-tolerated in young patients with high grade glioma, but did not demonstrate sufficient anti-tumor activity as a single agent, with no patients achieving a sustained objective response (PMID: 27109549). 27109549
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Sunitinib Phase III Actionable In a Phase III clinical trial, treatment with Sutent (sunitinib) resulted in prolonged disease free survival (HR = 0.761) compared to placebo in post-nephrectomy patients with renal cell carcinoma (ESMO 2016 Congress in Copenhagen, Abstract LBA11_PR). detail...
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Sutent (sunitinib) resulted in improved median progression free survival (47.3 weeks) and objective response rate (27.5%) in patients with renal cell carcinoma (PMID: 19707433). 19707433 detail...
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) treatment in non-small cell lung cancer patients resulted in an objective response rate of 11.1% (7/63), stable disease in 28.6% (18/63), and a PFS of 12 weeks and OS of 23.4 weeks (PMID: 18235126). 18235126
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Sunitinib Phase III Actionable In a Phase III trial, Sutent (sunitinib) as maintenance therapy resulted in improved progression free survival (4.3 vs 2.6 months) but not overall survival (11.7 vs 12.1 months) compared to placebo in patients with stage IIIB/IV non-small cell lung cancer (PMID: 28161554). 28161554
Unknown unknown endometrial cancer not applicable KIT Inhibitor Sunitinib Phase II Actionable In Phase II clinical trials, Sutent (sunitinib) demonstrated efficacy in patients with metastatic or recurrent endometrial carcinoma (PMID: 24882554). 24882554
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Sunitinib + Trametinib Preclinical - Cell line xenograft Actionable In a preclinical study, combination of Mekinist (trametinib) and Sutent (sunitinib) effectively inhibited tumor angiogenesis and growth in cell line xenograft models of Sutent (sunitinib)-refractory renal cell carcinoma (PMID: 26487278). 26487278
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor SU14813 Phase I Actionable In a Phase I trial, SU14813 demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with an objective response rate of 20% (13/65), including 1 complete response and 12 partial responses (PMID: 20605934). 20605934
Unknown unknown acute myeloid leukemia not applicable KIT Inhibitor Bortezomib + Midostaurin + MEC Phase I Actionable In a Phase I trial, Rydapt (midostaurin), in combination with Velcade (bortezomib) and mitoxantrone, Vepesid (etoposide), and Cytosar-U (cytarabine) (MEC), resulted in an overall response rate of 82.5% (19/23) in patients with relapsed or refractory acute myeloid leukemia receiving dose level 3 and above, with complete responses in 56.5% (13/23) of patients (PMID: 26784138). 26784138
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Midostaurin Phase I Actionable In a Phase I trial, Rydapt (midostaurin) demonstrated safety in patients with advanced solid tumors (PMID: 11230495). 11230495
Unknown unknown mast-cell leukemia not applicable KIT Inhibitor Midostaurin FDA approved Actionable In a Phase II trial that supported FDA approval, Rydapt (midostaurin) treatment resulted in an overall response rate of 60% (53/89), a median overall survival of 28.7 months, and a median progression-free survival of 14.1 months in patients with systemic mastocytosis including aggressive systemic mastocytosis, systemic mastocytosis with an associated hematologic neoplasm, and mast-cell leukemia (PMID: 27355533; NCT00782067). 27355533
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Linifanib Phase II Actionable In a Phase II clinical trial, single-agent linifanib was found safe and efficacious in patients with advanced hepatocellular carcinoma (PMID: 22833179). 22833179
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Linifanib + Paclitaxel + Carboplatin Phase II Actionable In a Phase II clinical, Linifanib (ABT-869), in combination with Taxol (paclitaxel) and Paraplatin (carboplatin), increased progression free survival in patients with nonsquamous NSCLC (PMID: 25559798). 25559798
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Bevacizumab + Cediranib Phase I Actionable In a Phase I trial, the combination of Cediranib (AZD-2171) and Avastin (bevacizumab) demonstrated preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 24752867). 24752867
Unknown unknown melanoma no benefit KIT Inhibitor Cediranib Phase II Actionable In a Phase II trial, treatment with Cediranib (AZD-2171) in melanoma patients resulted in only two patients with stable disease at 6 months, no objective responses, and a short median time to progression of 3.5 months thereby demonstrating no benefit (PMID: 26841902). 26841902
Unknown unknown ovarian cancer not applicable KIT Inhibitor Cediranib Phase III Actionable In a Phase III trial, Cediranib (AZD-2171) given with chemotherapy and as maintenance therapy resulted in improved median overall survival (27.3 vs 19.9 months) in platinum-sensitive ovarian cancer patients (J Clin Oncol 35, 2017 (suppl; abstr 5506)). detail...
