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. 2016 Mar 19;7(28):44735–44762. doi: 10.18632/oncotarget.8203

Table 6. Clinical trials related to FGF2/FGFR pathway.

Clinical Trial Description ( Trial #) Participants # Start Date/Trial Status Originator Sponsor Mechanism of Action Study Type/Purpose
FGF2 Inhibitors
Phase II study of low dose Pegintron (PEG interferon alfa-2b) in patients with metastatic melanoma over-expressing FGF2 (NCT00049530) 32 Sept 2003 / not recruiting - ongoing Enzon Pharmaceuticals (Piscataway, NJ) Eastern Cooperative Oncology Group (Boston, MA) FGF2 inhibitor, interferon alpha stimulant Interventional, response level of suppression of plasma FGF2 level with low dose Pegintron
Phase I, open-label, dose-finding study of FP-1039 in advanced solid tumors (NCT00687505) 39 July 2008 / completed Five Prime Therapeutics, Inc. (San Francisco, CA) FGFR1 inhibitor Interventional, assess safety and tolerability
Non-Specific Tyrosine Kinase Inhibitors
Phase I dose escalation study of Lenvima (Lenvatinib) in patients with solid tumors (NCT00280397) 27 Jan 2006 – Nov 2008 / completed Eisai Inc. (Japan) PDGFR-beta inhibitor; c-kit inhibitor; FGFR inhibitor; VEGFR 1-3 inhibitor Interventional; adverse events, safety, tolerability
Phase Ib/II, open-label, multicenter study of Lenvima (lenvatinib) alone, and in combination with Everolimus in subjects with unresectable advanced or metastatic renal cell carcinoma following one prior VEGF-targeted treatment (NCT01136733) 180 Aug 2010 / not recruiting - ongoing Eisai Co. Ltd. (Japan) PDGFR-b antagonist; VEGFR-2 antagonist, FGFR inhibitor Interventional, assess the dose-limiting and maximally tolerated toxicity, recommended dose, progression-free survival
Phase II, multicenter, randomized, open-label study of Votrient (Pazopanib) in thyroid carcinoma (NCT01813136) 168 Mar 2013 / ongoing - recruiting Centre Leon Berard (France) GlaxoSmithKline (Philadelphia, PA) PDGFR antagonist; BRAF inhibitor; c-kit inhibitor; VEGFR 1-3 antagonist Interventional, efficacy (objective response rate)
Phase I/II study of Orantinib for advanced hepatocellular carcinoma (NCT00784290) 35 Sept 2003 / completed Pfizer (New York City, NY) Taiho Pharmaceutical Co., Ltd. (Japan) FGF inhibitor; PDGF inhibitor; VEGFR-2 antagonist Interventional, assess the safety and response rate
Phase II study of Dovitinib in patients with gastrointestinal stromal tumors refractory and/or Intolerant to Imatinib (NCT01478373) 150 Jan 2012 - July 2014 / completed Novartis (East Hanover, NJ) FGF2 inhibitor; PDGFR Δ inhibitor; VEGFR inhibitor Interventional, measure safety and efficacy
Phase II, open-label study of Lucitanib in patients with FGFR1-driven lung cancer (NCT02109016) 40 Apr 2014/ recruiting - ongoing Advenchen Laboratories (Moorpark, CA) Clovis Oncology, Inc. (Boulder, CO) FGFR 1-3 inhibitor; VEGFR1-3 inhibitor Interventional, efficacy (objective response rate)
Phase II study of Vargatef (Nintedanib) in patients with advanced FGFR3 mutated, overexpressed, or wild type urothelial carcinoma of urinary bladder (NCT02278978) 129 Oct 2014/not recruiting - ongoing Boehringer Ingelheim (Germany) National Taiwan University Hospital (Taiwan) PDGFR α-Δ inhibitor; FGFR 1-3 inhibitor; VEGFR 1-3 inhibitor Interventional, safety study with primary purpose of treatment
Phase I/II, multicenter, randomized, double-blind study of Vargatef (Nintedanib) in combination with paclitaxel for treatment of patients with BRAF wild-type metastatic melanoma (NCT02308553) 126 Jan 2015 / ongoing - recruiting Boehringer Ingelheim (Ridgefield, CT) Prof. Dr.Dirk Schadendorf (Germany) PDGFR-alpha/beta inhibitor; FGFR1-3 inhibitor; VEGF1/2 inhibitor Interventional, measure of progression-free survival, safety, tolerability
