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Therapy Name | Nilotinib |
Synonyms | |
Therapy Description |
Tasigna (nilotinib) inhibits several tyrosine kinases including BCR-ABL, PDGFR, KIT, DDR and CSF-1R, potentially resulting in reduced tumor cell proliferation (PMID: 21419934, PMID: 25284748). Tasigna (nilotinib) is FDA approved for use in patients with Ph+ (BCR-ABL1) chronic myeloid leukemia (FDA.gov). |
Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
---|---|---|---|---|
Nilotinib | Tasigna | AMN-107|AMN107 | ABL Inhibitor (pan) 8 BCR-ABL Inhibitor 27 CSF1R Inhibitor 25 DDR1 Inhibitor 8 DDR2 inhibitor 7 KIT Inhibitor 51 PDGFR Inhibitor (Pan) 27 | Tasigna (nilotinib) inhibits several tyrosine kinases including BCR-ABL, PDGFR, KIT, DDR and CSF-1R, potentially resulting in reduced tumor cell proliferation (PMID: 21419934, PMID: 25284748). Tasigna (nilotinib) is FDA approved for use in patients with Ph+ (BCR-ABL1) chronic myeloid leukemia (FDA.gov). |
Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
---|---|---|---|---|---|---|---|
KIT V559A | melanoma | sensitive | Nilotinib | Case Reports/Case Series | Actionable | In a Phase II trial, Tasigna (nilotinib) resulted in an overall response rate of 26.2% (11/41), including a melanoma patient harboring KIT V559A demonstrating a partial response and progression-free survival of 19.4 months and overall survival of 32.9 months (PMID: 28327988). | 28327988 |
KIT V559D KIT D820Y | Advanced Solid Tumor | sensitive | Nilotinib | Preclinical - Cell culture | Actionable | In a preclinical study, Tasigna (nilotinib) inhibited proliferation of transformed cells expressing a KIT V559D/D820Y double mutation in culture, with increased potency compared to Sprycel (dasatinib) or Nexavar (sorafenib) (PMID: 17699867). | 17699867 |
KIT W557_K558del | Advanced Solid Tumor | sensitive | Nilotinib | Preclinical - Cell culture | Actionable | In a preclinical study, Tasigna (nilotinib) inhibited growth of transformed cells expressing KIT W557_K558del in culture (PMID: 17699867). | 17699867 |
ABL1 rearrange | myeloid neoplasm | sensitive | Nilotinib | Guideline | Actionable | Tasigna (nilotinib) is included in guidelines for patients with a myeloid/lymphoid neoplasm with eosinophilia harboring a ABL1 rearrangement (NCCN.org). | detail... |
KIT A829P | melanoma | sensitive | Nilotinib | Preclinical | Actionable | In a preclinical study, Tasigna (nilotinib) demonstrated efficacy by inhibiting proliferation of cultured melanoma cells expressing KIT A829P (PMID: 23582185, PMID: 25594040). | 23582185 25594040 |
KIT V560G | Advanced Solid Tumor | sensitive | Nilotinib | Preclinical | Actionable | In a preclinical study, transformed cells expressing KIT V560G demonstrated inhibition of cell proliferation and tumor regression both in culture and in vivo when treated with Tasigna (nilotinib) (PMID: 20442311). | 20442311 |
KIT T670I | melanoma | resistant | Nilotinib | Preclinical | Actionable | In a preclinical study, melanoma cells expressing KIT T670I demonstrated resistance to treatment with Tasigna (nilotinib) (PMID: 23582185, PMID: 25594040). | 23582185 25594040 |
KIT V559D | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) resulted in an overall response rate of 26.2% (11/41) including a melanoma patient harboring KIT V559D demonstrating a partial response and progression free survival of 28.3 months and overall survival of 28.3 months (PMID: 28327988). | 28327988 |
ETV6 - ABL1 | myeloid neoplasm | predicted - sensitive | Nilotinib | Case Reports/Case Series | Actionable | In a clinical study, treatment with Tasigna (nilotinib) in patients with a myeloid neoplasm harboring ETV6-ABL1 resulted in a complete cytogenetic response in three patients, including two patients who had been previously treated with Gleevec (imatinib), one of which who also developed a complete molecular response to Tasigna (nilotinib), and led to a complete hematologic response in another patient (PMID: 32279331). | 32279331 |
PDGFRA Y288C | Advanced Solid Tumor | resistant | Nilotinib | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing PDGFRA Y288C demonstrated resistance to Tasigna (nilotinib) in culture (PMID: 30389923). | 30389923 |
