Molecular Profile Detail

Profile Name PDGFRA R748G
Gene Variant Detail

PDGFRA R748G (gain of function)

Relevant Treatment Approaches PDGFR Inhibitor (Pan) PDGFR-alpha 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) Avelumab + Axitinib FDA approved Actionable In a Phase III trial that supported FDA approval (JAVELIN Renal 101), Inlyta (axitinib) plus Bavencio (avelumab) resulted in a median progression-free survival of 13.8 mo. and an objective response rate of 51.4%, vs. 8.4 mo. and 25.7% with Sutent (sunitinib), respectively 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). detail... 30779531
Unknown unknown Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) 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 lung non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 nasopharynx carcinoma not applicable PDGFR Inhibitor (Pan) 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 nasopharynx carcinoma not applicable PDGFR Inhibitor (Pan) 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 thyroid cancer not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 detail...
Unknown unknown renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 breast cancer not applicable PDGFR Inhibitor (Pan) 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 neuroendocrine tumor not applicable PDGFR Inhibitor (Pan) 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 melanoma not applicable PDGFR Inhibitor (Pan) 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 brain glioblastoma multiforme not applicable PDGFR Inhibitor (Pan) 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 Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) 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 ovarian cancer not applicable PDGFR Inhibitor (Pan) Axitinib + Carboplatin + Paclitaxel 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) Axitinib + Carotuximab 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 PDGFR Inhibitor (Pan) Axitinib + Dalantercept 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 gastrointestinal system cancer not applicable PDGFR Inhibitor (Pan) Axitinib + Fluorouracil + Irinotecan + Leucovorin 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 PDGFR Inhibitor (Pan) Axitinib + Fluorouracil + Leucovorin + Oxaliplatin 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 PDGFR Inhibitor (Pan) 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) Axitinib + Pembrolizumab FDA approved Actionable In a Phase III trial (KEYNOTE-426) that supported FDA approval, the combination therapy of Keytruda (pembrolizumab) and Inlyta (axitinib) resulted in a greater 12-month overall survival rate (89.9% vs 78.3%), median progression-free survival (15.1mo vs 11.1mo), and objective response rate (59.3% vs 35.7%) compared to treatment with Sutent (sunitinib) in patients with untreated advanced renal cell carcinoma (PMID: 30779529; NCT02853331). detail... 30779529
Unknown unknown lung non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) 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 glioblastoma multiforme not applicable PDGFR Inhibitor (Pan) Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in stable disease in a patient with glioblastoma (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown renal cell carcinoma not applicable PDGFR Inhibitor (Pan) Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in partial response in 2 patients and stable disease in 1 patient with renal cell carcinoma (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown neuroendocrine tumor not applicable PDGFR Inhibitor (Pan) Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in stable disease ranging from 3-23 months in 10 patients with neuroendocrine tumors (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in partial response in 9.5% (2/21) and stable disease in 57.1% (12/21) of patients with advanced solid tumors (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) Vorolanib Phase I Actionable In a Phase I clinical trial, X-82 was tolerated and demonstrated preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 30, 2012 (suppl; abstr 3041)). detail...
Unknown unknown papillary renal cell carcinoma not applicable PDGFR Inhibitor (Pan) Abexinostat + Pazopanib 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 clear cell renal cell carcinoma not applicable PDGFR Inhibitor (Pan) Abexinostat + Pazopanib 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 Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) Abexinostat + Pazopanib 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 PDGFR Inhibitor (Pan) Abexinostat + Pazopanib 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 Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) Carboplatin + Paclitaxel + 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 sarcoma not applicable PDGFR Inhibitor (Pan) 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 angiosarcoma not applicable PDGFR Inhibitor (Pan) 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 head and neck squamous cell carcinoma not applicable PDGFR Inhibitor (Pan) Cetuximab + Pazopanib 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 clear cell renal cell carcinoma no benefit PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 transitional cell carcinoma not applicable PDGFR Inhibitor (Pan) Paclitaxel + Pazopanib 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 Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) Paclitaxel + Pazopanib 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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). 20100962 detail...
