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| Profile Name | FGFR3 mutant |
| Gene Variant Detail | |
| Relevant Treatment Approaches |
| Molecular Profile | Indication/Tumor Type | Response Type | Relevant Treatment Approaches | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|---|
| FGFR3 mutant | cholangiocarcinoma | predicted - sensitive | Erdafitinib | Phase I | Actionable | In a Phase I trial, Balversa (erdafitinib) treatment resulted in an objective response rate of 27% (3/11) in patients with cholangiocarcinoma harboring FGFR genomic alterations, including 1 with FGFR2 mutation, 2 with FGFR3 mutations, and 8 with FGFR2 fusions (PMID: 31088831; NCT01703481). | 31088831 | |
| FGFR3 mutant | transitional cell carcinoma | no benefit | Atezolizumab | Phase II | Actionable | In a Phase II trial (IMVigor 210, CheckMate 275), Tecentriq (atezolizumab) (n=119) treatment resulted in similar response rate (24% vs 21%, p=0.8) in patients with FGFR3 mutant or wild-type metastatic transitional cell carcinoma (PMID: 31272788). | 31272788 | |
| FGFR3 mutant | bladder urothelial carcinoma | sensitive | Infigratinib | Phase I | Actionable | In a Phase I trial, patients with bladder urothelial carcinoma harboring an FGFR3 mutation demonstrated a disease control rate of 75% (6/8) when treated with Infigratinib (BGJ398), including 3 patients with a partial response and 3 with stable disease (PMID: 27870574; NCT01004224). | 27870574 | |
| FGFR3 mutant | transitional cell carcinoma | predicted - sensitive | Docetaxel + Vofatamab | Phase Ib/II | Actionable | In a Phase I/II trial, Vofatamab (B-701) in combination with Taxotere (docetaxel) demonstrated safety and preliminary efficacy, resulted in enhanced activity in patients with locally advanced or metastatic urothelial carcinoma harboring FGFR3 mutations or fusions comparing to wild-type patients (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 4534-4534; NCT02401542). | detail... | |
| FGFR3 mutant | urinary bladder cancer | sensitive | AZD4547 | Preclinical - Cell culture | Actionable | In a preclinical study, AZD4547 inhibited survival of bladder cancer cells harboring FGFR3 mutation in culture (PMID: 27550940). | 27550940 | |
| FGFR3 mutant | Advanced Solid Tumor | sensitive | Pemigatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a variety of cancer cell lines harboring mutations in FGFR1, FGFR2, and/or FGFR3 demonstrated sensitivity to Pemazyre (pemigatinib) in culture and in cell line xenograft models, resulting in inhibition of tumor growth (Cancer Res 2015;75(15 Suppl):Abstract nr 771). | detail... | |
| FGFR3 mutant | Advanced Solid Tumor | sensitive | Infigratinib | Preclinical | Actionable | In a preclinical study, tumor cell lines with FGFR3 mutations demonstrated sensitivity to Infigratinib (BGJ398) in culture (PMID: 23002168). | 23002168 | |
| FGFR3 mutant | transitional cell carcinoma | sensitive | Erdafitinib | Phase I | Actionable | In a Phase I trial, Balversa (erdafitinib) treatment resulted in an objective response rate of 46% (12/26) in patients with urothelial carcinoma harboring FGFR genomic alterations, including 17 with FGFR3 mutations, and 11 with FGFR2 and/or FGFR3 fusions (PMID: 31088831; NCT01703481). | 31088831 | |
| FGFR3 mutant | transitional cell carcinoma | sensitive | Erdafitinib | Guideline | Actionable | Balversa (erdafitinib) is included in the guidelines for patients with advanced or metastatic urothelial carcinoma harboring Fgfr3 alterations after progression on platinum-based regimens (NCCN.org). | detail... | |
| FGFR3 mutant | transitional cell carcinoma | sensitive | Erdafitinib | Case Reports/Case Series | Actionable | In a Phase I trial, Balversa (erdafitinib) treatment resulted in 25% tumor shrinkage at week 8 in an urothelial cancer patient harboring FGFR3 mutations (PMID: 26324363; NCT01703481). | 26324363 | |
| FGFR3 mutant | transitional cell carcinoma | sensitive | Erdafitinib | FDA approved - Has Companion Diagnostic | Actionable | In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations (PMID: 31340094; NCT02365597). | detail... 31340094 detail... | |