Unknown unknown | chronic myeloid leukemia | not applicable | 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 | 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 |
KIT W557R | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) resulted in an overall response rate of 26.2% (11/41) including a melanoma patient harboring KIT W557R demonstrating a partial response and progression free survival of 35.4 months and overall survival of 35.4 months (PMID: 28327988). | 28327988 |
KIT V560del KIT V654A | Advanced Solid Tumor | sensitive | Nilotinib | Preclinical - Cell culture | Actionable | In a preclinical study, Tasigna (nilotinib) inhibited proliferation of transformed cells expressing a KIT V560del/V654A double mutation in culture (PMID: 17699867). | 17699867 |
ETV6 - ABL1 | chronic myeloid leukemia | predicted - sensitive | Nilotinib | Case Reports/Case Series | Actionable | In a clinical case study, Tasigna (nilotinib) treatment resulted in a complete hematological response after 4 weeks of treatment and sustained complete cytogenetic response and major molecular response at 22 months from diagnosis in a patient with chronic myeloid leukemia harboring ETV6-ABL1, whose disease progressed while on Gleevec (imatinib mesylate) at 6 months from diagnosis (PMID: 24053143). | 24053143 |
KIT exon11 | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) treatment resulted in complete response in 5% (1/19) and partial response in 21% (4/19) of melanoma patients harboring KIT exon 11 or exon 13 mutations (PMID: 28843487; NCT01168050). | 28843487 |
KIT exon11 | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) resulted in an overall response rate of 26.2% (11/41), which included a partial response of 38.5% (10/26) and a progression free survival of 5.4 months in melanoma patients harboring KIT exon 11 mutations (PMID: 28327988). | 28327988 |
PDGFRA D842V | Advanced Solid Tumor | resistant | Nilotinib | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated resistance to Tasigna (nilotinib) in culture (PMID: 30389923). | 30389923 |
PDGFRA D842V | Advanced Solid Tumor | resistant | Nilotinib | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing PDGFRA D842V demonstrated resistance to growth inhibition by Tasigna (nilotinib) in culture (PMID: 18794084). | 18794084 |
KIT W557R KIT L576P | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) resulted in an overall response rate of 26.2% (11/41) including a melanoma patient co-harboring KIT L576P and KIT W557R demonstrating a partial response and a progression free survival of 5.3 months and overall survival of 14.7 months when treated with Tasigna (nilotinib) (PMID: 28327988). | 28327988 |
KIT K642E | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) resulted in an overall response rate of 26.2% (11/41) including a melanoma patient harboring KIT K642E demonstrating a partial response and a progression free survival of 5.8 months and overall survival of 18.6 months (PMID: 28327988). | 28327988 |
KIT W557_K558del KIT T670I | Advanced Solid Tumor | resistant | Nilotinib | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing a KIT W557_K558del/T670I double mutation demonstrated resistance to Tasigna (nilotinib) in culture (PMID: 17699867). | 17699867 |
PDGFRA V561D | Advanced Solid Tumor | sensitive | Nilotinib | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing PDGFRA V561D demonstrated sensitivity to Tasigna (nilotinib) in culture, resulting in decreased cell proliferation (PMID: 30389923). | 30389923 |
KIT D816V | Advanced Solid Tumor | decreased response | Nilotinib | Preclinical | Actionable | In a preclinical study, Tasigna (nilotinib) decreased viability of cells expressing KIT D816V in culture, but did not inhibit KIT phosphorylation or growth of KIT D816V-expressing tumors in mouse models (PMID: 20442311). | 20442311 |
KIT L576P | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) resulted in an overall response rate of 26.2% (11/41) including three melanoma patients harboring KIT L576P demonstrating a partial response (PMID: 28327988). | 28327988 |