Unknown unknown endometrial cancer not applicable KIT Inhibitor Cediranib Phase II Actionable In a Phase II clinical trial, treatment with Cediranib (AZD-2171) resulted in an overall response rate of 12.5% (6/48, all partial responses), stable disease in 37.5% (18/48), and a six-month event-free survival rate of 29.2% (14/48) in patients with recurrent or persistent endometrial cancer (PMID: 26186911). 26186911
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor Cediranib Phase II Actionable In a Phase II trial, treatment with Cediranib (AZD-2171) resulted in stable disease as best response in 55% (11/20) gastrointestinal stromal tumor patients, with 8 patients achieving stable disease for greater than or equal to 16 weeks (PMID: 24714778). 24714778
Unknown unknown ovarian cancer not applicable KIT Inhibitor Cediranib + Carboplatin Phase III Actionable In a Phase III clinical trial, the addition of Cediranib (AZD-2171) to platinum-based chemotherapy, including Paraplatin (carboplatin)-based therapy, followed by Cediranib (AZD-2171) maintenance therapy, resulted in an improved median progression-free survival of 11 months versus 8.7 months with platinum-based therapy plus placebo in platinum-sensitive ovarian cancer patients (PMID: 27025186). 27025186
Unknown unknown lung cancer no benefit KIT Inhibitor Cediranib + Carboplatin + Paclitaxel Phase I Actionable In a Phase II clinical trial of patients with advanced NSCLC, the combination of Cediranib (AZD-2171) and carboplatin/paclitaxel (CP) chemotherapy resulted in improved response rate over placebo plus CP, but did not improve progression-free survival, and the study did not proceed to Phase III due to toxicity (PMID: 24360368). 24360368
Unknown unknown female reproductive organ cancer not applicable KIT Inhibitor Cediranib + Durvalumab Phase I Actionable In a Phase I trial, the combination of Imfinzi (durvalumab) and Cediranib (AZD-2171) resulted in a partial response in 50% (6/12) of patients with female reproductive organ cancer (PMID: 28471727). 28471727
Unknown unknown ovarian cancer not applicable KIT Inhibitor Cediranib + Durvalumab + Olaparib Phase I Actionable In a Phase I trial, the combination of Cediranib (AZD-2171), Imfinzi (durvalumab), and Lynparza (olaparib) treatment demonstrated tolerability and activity in female patients with ovarian, endometrial, or triple-negative breast cancer, with a response rate of 33% (3/9; 2 pts with ovarian cancer, and 1 pt with endometrial cancer), and stable disease in 4/9 pts (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #390P; NCT02484404). detail...