| FGFR3 mutant | transitional cell carcinoma | no benefit | Nivolumab | Phase II | Actionable | In a Phase II trial (CheckMate 275), Opdivo (nivolumab) (n=270) treatment resulted in similar response rate (20% vs 21%, p=0.2) in patients with FGFR3 mutant or wild-type metastatic transitional cell carcinoma (PMID: 31272788). | 31272788 | |
| FGFR3 mutant | bladder urothelial carcinoma | sensitive | Dovitinib | Phase II | Actionable | In a Phase II trial, Dovitinib (TKI258) treatment resulted in complete response in 33% (1/3) of patients with BCG-unresponsive, non-muscle-invasive, urothelial carcinoma of the bladder harboring FGFR3 mutations (PMID: 27932416). | 27932416 | |
| FGFR3 mutant | transitional cell carcinoma | sensitive | Infigratinib | Clinical Study | Actionable | In a clinical study, Infigratinib (BGJ398) treatment in patients with FGFR3 alterations led to 25.4% (17/67) confirmed responses and a disease control rate of 64% (43/67), which included complete responses, partial responses, and stable disease, and resulted in a median progression-free survival of 3.75 months and a median overall survival of 7.75 months (PMID: 29848605). | 29848605 | |
| FGFR3 mutant | transitional cell carcinoma | sensitive | Infigratinib | Phase I | Actionable | In a Phase I trial, Infigratinib (BGJ398) treatment resulted in complete response in 4% (1/25) and partial response in 32% (8/25) of urothelial carcinoma patients harboring FGFR3 mutations or fusions (J Clin Oncol 34, 2016 (suppl; abstr 4517); NCT01004224). | detail... | |
| FGFR3 mutant | Advanced Solid Tumor | predicted - sensitive | Debio 1347 | Phase I | Actionable | In a Phase I trial, Debio 1347 treatment resulted in partial response in 10.5% (6/57) and stable disease in 28.1% (16/57) of patients with advanced solid tumors harboring genomic alterations of FGFR1/2/3, including amplifications, fusions, and mutations (PMID: 30745300; NCT01948297). | 30745300 | |
| FGFR3 mutant | bladder urothelial carcinoma | sensitive | Erdafitinib | FDA approved - Has Companion Diagnostic | Actionable | In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations (PMID: 31340094; NCT02365597). | detail... 31340094 detail... | |
| FGFR3 mutant | bladder urothelial carcinoma | sensitive | Erdafitinib | Guideline | Actionable | Balversa (erdafitinib) is included in the guidelines for patients with advanced or metastatic urothelial carcinoma harboring Fgfr3 alterations after progression on platinum-based regimens (NCCN.org). | detail... |
| Clinical Trial | Phase | Therapies | Title | Recruitment Status |
|---|---|---|---|---|
| NCT04003623 | Phase II | Pemigatinib | Efficacy and Safety of Pemigatinib in Participants With Solid Tumors With FGFR Mutations or Translocations (FIGHT-208) | Recruiting |
| NCT01948297 | Phase I | Debio 1347 | Debio 1347-101 Phase I Trial in Advanced Solid Tumours With Fibroblast Growth Factor Receptor (FGFR) Alterations | Active, not recruiting |
| NCT02706691 | Phase II | Infigratinib | Pan FGFR Kinase Inhibitor BGJ398 in Treating Patients With FGFR1-3 Translocated, Mutated, or Amplified Recurrent Head and Neck Cancer | Terminated |
| NCT03822117 | Phase II | Pemigatinib | Efficacy and Safety of Pemigatinib in Previously Treated Locally Advanced/Metastatic or Surgically Unresectable Solid Tumor Malignancies Harboring Activating FGFR Mutations or Translocations (FIGHT-207) | Recruiting |
| NCT02546661 | Phase I | AZD9150 + Durvalumab Durvalumab + Selumetinib AZD4547 Durvalumab + Olaparib Durvalumab AZD4547 + Durvalumab Durvalumab + Vistusertib Adavosertib + Durvalumab | Open-Label, Randomised, Multi-Drug, Biomarker-Directed, Phase 1b Study in Pts w/ Muscle Invasive Bladder Cancer (BISCAY) | Active, not recruiting |
| NCT04233567 | Phase II | Infigratinib | Infigratinib for the Treatment of Advanced or Metastatic Solid Tumors in Patients With FGFR Gene Mutations | Recruiting |
| NCT02312804 | Phase I | Paclitaxel Carboplatin Infigratinib | Ph Ib/BGJ398/Cervix and Other Solid Tumors | Withdrawn |
| NCT02952573 | Phase II | Dexamethasone + Erdafitinib | Testing JNJ-42756493 In Combination With Dexamethasone in Multiple Myeloma That Came Back After a Period of Improvement | Recruiting |
| NCT02529553 | Phase I | LY3076226 | A Study of LY3076226 in Participants With Advanced or Metastatic Cancer | Completed |
| NCT03210714 | Phase II | Erdafitinib | Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial) | Recruiting |