KIT S476I | mast-cell leukemia | predicted - sensitive | Nilotinib | Preclinical - Patient cell culture | Actionable | In a preclinical study, treatment with Tasigna (nilotinib) decreased proliferation of patient-derived neoplastic mast cells harboring KIT S476I in culture (PMID: 25209843). | 25209843 |
KIT V560D | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) resulted in an overall response rate of 26.2% (11/41) including a melanoma patient harboring KIT V560D demonstrating a partial response and progression free survival of 8.6 months and overall survival of 23.5 months (PMID: 28327988). | 28327988 |
ABL1 G250R ABL1 G251D | oral cavity cancer | no benefit | Nilotinib | Case Reports/Case Series | Actionable | In a clinical case study, Tasigna (nilotinib) treatment resulted in no benefit in a patient with oral cavity cancer harboring ABL1 G250R and G251D (PMID: 28514312). | 28514312 |
KIT exon13 | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) resulted in an overall response rate of 26.2% (11/41), which included a partial response in 7.7% (1/13) of melanoma patients harboring KIT exon 13 mutations (PMID: 28327988). | 28327988 |
KIT exon13 | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) treatment resulted in complete response in 5% (1/19) and partial response in 21% (4/19) of melanoma patients harboring KIT exon 11 or exon 13 mutations (PMID: 28843487; NCT01168050). | 28843487 |
KIT mutant | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) treatment in patients with melanoma harboring KIT mutations resulted in an overall response rate of 26.2% (11/42), which included 11 patients with a partial response, a median duration of response of 7.1 months, and an overall survival of 18 months (PMID: 28327988; NCT01028222). | 28327988 |
KIT mutant | melanoma | sensitive | Nilotinib | Phase II | Actionable | In a Phase II trial, Tasigna (nilotinib) treatment resulted in complete response in 4% (1/25), durable partial response in 16% (4/25), and stable disease in 56% (14/25) of melanoma patients harboring KIT mutations (PMID: 28843487; NCT01168050). | 28843487 |
KIT exon11 | gastrointestinal stromal tumor | sensitive | Nilotinib | Phase III | Actionable | In a Phase III trial, a 24 month progression free survival was observed in 69.6% of advanced GIST patients harboring KIT exon 11 mutations when treated with Tasigna (nilotinib) (J Clin Oncol May 2013 vol. 31 no. 15_suppl 10501). | detail... |
Clinical Trial | Phase | Therapies | Title | Recruitment Status | Covered Countries | Other Countries |
---|---|---|---|---|---|---|
NCT01244750 | Phase I | Imatinib Dasatinib Nilotinib | Studying First Line Treatment of Chronic Myeloid Leukemia (CML) in a Real-world Setting | Unknown status | USA | 7 |
NCT00471497 | Phase III | Nilotinib Imatinib | A Study of Imatinib Versus Nilotinib in Adult Patients With Newly Diagnosed Philadelphia Chromosome Positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) (ENESTnd) | Completed | USA | CAN | 33 |
NCT01620216 | Phase II | Sunitinib Dasatinib Nilotinib Ponatinib Sorafenib | Validation of an in Vitro Assay to Predict Targeted Therapies for Acute Leukemia Patients | Terminated | USA | 0 |
NCT01844765 | Phase II | Nilotinib | Open Label, Phase II Study to Evaluate Efficacy and Safety of Oral Nilotinib in Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia (CML) Pediatric Patients. | Completed | USA | 12 |
NCT00129740 | Phase II | Nilotinib | Phase II Nilotinib With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia (CML) | Completed | USA | 0 |
NCT03578367 | Phase II | Imatinib Nilotinib Asciminib + Imatinib | Study of Efficacy and Safety of Asciminib in Combination With Imatinib in Patients With Chronic Myeloid Leukemia in Chronic Phase (CML-CP) | Recruiting | USA | CAN | 17 |
NCT02779283 | Phase I | Dasatinib Nilotinib Ponatinib Sorafenib Cytarabine + Idarubicin Sunitinib | Personalized Kinase Inhibitor Therapy Combined With Chemotherapy in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia | Completed | USA | 0 |
NCT01077544 | Phase I | Nilotinib | A Pharmacokinetic (PK) Study of Nilotinib in Pediatric Patients With Philadelphia Chromosome-positive (Ph+) Chronic Myelogenous Leukemia (CML) or Acute Lymphoblastic Leukemia (ALL) | Completed | 4 | |