Unknown unknown glioblastoma multiforme not applicable KIT Inhibitor Cediranib + Gefitinib Phase II Actionable In a Phase II trial, treatment with the combination of Cediranib (AZD-2171) and Iressa (gefitinib) resulted in a trend toward improved progression-free survival compared to Cediranib (AZD-2171) and placebo (3.6 months vs 2.8 months), and resulted in a response rate of 42% (8/19), compared to 26% (5/19) with Cediranib (AZD-2171) plus placebo in patients with recurrent glioblastoma (PMID: 27232884). 27232884
Unknown unknown lung cancer not applicable KIT Inhibitor Cediranib + Gefitinib Phase I Actionable In a Phase I trial, the combination of Cediranib and Iressa (gefitinib) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including a partial response in a patient with lung cancer (PMID: 20061136). 20061136
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Cediranib + Gemcitabine + Cisplatin Phase I Actionable In a Phase I trial, the combination of Cediranib with Gemzar (gemcitabine) and Platinol (cisplatin) demonstrated preliminary efficacy in patients with advanced non-small cell lung cancer (PMID: 19091548). 19091548
Unknown unknown glioblastoma multiforme not applicable KIT Inhibitor Cediranib + Lomustine Clinical Study Actionable In a clinical case study, a patient with recurrent glioblastoma treated with Cediranib (AZD-2171), in combination with Lomustine (CCNU), demonstrated 50% tumor regression at 6 weeks, complete response at 24 weeks, and achieved clinical remission for over 6 years (PMID: 26929887). 26929887
Unknown unknown ovarian cancer not applicable KIT Inhibitor Cediranib + Olaparib Phase I Actionable In a Phase I clinical trial, the combination therapy of Cediranib (AZD-2171) and Lynparza (olaparib) demonstrated safety and efficacy in patients with ovarian cancer (J Clin Oncol 32:5s, 2014 (suppl; abstr LBA5500)). detail...
Unknown unknown clear cell renal cell carcinoma not applicable KIT Inhibitor Cediranib + Saracatinib Phase II Actionable In a Phase II clinical trial, Saracatinib (AZD0530) did not increase the efficacy of Recentin (cediranib) in patients with metastatic clear-cell renal cell carcinoma (n=69 for both trial arms) (PMID: 26802156). 26802156
Unknown unknown liposarcoma not applicable KIT Inhibitor Doxorubicin + Nilotinib Clinical Study Actionable In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin resulted in 1 partial response and 3 stable disease in 4 patients with liposarcoma, consistent with synergistic growth inhibition in liposarcoma cells in culture (PMID: 30037815; NCT02587169). 30037815
Unknown unknown sarcoma not applicable KIT Inhibitor Doxorubicin + Nilotinib Phase I Actionable In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin demonstrated safety and preliminary efficacy, resulted in 1 partial response and 9 stable disease in 13 patients with sarcomas (PMID: 30037815; NCT02587169). 30037815
Unknown unknown chondrosarcoma not applicable KIT Inhibitor Doxorubicin + Nilotinib Phase I Actionable In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin resulted in 5 stable disease and 2 progressive disease in 7 patients with chondrosarcoma, with a median progression-free survival of 14 months and a median overall survival of 25 months (PMID: 30037815; NCT02587169). 30037815
Unknown unknown leiomyosarcoma not applicable KIT Inhibitor Doxorubicin + Nilotinib Phase I Actionable In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin resulted in 1 short-duration stable disease and 1 progressive disease in 2 patients with leiomyosarcoma, consistent with synergistic growth inhibition in liposarcoma cells in culture and in xenograft models (PMID: 30037815; NCT02587169). 30037815
Unknown unknown chronic myeloid leukemia not applicable KIT Inhibitor Nilotinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Tasigna (nilotinib) resulted in improved hematologic response and overall survival rates compared to treatment with Gleevec (imatinib) in patients with Philadelphia chromosome positive chronic myeloid leukemia (PMID: 21091142). 21091142
Unknown unknown chronic myeloid leukemia not applicable KIT Inhibitor Nilotinib Clinical Study Actionable In a meta-analysis, Tasigna (nilotinib) treatment was associated with increased rate of major molecular response compared with Gleevec (imatinib) (Odds Ratio (OR): 2.45 [1.85-3.24]), but not improved overall survival (OR: 1.51 [0.38-5.99]), and was associated with increased risk of vascular occlusive events (OR: 3.42 [2.07-5.63]) in patients with chronic myeloid leukemia (PMID: 26847662). 26847662
Unknown unknown chronic myeloid leukemia not applicable KIT Inhibitor Nilotinib + Tazemetostat Preclinical Actionable In a preclinical study, treatment with the combination of Tasigna (Nilotinib) and EPZ-6438 resulted in decreased levels of leukemic cells and progenitors in primary chronic myeloid leukemia cell xenograft models, with increased efficacy compared to Tasigna (Nilotinib) alone (PMID: 27630125). 27630125
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Famitinib Phase I Actionable In a Phase I trial, patients with advanced solid tumors had antitumor activity in response to the receptor tyrosine kinase inhibitor, famitinib (PMID: 24043137). 24043137
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Famitinib Phase I Actionable In a Phase I trial, renal carcinoma patients treated with Famitinib demonstrated a disease control rate of 87.5%, which included 50% (12/24) with a partial response and 37.5% (9/24) with stable disease, and a PFS of 10.7 mo and an OS of 33 mo (PMID: 24238512). 24238512
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor DCC-2618 Phase I Actionable In a Phase I study, DCC-2618 treatment resulted in an overall response rate of 16% (16/99) in patients with drug resistant gastrointestinal stromal tumors, 71% (55/77) of analyzed patients harbored baseline KIT or PDGFRA activating mutations (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 11511-11511; NCT02571036). detail...