| NCT04083976 | Phase II | Erdafitinib | A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations | Recruiting |
| NCT04045613 | Phase Ib/II | Derazantinib Atezolizumab + Derazantinib | Derazantinib and Atezolizumab in Patients With Urothelial Cancer (FIDES-02) | Recruiting |
| NCT02846766 | Phase II | Lenvatinib | Study of Lenvatinib in Patients With Advanced Cancer and Aberrations in FGF/FGFR Signaling | Withdrawn |
| NCT02393248 | Phase Ib/II | Cisplatin + Gemcitabine + Pemigatinib Docetaxel + Pemigatinib Pemigatinib + Retifanlimab Pemigatinib + Trastuzumab Epacadostat + Pembrolizumab | Open-Label, Dose-Escalation Study of Pemigatinib in Subjects With Advanced Malignancies - (FIGHT-101) | Recruiting |
| NCT02872714 | Phase II | Pemigatinib | A Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Urothelial Carcinoma - (FIGHT-201) | Recruiting |
| NCT03297606 | Phase II | Bosutinib Palbociclib Vismodegib Ipilimumab + Nivolumab Cobimetinib + Vemurafenib Temsirolimus Olaparib Erlotinib Crizotinib Sunitinib Afatinib Dasatinib Pertuzumab + Trastuzumab Axitinib | Canadian Profiling and Targeted Agent Utilization Trial (CAPTUR) (CAPTUR) | Recruiting |
| NCT02693535 | Phase II | Cobimetinib + Vemurafenib Regorafenib Ipilimumab + Nivolumab Palbociclib Afatinib Talazoparib Pembrolizumab Temsirolimus Pertuzumab + Trastuzumab Crizotinib Abemaciclib Sunitinib Olaparib | TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer | Recruiting |
| NCT02558348 | Phase Ib/II | Anlotinib | Phase 1/2a Evaluation of AL3818 in Subjects With Recurrent or Metastatic Endometrial, Ovarian or Cervical Cancer (AL3818-US-001) | Terminated |
| NCT01004224 | Phase I | Infigratinib | A Dose Escalation Study in Adult Patients With Advanced Solid Malignancies | Completed |
| NCT04172675 | Phase II | Erdafitinib Gemcitabine Mitomycin C | A Study of Erdafitinib Versus Investigator Choice of Intravesical Chemotherapy in Participants Who Received Bacillus Calmette-Guérin (BCG) and Recurred With High Risk Non-Muscle-Invasive Bladder Cancer (NMIBC) | Recruiting |
| NCT02401542 | Phase Ib/II | Docetaxel + Vofatamab Vofatamab Docetaxel | Dose Escalation, Expansion Study of Vofatamab (B-701) in Treatment of Locally Advanced or Metastatic Urothelial Cell Carcinoma (FIERCE-21) | Terminated |
| NCT02465060 | Phase II | Erdafitinib Copanlisib Trametinib Crizotinib Sunitinib Sapanisertib Nivolumab AZD4547 Dasatinib Pertuzumab + Trastuzumab Dabrafenib + Trametinib Binimetinib Adavosertib Osimertinib Palbociclib Afatinib Capivasertib Defactinib GSK2636771 Vismodegib Ado-trastuzumab emtansine Larotrectinib Taselisib | Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial) | Recruiting |
| NCT01831726 | Phase II | Dovitinib | Dovitinib for Patients With Tumor Pathway Activations Inhibited by Dovitinib | Completed |
| NCT03155620 | Phase II | Tazemetostat Larotrectinib LY3023414 Vemurafenib Palbociclib Olaparib Ulixertinib Erdafitinib Selumetinib Ensartinib | Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial) | Recruiting |
| NCT04088188 | Phase I | Cisplatin + Gemcitabine + Pemigatinib Cisplatin + Gemcitabine + Ivosidenib | Gemcitabine and Cisplatin With Ivosidenib or Pemigatinib for the Treatment of Unresectable or Metastatic Cholangiocarcinoma | Not yet recruiting |
| NCT02747797 | Phase II | Lucitanib | Lucitanib (E3810) in Patients With Advanced Cancer and FGFR, VEGFR, or PDGFR Pathway Aberrations | Withdrawn |
| NCT02160041 | Phase II | Infigratinib | BGJ398 for Patients With Tumors With FGFR Genetic Alterations | Terminated |
| NCT02608125 | Phase I | PRN1371 | A Dose Escalation Study in Solid Tumors and a Dose Expansion Study of PRN1371 in Adult Patients With Metastatic Urothelial Carcinoma | Active, not recruiting |
| NCT04003610 | Phase II | Pembrolizumab + Pemigatinib Pembrolizumab Carboplatin + Gemcitabine Pemigatinib | Pemigatinib + Pembrolizumab vs Pemigatinib Alone vs Standard of Care for Urothelial Carcinoma (FIGHT-205) | Recruiting |
| NCT01928459 | Phase I | Alpelisib + Infigratinib | Phase 1b Trial of BGJ398/BYL719 in Solid Tumors | Completed |
| NCT02299141 | Phase I | Nintedanib | Nintedanib in Molecularly Selected Patients With Advanced Non-Small Cell Lung Cancer | Active, not recruiting |