Phase III study to compare efficacy and safety of Masitinib in combination with Bortezomib and Dexamethasone to placebo in combination with Bortezomib and Dexamethasone in patients with relapsing multiple myeloma (NCT01470131) 300 Apr 2013 Masitinib: AB Science (France) Masitinib: AB Science (France) FGFR modulator; PDGFR antagonist Interventional, assess overall time to progression and overall survival
Bortezomib: Millennium Pharmaceuticals (Cambridge, MA) Immunomodulator; proteasome inhibitor
Dexamethasone: Allergan (Ireland) Glucocorticoid receptor agonist
FGFR Inhibitors
Phase I, multicenter, open label study of oral Debio 1347 (CH5183284) in patients with advanced solid malignancies, whose tumors have an alteration of the FGFR 1, 2 or 3 genes (NCT01948297) 112 Aug 2013 / ongoing - recruiting Chugai Pharmaceutical (Japan) Debiopharm International SA (Switzerland) Interventional, measure of safety and tolerability in dose escalation study
Phase I, open-label, multicenter study of AZD4547 in patients with advanced solid tumors (NCT00979134) 979 Oct 2009 / not recruiting, ongoing AstraZeneca (England) FGFR inhibitor Investigate the safety, tolerability and maximum tolerated dose
Study of AZD4547 in patients with FGFR1 or FGFR2 amplified tumors (NCT01795768) 49 Sept 2012 / ongoing - recruiting Royal Marsden NHS Foundation Trust (England) FGFR inhibitor Interventional, assess efficacy within 8 weeks
Phase I, multi-center, open-label, dose escalation study of oral BGJ398, in adult patients with advanced solid malignancies (NCT01004224) 190 Dec 2009 / ongoing - recruiting Novartis (East Hanover, NJ) FGFR inhibitor Interventional, safety, tolerability, pharmacokinetics, pharmacodynamics
Phase I study of JNJ-42756493 in subjects with advanced or refractory solid tumors or lymphoma (NCT01703481) 260 Jun 2012 / ongoing - recruiting Astex Therapeutic (England) Janssen Research & Development, LLC (Belgium) FGFR inhibitor Interventional, safety, tolerability, pharmacokinetics, pharmacodynamics
Phase I dose escalation study of ARQ 087 in adult subjects with advanced solid tumors (NCT01752920) 120 Dec 2012 / ongoing - recruiting ArQule (Woburn, MA) FGFR inhibitor Interventional, measure of safety and tolerability
Phase I study of LY2874455 in patients with advanced cancer (NCT01212107) 94 Dec 2010 – Feb 2015 / completed Eli Lilly and Company (Indianopolis, IN) FGFR inhibitor Interventional, measure of safety and tolerability
Phase I study of TAS-120 in patients with advanced solid tumors with or without FGF/FGFR-Related abnormalities followed by a Phase II study in patients with advanced solid tumors or multiple myeloma with FGF/FGFR-related abnormalities (NCT02052778) 835 July 2014 / ongoing - recruiting Taiho Oncology, Inc. (Japan) FGFR inhibitor Interventional, measure of safety and tolerability
Phase I, multicenter, open-label study of MFGR1877S in patients with relapsed or refractory multiple myeloma (NCT01122875) 14 Nov 2010 – May 2012 / completed Genentech, Inc. (South San Francisco, CA) FGFR3 inhibitor Interventional, measure of safety and tolerability
Phase I, open-label, dose-escalation study of BAY 1179470 in subjects with advanced, refractory solid tumors (NCT01881217) 63 June 2013 / recruiting - ongoing Bayer (Whippany, NJ) FGFR2 inhibitor Interventional, measure of safety, tolerability, pharmacokinetics, and pharmacodynamics