NCT01140568 | Phase II | Nilotinib | Study of Platelet Derived Growth Factor Receptor (PDGFR) in Recurrent Malignant Gliomas | Completed | USA | 0 |
NCT02709083 | Phase II | Dasatinib Nilotinib Imatinib | Dasatinib or Nilotinib Followed by Imatinib in Patients With Newly Diagnosed, Chronic Phase Chronic Myeloid Leukemia | Terminated | USA | 0 |
NCT02551718 | Phase I | Bosutinib Irinotecan Romidepsin Busulfan Melphalan Nilotinib Crizotinib Cytarabine Mitoxantrone Dasatinib Pazopanib Paclitaxel Clofarabine Hydroxyurea Tretinoin Carfilzomib Nelarabine Bexarotene Pentostatin Everolimus Cabozantinib Mercaptopurine Methotrexate Cladribine Thioguanine Daunorubicin Ponatinib Etoposide Afatinib Gefitinib Gemcitabine Regorafenib Arsenic trioxide Trametinib Imatinib Erlotinib Dabrafenib Decitabine Axitinib Azacitidine Ruxolitinib Fludarabine Lapatinib Ceritinib Sirolimus Sorafenib Lomustine Sunitinib Cabazitaxel Temsirolimus Topotecan Bortezomib Pralatrexate | High Throughput Drug Sensitivity Assay and Genomics- Guided Treatment of Patients With Relapsed or Refractory Acute Leukemia | Active, not recruiting | USA | 0 |
NCT02788201 | Phase II | Erlotinib Thiotepa Imatinib Dacarbazine Arsenic trioxide Idarubicin Mitomycin C Thioguanine Mercaptopurine Methotrexate Cladribine Epirubicin Gemcitabine Doxorubicin Bleomycin Etoposide Gefitinib Daunorubicin Lomustine Sorafenib Sunitinib Ifosfamide Asparaginase Ixabepilone Abiraterone Azacitidine Ruxolitinib Decitabine Axitinib Estramustine Floxuridine Lapatinib Carmustine Fludarabine Nilotinib Cisplatin Vismodegib Vandetanib Melphalan Busulfan Carboplatin Toremifene Crizotinib Dactinomycin Temsirolimus Vorinostat Romidepsin Fluorouracil Irinotecan Bortezomib Tamoxifen Topotecan Chlorambucil Pentostatin Eribulin Carfilzomib Vemurafenib Hydroxyurea Exemestane Vincristine Sulfate Dasatinib Mitoxantrone Vinblastine Cytarabine Tretinoin Clofarabine Teniposide Docetaxel Pazopanib Oxaliplatin Streptozocin Paclitaxel Bendamustine Mechlorethamine Mitotane | Genomic Based Assignment of Therapy in Advanced Urothelial Carcinoma | Completed | USA | 0 |
NCT01698905 | Phase II | Nilotinib | Treatment-free Remission After Achieving Sustained MR4.5 on Nilotinib (ENESTop) | Active, not recruiting | USA | CAN | 16 |
NCT01744665 | Phase II | Nilotinib | Safety and Efficacy of CML Patients Who Switch to Nilotinib and Stop Treatment After Achieving and Sustaining MR4.5. (ENESTgoal) | Completed | USA | 0 |
NCT01784068 | Phase II | Nilotinib | Nilotinib Treatment-free Remission Study in CML (Chronic Myeloid Leukemia) Patients | Active, not recruiting | USA | 18 |
NCT01201538 | Phase II | Nilotinib | A Study of Nilotinib in Growing Vestibular Schwannomas | Terminated | CAN | 0 |
NCT01806571 | Phase II | Cytarabine + Daunorubicin Nilotinib | Daunorubicin Hydrochloride, Cytarabine, and Nilotinib in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia | Completed | USA | 0 |
NCT01028222 | Phase II | Nilotinib | A Study of AMNN107 in the Treatment of Metastatic and/or Inoperable Melanoma Harboring a c-Kit Mutation | Completed | USA | CAN | 12 |
NCT01207492 | Phase II | Nilotinib | Nilotinib in Patients With Relapsed or Metastatic Pigmented Villonodular Synovitis/Tenosynovial Giant Cell Tumor/Diffuse-Type Giant Cell Tumor | Active, not recruiting | USA | 0 |
NCT03654768 | Phase II | Bosutinib Dasatinib Nilotinib Dasatinib + Ruxolitinib Bosutinib + Ruxolitinib Nilotinib + Ruxolitinib | Testing the Addition of Ruxolitinib to the Usual Treatment (Tyrosine Kinase Inhibitors) for Chronic Myeloid Leukemia | Recruiting | USA | 0 |
NCT02353728 | Phase II | Nilotinib | Stem Cell Monitoring for CML Patients Undergoing Nilotinib Therapy | Active, not recruiting | USA | 0 |
NCT01222143 | Phase Ib/II | Cytarabine + Etoposide + Mitoxantrone Nilotinib | Safety and Efficacy Study of Nilotinib Combined With Mitoxantrone, Etoposide, and High-dose Cytarabine Induction Chemotherapy Followed by Consolidation for Patients With C-kit Positive Acute Myeloid Leukemia | Terminated | CAN | 0 |
NCT01407198 | Phase I | Nilotinib | Nilotinib With Radiation for High Risk Chordoma | Active, not recruiting | USA | 0 |