Unknown unknown fibrous histiocytoma not applicable PDGFR Inhibitor (Pan) 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 gastrointestinal stromal tumor not applicable PDGFR Inhibitor (Pan) 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 breast cancer not applicable PDGFR Inhibitor (Pan) 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 uterine corpus sarcoma not applicable PDGFR Inhibitor (Pan) 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 synovial sarcoma not applicable PDGFR Inhibitor (Pan) 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 sarcoma not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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). 22595799 detail...
Unknown unknown lung non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 gastrointestinal neuroendocrine tumor not applicable PDGFR Inhibitor (Pan) 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 uterus leiomyosarcoma not applicable PDGFR Inhibitor (Pan) 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 liposarcoma not applicable PDGFR Inhibitor (Pan) 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 Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 germ cell cancer not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 clear cell renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 leiomyosarcoma not applicable PDGFR Inhibitor (Pan) 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 renal cell carcinoma no benefit PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 lung non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) Carboplatin + Lenvatinib + 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 metastatic renal cell carcinoma who had progressed after one previous VEGF-targeted therapy (PMID: 26482279; NCT01136733). 26482279 detail...
Unknown unknown renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 colon cancer not applicable PDGFR Inhibitor (Pan) 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 hepatocellular carcinoma not applicable PDGFR Inhibitor (Pan) 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 hepatocellular carcinoma not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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). detail... 29433850
Unknown unknown thyroid medullary carcinoma not applicable PDGFR Inhibitor (Pan) 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 sarcoma not applicable PDGFR Inhibitor (Pan) 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 Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) 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 Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) 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 thyroid cancer not applicable PDGFR Inhibitor (Pan) Lenvatinib FDA approved Actionable In a Phase III trial (SELECT) that supported FDA approval, treatment with Lenvima (lenvatinib) improved progression free survival (18.3 vs 3.6 months, HR=0.21, p<0.001) and response rates (64.8% vs 1.5%, p<0.001) compared to placebo in patients with radioiodine-refractory differentiated thyroid cancer (PMID: 25671254; NCT01321554). 25671254 detail...
Unknown unknown thyroid cancer not applicable PDGFR Inhibitor (Pan) 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 lung non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 papillary thyroid carcinoma not applicable PDGFR Inhibitor (Pan) 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 melanoma not applicable PDGFR Inhibitor (Pan) 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 lung non-squamous non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) Carboplatin + Motesanib 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 non-small cell lung cancer (PMID: 24419239; NCT00460317). 24419239
Unknown unknown lung non-squamous non-small cell carcinoma no benefit PDGFR Inhibitor (Pan) Carboplatin + Motesanib + Paclitaxel 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 lung non-squamous non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 non-small cell lung cancer (PMID: 24419239; NCT00460317). 24419239
Unknown unknown head and neck squamous cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 epithelioid sarcoma not applicable PDGFR Inhibitor (Pan) 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 chordoma not applicable PDGFR Inhibitor (Pan) 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 rhabdomyosarcoma not applicable PDGFR Inhibitor (Pan) 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 peripheral nerve sheath tumor not applicable PDGFR Inhibitor (Pan) 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 sarcoma not applicable PDGFR Inhibitor (Pan) 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 chondrosarcoma not applicable PDGFR Inhibitor (Pan) 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 malignant pleural solitary fibrous tumor not applicable PDGFR Inhibitor (Pan) 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 liposarcoma not applicable PDGFR Inhibitor (Pan) 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 bone Ewing's sarcoma not applicable PDGFR Inhibitor (Pan) 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 gastrointestinal stromal tumor not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 alveolar soft part sarcoma not applicable PDGFR Inhibitor (Pan) 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 lung non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 osteosarcoma not applicable PDGFR Inhibitor (Pan) 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 chronic myeloid leukemia not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) Dasatinib + Vandetanib 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 kidney cancer not applicable PDGFR Inhibitor (Pan) 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 colon cancer not applicable PDGFR Inhibitor (Pan) 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 brain glioblastoma multiforme not applicable PDGFR Inhibitor (Pan) Irinotecan + Sirolimus + Sunitinib + Temozolomide 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 Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 thyroid cancer not applicable PDGFR Inhibitor (Pan) 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 pancreatic endocrine carcinoma not applicable PDGFR Inhibitor (Pan) 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 endometrial cancer not applicable PDGFR Inhibitor (Pan) 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 glioblastoma multiforme no benefit PDGFR Inhibitor (Pan) 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 malignant ependymoma not applicable PDGFR Inhibitor (Pan) 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 gastrointestinal stromal tumor not applicable PDGFR Inhibitor (Pan) 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). 17332278 detail...