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Dovitinib Phase III Actionable In a Phase III clinical trial, Dovitinib (TKI258) demonstrated efficacy equivalent to Nexavar (sorafenib) in metastatic renal cell carcinoma patients (PMID: 24556040). 24556040
Unknown unknown renal cell carcinoma not applicable KIT Inhibitor Dovitinib Phase I Actionable In a Phase I trial, Dovitinib (TKI258) demonstrated safety and efficacy resulting in 10% (2/20) partial response and 60% (12/20) stable disease in renal cell carcinoma patients (PMID: 23339124). 23339124
Unknown unknown melanoma not applicable KIT Inhibitor Dovitinib Phase Ib/II Actionable In a Phase I/II study, Dovitinib (TKI258) was demonstrated safe, but of limited clinical benefit in patients with advanced melanoma (PMID: 21976540). 21976540
Unknown unknown adenoid cystic carcinoma not applicable KIT Inhibitor Dovitinib Phase II Actionable In a Phase II trial, Dovitinib (TKI258) treatment was tolerated and demonstrated limited clinical activity in patients with adenoid cystic carcinoma, resulting in partial response in 5.9% (2/34) of patients, suppression of overall tumor growth rate, and a median progression-free survival of 8.2 months (PMID: 28377480). 28377480
Unknown unknown glioblastoma multiforme not applicable KIT Inhibitor Dovitinib Phase I Actionable In a Phase I trial, Dovitinib (TKI258) treatment resulted in a progression free survival rate at 6 months of 16.7% (2/12) in patients with recurrent glioblastoma (PMID: 27100354). 27100354
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor Dovitinib Phase II Actionable In a Phase II clinical trial, Dovitinib (TKI258) demonstrated safety and efficacy in heavily pretreated patients with advanced GISTs (PMID: 24084771). 24084771
Unknown unknown indolent systemic mastocytosis not applicable KIT Inhibitor Avapritinib Phase I Actionable In a Phase I trial, BLU-285 treatment reduced bone marrow mast cell infiltration in a patient with systemic mastocytosis (PMID: 29093181; NCT02561988). 29093181
Unknown unknown triple-receptor negative breast cancer not applicable KIT Inhibitor Apatinib Phase II Actionable In a Phase II trial, 500 mg of Apatinib (YN968D1) produced a median progression-free survival of 3.3 months in TNBC patients (PMID: 24604288). 24604288
Unknown unknown colon cancer not applicable KIT Inhibitor Apatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Apatinib (YN968D1) inhibited tumor growth in colon cancer cell line xenograft models (PMID: 21443688). 21443688
Unknown unknown stomach cancer not applicable KIT Inhibitor Apatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Apatinib (YN968D1) inhibited tumor growth in gastric cancer cell line xenograft models (PMID: 21443688). 21443688
Unknown unknown gastric adenocarcinoma not applicable KIT Inhibitor Apatinib Phase III Actionable In a Phase III trial, Apatinib (YN968D1) treatment significantly improved median overall survival (6.5 vs 4.7 months) and median progression-free survival (2.6 vs 1.8 months) compared to placebo in chemotherapy-refractory patients with advanced gastric or gastroesophageal junction adenocarcinoma (PMID: 26884585). 26884585
Unknown unknown gastroesophageal junction adenocarcinoma not applicable KIT Inhibitor Apatinib Phase III Actionable In a Phase III trial, Apatinib (YN968D1) treatment significantly improved median overall survival (6.5 vs 4.7 months) and median progression-free survival (2.6 vs 1.8 months) compared to placebo in chemotherapy-refractory patients with advanced gastric or gastroesophageal junction adenocarcinoma (PMID: 26884585). 26884585
