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REFOCUS: A First-in-Human Study of Highly Selective FGFR2 Inhibitor, RLY-4008, in Patients With ICC and Other Advanced Solid Tumors | Clinical Trials | Clareo Health

COMPLETEDPHASE1/PHASE2

REFOCUS: A First-in-Human Study of Highly Selective FGFR2 Inhibitor, RLY-4008, in Patients With ICC and Other Advanced Solid Tumors

A First-in-Human Study of Highly Selective FGFR2 Inhibitor, RLY-4008, in Patients With Intrahepatic Cholangiocarcinoma (ICC) and Other Advanced Solid Tumors

NCT04526106•Elevar Therapeutics

View on ClinicalTrials.gov

Summary

This is a Phase 1/2, open-label, FIH study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDy), and antineoplastic activity of RLY-4008, a potent and highly selective FGFR2 inhibitor, in patients with unresectable or metastatic cholangiocarcinoma (CCA) and other solid tumors. The study consists of 4 parts: a dose escalation (Part 1), a dose expansion (Part 2), and an extension (Part 3) and a rollover (Part 4).

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Histologically or cytologically confirmed unresectable or metastatic solid tumor
•Documented FGFR2 gene fusion, mutation, or amplification per local testing of blood and/or tumor
•Patient must have measurable disease per RECIST v1.1
•Patient has ECOG performance status of 0-1
•Patient must have disease that is refractory to standard therapy, disease that has not adequately responded to standard therapy, disease for which standard or curative therapy does not exist, or the patient must be intolerant to or have declined standard therapy
•Part 2 dose expansion patients with Cholangiocarcinoma:
•* Group 1: CCA patients with an FGFR2 fusion previously treated with an FGFRi
•* Group 2: CCA patients with an FGFR2 fusion with prior chemotherapy but not previously treated with an FGFRi
•* Group 6: CCA patients with an FGFR2 fusion with no prior chemotherapy and not previously treated with an FGFRi. Prior adjuvant/neo-adjuvant treatment completed \&gt;6 months before enrollment is acceptable. Up to 2 cycles of palliative chemotherapy are allowed during screening

Exclusion Criteria

•Part 2 dose expansion patients with other solid tumors (NOT Cholangiocarcinoma):
•* Group 3: Non-CCA patients with an FGFR2 fusion and not previously treated with an FGFRi.
•* Group 4: Non-CCA patients with an FGFR2 amplification and not previously treated with an FGFRi.
•* Group 5: Non-CCA patients with an FGFR2 mutation and not previously treated with an FGFRi
•Part 3 extension:
•o CCA patients with an FGFR2 fusion with prior chemotherapy but not previously treated with an FGFRi
•Part 4:
•* Patient is receiving RLY-4008 on RLY-4008-101 study and benefiting from treatment as assessed by the investigator.
•Parts 1, 2, and 3
•* Ongoing, clinically significant FGFRi-induced retinal detachment or an ongoing clinically significant corneal or retinal disorder
+7 more criteria

Locations (48)

The University of Alabama at Birmingham

Birmingham, Alabama, United States

Mayo Clinic

Phoenix, Arizona, United States

USC Norris Comprehensive Cancer Center

Los Angeles, California, United States

UCSF Helen Diller Family Comprehensive Cancer Center

San Francisco, California, United States

Mayo Clinic

Jacksonville, Florida, United States

Moffitt Cancer Center

Tampa, Florida, United States

Winship Cancer Institute, Emory University

Atlanta, Georgia, United States

University of Chicago Medical Center

Chicago, Illinois, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

University of Michigan

Ann Arbor, Michigan, United States

Key Dates

Start Date

September 2, 2020

Primary Completion Date

September 5, 2025

Completion Date

September 5, 2025

Last Updated

February 27, 2026

Enrollment

490

ACTUAL participants

Conditions

FGFR2 AmplificationFGFR2 Gene MutationFGFR2 Gene Fusion/RearrangementFGFR2 Gene TranslocationFGFR2 Gene ActivationIntrahepatic CholangiocarcinomaCholangiocarcinomaOther Solid Tumors, Adult

Interventions

RLY-4008

DRUG

Study to Evaluate the Efficacy and Safety of TT-00420 (Tinengotinib) in Cholangiocarcinoma

NCT04919642

CholangiocarcinomaFGFR2 Fusion+3 more

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UNKNOWNPHASE2

Nivolumab in Combination With Chemotherapy for FGFR2-positive Metastatic Gastric Cancer

NCT05859477

Metastatic Gastric CancerPD-L1 Gene Amplification+1 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 27, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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REFOCUS: A First-in-Human Study of Highly Selective FGFR2 Inhibitor, RLY-4008, in Patients With ICC and Other Advanced Solid Tumors

Inclusion Criteria

Exclusion Criteria

Study to Evaluate the Efficacy and Safety of TT-00420 (Tinengotinib) in Cholangiocarcinoma

Nivolumab in Combination With Chemotherapy for FGFR2-positive Metastatic Gastric Cancer

Biomarker Research Study for Patients With FGFR-Mutant Bladder Cancer Receiving Erdafitinib