Unknown unknown ovarian cancer not applicable PDGFR Inhibitor (Pan) 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, PMID: 24070205). 22377563 24070205
Unknown unknown colon cancer not applicable PDGFR Inhibitor (Pan) 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 malignant glioma not applicable PDGFR Inhibitor (Pan) 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 malignant glioma not applicable PDGFR Inhibitor (Pan) 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 lung small cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 lung non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 lung non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 pancreatic ductal adenocarcinoma not applicable PDGFR Inhibitor (Pan) 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 colorectal cancer not applicable PDGFR Inhibitor (Pan) ABT-348 Preclinical - Cell culture Actionable In a preclinical study, ABT-348 reduced proliferation of colorectal cancer cell lines in culture (PMID: 22935731). 22935731
Unknown unknown hematologic cancer not applicable PDGFR Inhibitor (Pan) ABT-348 Phase I Actionable In a Phase I trial, ABT-348 (Ilorasertib) demonstrated safety and preliminary efficacy in patients with various hematological malignancies (PMID: 25933833). 25933833
Unknown unknown acute lymphocytic leukemia not applicable PDGFR Inhibitor (Pan) ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, Ilorasertib (ABT-348) displayed efficacy in acute lymphoblastic leukemia cell line xenograft models (PMID: 22935731). 22935731
Unknown unknown multiple myeloma not applicable PDGFR Inhibitor (Pan) ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, Ilorasertib (ABT-348) displayed efficacy in multiple myeloma cell line xenograft models (PMID: 22935731). 22935731
Unknown unknown fibrosarcoma not applicable PDGFR Inhibitor (Pan) ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, Ilorasertib (ABT-348) inhibited tumor growth in cell line xenograft models of fibrosarcoma (PMID: 22935731). 22935731
Unknown unknown pancreatic carcinoma not applicable PDGFR Inhibitor (Pan) ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with Ilorasertib (ABT-348) inhibited tumor growth and led to regression of advanced tumors in cell line xenograft models of pancreatic carcinoma (PMID: 22935731). 22935731
Unknown unknown Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) ABT-348 Preclinical - Pdx & cell culture Actionable In a preclinical study, ABT-348 (Ilorasertib) inhibited proliferation and promoted apoptosis in various solid tumor cell culture and Pdx models, including NSCLC, ovarian, and colon (AACR; Mol Cancer Ther 2011;10(11 Suppl):Abstract nr B231). detail...
Unknown unknown renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 renal cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 gastrointestinal stromal tumor not applicable PDGFR Inhibitor (Pan) 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 melanoma not applicable PDGFR Inhibitor (Pan) 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 glioblastoma multiforme not applicable PDGFR Inhibitor (Pan) 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 adenoid cystic carcinoma not applicable PDGFR Inhibitor (Pan) 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 Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) 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 chondrosarcoma not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 sarcoma not applicable PDGFR Inhibitor (Pan) 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 liposarcoma not applicable PDGFR Inhibitor (Pan) 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 chronic myeloid leukemia not applicable PDGFR Inhibitor (Pan) 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 detail...