Unknown unknown lung cancer not applicable KIT Inhibitor Apatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Apatinib (YN968D1) inhibited tumor growth in lung cancer cell line xenograft models (PMID: 21443688). 21443688
Unknown unknown gastrointestinal system cancer not applicable KIT Inhibitor Apatinib Phase III Actionable In a Phase III trial, treatment with Apatinib (YN968D1) at 850mg resulted in a greater progression free survival (2.6 mo vs 1.8 mo) and overall survival (6.5 mo vs 4.7 mo) when compared to placebo in patients with either gastric cancer or gastroesophageal junction adenocarcinoma (PMID: 26884585). 26884585
Unknown unknown gastrointestinal system cancer not applicable KIT Inhibitor Apatinib Phase II Actionable In a Phase II trial, Apatinib (YN968D1) improved progression-free survival and overall survival in metastatic gastric cancer patients (PMID: 23918952). 23918952
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor Apatinib Phase I Actionable In a Phase I trial, Apatinib (YN968D1) produced a partial response in 18.9% (7/37) and stable disease in 64.9% (24/37) of patients with advanced solid tumors, including partial response in one patient with GIST (PMID: 20923544). 20923544
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Apatinib Phase I Actionable In a Phase I trial, Apatinib (YN968D1) demonstrated safety and efficacy in patients with a variety of solid tumor types (PMID: 20923544). 20923544
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Apatinib + Camrelizumab Phase Ib/II Actionable In a Phase Ib trial, combined Camrelizumab (SHR-1210) and Apatinib (YN968D1) treatment resulted in an overall response rate of 50% (8/16), a disease control rate of 93.8% (15/16, stable disease or better), a median progression-free survival (PFS) of 5.8 months, and a 9-month PFS rate of 41.0% in evaluable patients with advanced hepatocellular carcinoma (PMID: 30348638; NCT02942329). 30348638
Unknown unknown gastroesophageal junction adenocarcinoma not applicable KIT Inhibitor Apatinib + Camrelizumab Phase Ib/II Actionable In a Phase Ib trial, combined Camrelizumab (SHR-1210) and Apatinib (YN968D1) treatment resulted in an overall response rate of 17.4% (4/23), a disease control rate of 78.3% (18/23), a median progression-free survival (PFS) of 2.9 months, and an overall survival of 11.4 months in evaluable patients with gastric or gastroesophageal junction cancer (PMID: 30348638; NCT02942329). 30348638
Unknown unknown stomach cancer not applicable KIT Inhibitor Apatinib + Camrelizumab Phase Ib/II Actionable In a Phase Ib trial, combined Camrelizumab (SHR-1210) and Apatinib (YN968D1) treatment resulted in an overall response rate of 17.4% (4/23), a disease control rate of 78.3% (18/23), a median progression-free survival (PFS) of 2.9 months, and an overall survival of 11.4 months in evaluable patients with gastric or gastroesophageal junction cancer (PMID: 30348638; NCT02942329). 30348638
Unknown unknown acute myeloid leukemia not applicable KIT Inhibitor Cytarabine + Daunorubicin + Quizartinib Phase I Actionable In a Phase I trial (QuANTUM-First), the combination therapy, Quizartinib (AC220) with Cytosar-U (cytarabine) and Cerubidine (daunorubicin), resulted in a response in 84% (16/19) of patients with acute myeloid leukemia, including fourteen patients with a composite complete response and two patients achieving morphologic leukemia-free state (PMID: 29139135; NCT01390337). 29139135