Unknown unknown chronic myeloid leukemia not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 ovarian cancer not applicable PDGFR Inhibitor (Pan) Carboplatin + Nintedanib + Paclitaxel Phase III Actionable In a Phase III clinical trial, patients with advanced ovarian cancer treated with Ofev (nintedanib), in combination with Paraplatin (carboplatin) and Taxol (paclitaxel), experienced a progression free survival of 17.2 months versus 16.6 months in patients treated with a combination of placebo, carboplatin, and paclitaxel in first-line treatment (PMID: 26590673). 26590673
Unknown unknown lung non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) Docetaxel + Nintedanib Phase III Actionable In a Phase III clinical trial, the combination of Ofev (nintedanib) and Taxotere (docetaxel) resulted in improved progression-free survival and overall survival compared to placebo plus Taxotere (docetaxel) in non-small cell lung cancer patients (PMID: 24411639). 24411639
Unknown unknown lung adenocarcinoma not applicable PDGFR Inhibitor (Pan) Docetaxel + Nintedanib Phase III Actionable In a Phase III trial, the combination of Ofev (nintedanib) and Taxotere (docetaxel) resulted in an improved overall survival in lung adenocarcinoma patients, particularly those patients with shorter time to progression after first line chemotherapy, which included measures that were time from start or time from end of first line chemotherapy (PMID: 28702806). 28702806
Unknown unknown pancreatic cancer not applicable PDGFR Inhibitor (Pan) Nintedanib Preclinical - Cell line xenograft Actionable In a preclinical study, Nintedanib, alone or with chemotherapy, inhibited tumor growth in cell line xenograft models of lung and pancreatic cancer but not in cell culture (PMID: 23729403). 23729403
Unknown unknown lung cancer not applicable PDGFR Inhibitor (Pan) Nintedanib Preclinical - Cell line xenograft Actionable In a preclinical study, Nintedanib, alone or with chemotherapy, inhibited tumor growth in cell line xenograft models of lung and pancreatic cancer but not in cell culture (PMID: 23729403). 23729403
Unknown unknown Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) Nintedanib Phase I Actionable In a Phase I trial, Ofev (nintedanib) demonstrated safety and some preliminary efficacy in patients with advanced solid tumors (PMID: 25795637). 25795637
Unknown unknown pancreatic endocrine carcinoma not applicable PDGFR Inhibitor (Pan) Nintedanib Preclinical Actionable In a preclinical study, Ofev (nintedanib) induced tumor cell apoptosis, decreased microvessel density, inhibited tumor growth, and improved survival in transgenic mouse models of pancreatic neuroendocrine carcinoma (PMID: 26206868). 26206868
Unknown unknown ovarian cancer not applicable PDGFR Inhibitor (Pan) Nintedanib Phase I Actionable In a Phase I clinical trial, Vargatef (nintedanib) demonstrated safety in patients with ovarian cancers, clinical trials to determine efficacy in these patients are ongoing (PMID: 19889612). 19889612
Unknown unknown colorectal cancer not applicable PDGFR Inhibitor (Pan) Nintedanib Phase I Actionable In a Phase I trial, Ofev (nintedanib) demonstrated safety and efficacy in patients with advanced colorectal cancer (PMID: 25012508). 25012508
Unknown unknown Indication other than cancer not applicable PDGFR Inhibitor (Pan) Nintedanib FDA approved Actionable Ofev (nintedanib) is FDA approved for use in patients with idiopathic pulmonary fibrosis (FDA.gov). detail... detail...
Unknown unknown lung adenocarcinoma not applicable PDGFR Inhibitor (Pan) Nintedanib Phase III Actionable In a Phase III clinical trial, the combination of Ofev (nintedanib) and Taxotere (docetaxel) improved progression-free survival and overall survival in patients with lung adenocarcinoma compared to Taxotere (docetaxel) alone (PMID: 24411639). 24411639
Unknown unknown brain glioma not applicable PDGFR Inhibitor (Pan) Nintedanib Phase II Actionable In a Phase II clinical trial, Ofev (nintedanib) failed to show any efficacy in patients with recurrent high-grade glioma, regardless of prior bevacizumab therapy (PMID: 25338318). 25338318
Unknown unknown esophageal cancer not applicable PDGFR Inhibitor (Pan) PP-121 Preclinical - Cell line xenograft Actionable In a preclinical study, PP-121 inhibited proliferation and growth of esophageal cancer cells in culture and in cell line xenograft models (PMID: 26235881). 26235881
Unknown unknown Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) Cetuximab + Regorafenib 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 kidney cancer not applicable PDGFR Inhibitor (Pan) 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 colorectal cancer not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 Advanced Solid Tumor not applicable PDGFR Inhibitor (Pan) 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 PDGFR Inhibitor (Pan) 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 sarcoma not applicable PDGFR Inhibitor (Pan) 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 leiomyosarcoma not applicable PDGFR Inhibitor (Pan) 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 lung non-small cell carcinoma not applicable PDGFR Inhibitor (Pan) 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 esophageal cancer not applicable PDGFR Inhibitor (Pan) 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 liposarcoma no benefit PDGFR Inhibitor (Pan) 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 glioblastoma multiforme not applicable PDGFR Inhibitor (Pan) 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 gastric adenocarcinoma not applicable PDGFR Inhibitor (Pan) 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 synovial sarcoma not applicable PDGFR Inhibitor (Pan) 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 stomach cancer not applicable PDGFR Inhibitor (Pan) 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 gastrointestinal stromal tumor not applicable PDGFR Inhibitor (Pan) 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 hepatocellular carcinoma not applicable PDGFR Inhibitor (Pan) 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). detail... 27932229
Unknown unknown breast cancer no benefit PDGFR Inhibitor (Pan) 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 gastrointestinal stromal tumor not applicable PDGFR Inhibitor (Pan) 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 dermatofibrosarcoma protuberans not applicable PDGFR Inhibitor (Pan) 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 Indication other than cancer not applicable PDGFR Inhibitor (Pan) 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... detail...
Unknown unknown indolent systemic mastocytosis not applicable PDGFR-alpha 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 lymphocytic leukemia not applicable PDGFR-alpha 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). 23935038 detail...
Unknown unknown gastrointestinal stromal tumor not applicable PDGFR-alpha 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 glioblastoma multiforme not applicable PDGFR-alpha 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 chronic myeloid leukemia not applicable PDGFR-alpha 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). 23935038 detail...
Unknown unknown chronic myeloid leukemia not applicable PDGFR-alpha 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 acute myeloid leukemia not applicable PDGFR-alpha 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 PDGFR-alpha 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 breast cancer not applicable PDGFR-alpha Inhibitor Lucitanib Phase II Actionable In a Phase II trial, Lucitanib (E-3810) demonstrated acceptable toxicity and activity, resulted in a median progression-free survival of 3.5 and 2.6 months for 10mg and 15mg treatment groups respectively, with no differences in response correlated with FGFR1 and 11q amplification status in metastatic breast cancer patients (Cancer Res 2017;77(4 Suppl):Abstract nr P6-11-03). detail...
Unknown unknown triple-receptor negative breast cancer not applicable PDGFR-alpha Inhibitor Lucitanib Preclinical Actionable In a preclinical study, Lucitanib (E-3810) demonstrated antitumor activity in mouse xenograft models of triple negative breast cancer with synergistic effects noted when using Lucatinib plus chemotherapy (PMID: 23270924). 23270924
Unknown unknown colon cancer not applicable PDGFR-alpha 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 hepatocellular carcinoma not applicable PDGFR-alpha 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 stomach cancer not applicable PDGFR-alpha 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 lung non-small cell carcinoma not applicable PDGFR-alpha 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 Advanced Solid Tumor not applicable PDGFR-alpha Inhibitor Amuvatinib + Carboplatin + Paclitaxel 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 gastrointestinal stromal tumor not applicable PDGFR-alpha 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...
Clinical Trial Phase Therapies Title Recruitment Status