Unknown unknown acute myeloid leukemia not applicable KIT Inhibitor Quizartinib Phase II Actionable In a Phase II trial, Quizartinib (AC220) treatment resulted in a composite complete remission (CCR) in 36% (16/44; 1 complete remission (CR)) of FLT3-ITD-negative patients vs. 56% (63/112; 3 CR) of FLT3-ITD-positive patients with relapsed/refractory acute myeloid leukemia (AML) after first-line therapy, and CCR in 30% (12/40; 1 CR) of FLT3-ITD-negative vs. 46% (62/136; 5 CR) in FLT3-ITD-positive patients with relapsed/refractory AML after salvage chemotherapy or transplant (PMID: 29859851; NCT00989261). 29859851
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor PLX9486 Phase I Actionable In a Phase I trial, PLX9486 demonstrated safety and preliminary efficacy, resulted in a progression-free survival of more than 24 weeks and partial response in 8.3% (2/24) of patients with advanced solid tumors, 20 of these patients had gastrointestinal stromal tumor that progressed on Gleevec (imatinib mesylate), and most harbored KIT exon 11 and exon 17 mutations (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 11509-11509; NCT02401815). detail...
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Bevacizumab + Telatinib Phase I Actionable In a Phase I trial, the combination of BAY 57-9352 (telatinib) with Avastin (bevacizumab) in patients with advanced solid tumors resulted in antitumor activity, but demonstrated increasing toxicity over time (PMID: 21378200). 21378200
Unknown unknown kidney cancer not applicable KIT Inhibitor DHA + Regorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, DHA and Stivarga (regorafenib) synergistically inhibited survival of kidney cancer cells in culture and reduced tumor growth in kidney cancer cell line xenograft models (PMID: 26921392). 26921392
Unknown unknown liposarcoma no benefit KIT Inhibitor Regorafenib Phase I Actionable In a Phase II trial, Stivarga (regorafenib) treatment did not result in significant difference in median progression-free survival (1.1 vs 1.7 months) or overall survival (HR=1.57, p=0.21) compared to placebo in patients with liposarcoma (PMID: 27751846). 27751846
Unknown unknown non-small cell lung carcinoma not applicable KIT Inhibitor Regorafenib Phase I Actionable In a Phase I trial, Stivarga (regorafenib) treatment in patients with non-small cell lung cancer resulted in stable disease (SD) in 76% (13/17) of patients, and one patient with SD experienced a progression free survival of 279 days and tumor reduction greater than 30% (J Clin Oncol 28:15s, 2010 (suppl; abstr 7585)). detail...
Unknown unknown synovial sarcoma not applicable KIT Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (5.6 vs 1.0 months), but no difference in overall survival (HR=0.87, p=0.79) compared to placebo in patients with synovial sarcoma (PMID: 27751846). 27751846
Unknown unknown esophageal cancer not applicable KIT Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) improved progression free survival compared to placebo in patients with refractory advanced oesophagogastric cancer (J Clin Oncol 33, 2015 (suppl; abstr 4003)). detail...
Unknown unknown colorectal cancer not applicable KIT Inhibitor Regorafenib FDA approved Actionable In a Phase III clinical trial (CORRECT) that supported FDA approval, Stivarga (regorafenib) demonstrated safety and improved overall survival compared to placebo (6.4 vs 5.0 months, HR=0.77, p=0.0052) in patients with refractory metastatic colorectal cancer (PMID: 23177514; NCT01103323). 23177514 detail...
Unknown unknown colorectal cancer not applicable KIT Inhibitor Regorafenib Phase III Actionable In a Phase III trial (CONSIGN), Stivarga (regorafenib) treatment demonstrated safety profile and efficacy consistent with previous studies, median progression-free survival (PFS) was 2.7 months overall, 2.8 months in KRAS wild-type, and 2.5 months in KRAS mutant colorectal cancer patients, and with no difference in KRAS status between long and short PFS groups (PMID: 30190299; NCT01538680). 30190299
Unknown unknown stomach cancer not applicable KIT Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) improved progression free survival compared to placebo in patients with refractory advanced oesophagogastric cancer (J Clin Oncol 33, 2015 (suppl; abstr 4003)). detail...
Unknown unknown glioblastoma multiforme not applicable KIT Inhibitor Regorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Stivarga, (regorafenib), inhibited tumor growth in cell line xenograft models of glioblastoma multiforme (PMID: 21170960). 21170960
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor Regorafenib FDA approved Actionable In a Phase III clinical trial (GRID) that supported FDA approval, Stivarga (regorafenib) demonstrated safety and improved progression free survival compared to placebo (4.8 vs 0.9 months, HR=0.27, p<0.0001) in patients with gastrointestinal stromal tumors (PMID: 23177515; NCT01271712). detail... 23177515
Unknown unknown sarcoma not applicable KIT Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (2.9 vs 1.0 months), but no difference in overall survival (HR=0.75, p=0.37) compared to placebo in patients with soft tissue sarcoma excluding liposarcoma, leiomyosarcoma, and synovial sarcoma (PMID: 27751846). 27751846
Unknown unknown gastric adenocarcinoma not applicable KIT Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) resulted in a PFS of 2.6 months compared to .9 months with placebo in gastric adenocarcinoma patients (PMID: 27325864). 27325864
Unknown unknown hepatocellular carcinoma not applicable KIT Inhibitor Regorafenib FDA approved Actionable In a Phase III trial (RESORCE) that supported FDA approval, treatment with Stivarga (regorafenib) following Nexavar (sorafenib) treatment resulted in improved overall survival (10.6 vs 7.8 months, HR=0.63) compared to Nexavar (sorafenib) followed by placebo in patients with hepatocellular carcinoma (PMID: 27932229; NCT01774344). 27932229
Unknown unknown leiomyosarcoma not applicable KIT Inhibitor Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (3.7 vs 1.8 months), but no difference in overall survival (HR=0.50, p=0.056) compared to placebo in patients with leiomyosarcoma (PMID: 27751846). 27751846
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Regorafenib Phase I Actionable In a Phase I trial, Stivarga (regorafenib) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22421192). 22421192
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Regorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Stivarga (regorafenib) showed antitumor activity in multiple murine xenograft models derived from melanoma, renal cell carcinoma, colorectal, breast, lung, pancreatic and ovarian tumor cell lines (PMID: 21170960). 21170960
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Regorafenib + Cetuximab Phase I Actionable In a Phase I trial, the combination of Stivarga (regorafenib) and Erbitux (cetuximab) resulted in a clinical benefit of either stable disease or partial response in 46% (11/24) of advanced solid tumor patients, including eight patients with colorectal cancer, and one patient with head and neck cancer, one with carcinoma of unknown primary, and one with glioblastoma (PMID: 28422758; NCT02095054). 28422758
Unknown unknown glioblastoma multiforme not applicable KIT Inhibitor Ponatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Iclusig (ponatinib) induced apoptosis and inhibited growth in glioblastoma cells in culture and in cell line xenograft models (PMID: 25378936). 25378936
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor Ponatinib Phase II Actionable In a Phase II trial, Iclusig (ponatinib) demonstrated preliminary activity in patients with advanced gastrointestinal stromal tumor (J Clin Oncol (Meeting Abstracts) 2014 32: 10506). detail...
Unknown unknown lymphocytic leukemia not applicable KIT Inhibitor Ponatinib FDA approved Actionable In a Phase II clinical trial which supported FDA approval, Iclusig (ponatinib) was effective in promoting disease regression in 52% of patients with accelerated phase chronic myeloid leukemia, 31% of patients with blast phase chronic myeloid leukemia, and 41% of patients with Philadelphia chromosome positive acute lymphoblastic leukemia (PMID: 23935038). detail... 23935038
Unknown unknown chronic myeloid leukemia not applicable KIT Inhibitor Ponatinib FDA approved Actionable In a Phase II clinical trial which supported FDA approval, Iclusig (ponatinib) was effective in promoting disease regression in 52% of patients with accelerated phase chronic myeloid leukemia, 31% of patients with blast phase chronic myeloid leukemia, and 41% of patients with Philadelphia chromosome positive acute lymphoblastic leukemia (PMID: 23935038). detail... 23935038
Unknown unknown chronic myeloid leukemia not applicable KIT Inhibitor Ponatinib Clinical Study Actionable In a meta-analysis, Iclusig (ponatinib) treatment was associated with increased rate of major molecular response compared with Gleevec (imatinib) (Odds Ratio (OR): 4.95 [0.97-25.19]), but not improved overall survival (OR: 2.00 [0.21-19.33]), and was associated with increased risk of vascular occlusive events (OR: 3.47 [1.23-9.78]) in patients with chronic myeloid leukemia (PMID: 26847662). 26847662
Unknown unknown Advanced Solid Tumor not applicable KIT Inhibitor Debio 0617B Preclinical - Patient cell culture Actionable In a preclinical study, Debio 0617B inhibited survival of a variety of patient-derived tumor cells in culture (PMID: 27439479). 27439479
Unknown unknown dermatofibrosarcoma protuberans not applicable KIT Inhibitor Imatinib FDA approved Actionable In a Phase II clinical trial that supported FDA approval, treatment with Gleevec (imatinib) resulted in a median time-to-progression of 23.9 months, and complete response in 33% (4/12) and partial response in 50% (6/12) of patients with dermatofibrosarcoma protuberans (PMID: 18451237). 18451237 detail...
Unknown unknown gastrointestinal stromal tumor not applicable KIT Inhibitor Imatinib Clinical Study Actionable In a retrospective study of 16 Phase I trials, treatment with kinase inhibitors including Gleevec (imatinib mesylate), Sutent (sunitinib), or Stivarga (regorafenib) resulted in stable disease in 47.6% (10/21) and partial response in 19% (4/21) of patients with gastrointestinal stromal tumors (PMID: 27842521). 27842521
Unknown unknown breast cancer no benefit KIT Inhibitor Imatinib Phase II Actionable In a Phase II trial, Gleevec (imatinib mesylate) treatment demonstrated significant toxicity and no clinical benefit in patients with heavily pre-treated metastatic breast cancer, of the 13 tested patients, one was positive for Kit and 4 were positive for Pdgfr (PMID: 15803362). 15803362
Unknown unknown Indication other than cancer not applicable KIT Inhibitor Imatinib FDA approved Actionable Gleevec (imatinib) is FDA approved for patients with aggressive systemic mastocytosis in which c-KIT mutational status is unknown (FDA.gov). detail...
Unknown unknown acute myeloid leukemia not applicable KIT Inhibitor AKN-028 Preclinical - Cell culture Actionable In a preclinical study, AKN-028 treatment induced apoptosis in acute myeloid leukemia cells in culture and inhibited growth and resulted in decreased tumor mass in acute myeloid leukemia cell line xenograft models (PMID: 22864397). 22864397
Unknown unknown acute myeloid leukemia not applicable KIT Inhibitor AKN-028 + Cytarabine Preclinical - Cell culture Actionable In a preclinical study, the sequential treatment of Cytosar-U (cytarabine) and AKN-028 resulted in a syntergistic effect in acute myeloid leukemia cells in culture, demonstrating antileukemic activity (PMID: 22864397). 22864397
Unknown unknown acute myeloid leukemia not applicable KIT Inhibitor AKN-028 + Daunorubicin Preclinical - Cell culture Actionable In a preclinical study, the sequential treatment of Cerubidine (daunorubicin) and AKN-028 resulted in a syntergistic effect in acute myeloid leukemia cells in culture, demonstrating antileukemic activity (PMID: 22864397). 22864397
Clinical Trial Phase Therapies Title Recruitment Status