Futibatinib and Pembrolizumab Combination in the Treatment of Advanced or Metastatic Urothelial Carcinoma

NCT04601857 | Terminated Phase 2 Results FDA Drug

• 2 other identifiers: TAS-120-2032020-000945-15
• Study Type: interventional
• Target: 43
• Locations: 3 countries
• Sites: 18

Brief Summary

The purpose of the trial is to evaluate the antitumor activity and confirm the safety for the combination of Fibroblast Growth Factor Receptor (FGFR) inhibitor futibatinib and anti-programmed cell death-1 (PD-1) antibody pembrolizumab in patients with advanced or metastatic urothelial cancer who are not candidates to receive a platinum-based treatment regimens.

Conditions

Advanced and Metastatic Urothelial Cancer

Keywords

Futibatinib; Pembrolizumab; Urothelial cancer; FGFR; TAS120; MK3475 B04

Outcome Measures

Primary Outcomes (1)

• Objective Response Rate (ORR)

  ORR was defined as the proportion of participants experiencing a best overall response of partial response (PR) or complete response (CR) according to response evaluation criteria in solid tumors, version 1.1 (RECIST 1.1) criteria based on investigator assessment. Evaluation of target lesions defined a CR as the disappearance of all target lesions and non-target lesions (if applicable), and normalization of tumor marker level; PR was defined as at least a 30 percent (%) decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum of diameters for all target lesion assessments. Evaluation of non-target lesions was defined as the persistence of one or more non-target lesion(s) and/or maintenance of tumor marker level above the normal limits. For both target and non-target evaluations, any pathological lymph node must have had a reduction in short axis to less than (\<)10 millimeters (mm).

  Up to 38 months

Secondary Outcomes (5)

• Disease Control Rate (DCR)

  Up to 38 months

• Duration of Response (DOR)

  Up to 38 months

• Progression-free Survival (PFS)

  Up to 38 months

• Overall Survival (OS)

  Up to 38 months

• Number of Participants With Treatment Emergent Adverse Events (TEAEs)

  Up to 38 months

Study Arms (2)

Cohort A

EXPERIMENTAL

Participants with metastatic urothelial carcinoma (UC) and FGFR3 mutation or FGFR1-4 fusion/rearrangement received futibatinib 20 milligrams (mg), orally, once daily (QD), in a 21-day cycle for maximum duration of 590 days along with pembrolizumab 200 mg, intravenously (IV), every 3 weeks (Q3W), in a 21-day cycle for a maximum of 35 doses or a maximum duration of 2 years.

Drug: Futibatinib; Drug: Pembrolizumab

Cohort B

EXPERIMENTAL

Participants with metastatic UC (including participants with other FGFR or non-FGFR genetic aberrations and participants with wild type \[non-mutated\] tumors) received futibatinib 20 mg, orally, QD, in a 21-day cycle for maximum duration of 563 days along with pembrolizumab 200 mg, IV, Q3W in a 21-day cycle for a maximum of 35 doses or a maximum duration of 2 years.

Drug: Futibatinib; Drug: Pembrolizumab

Interventions

Pembrolizumab DRUG

IV

Also known as: KEYTRUDA, MK-3475-B04

Cohort A; Cohort B

Futibatinib DRUG

Oral

Also known as: TAS120

Cohort A; Cohort B

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Willing and able to provide written informed consent for the trial.
• Age ≥ 18 years of age
• Histologically confirmed advanced or metastatic urothelial carcinoma who have not received systemic treatment for advanced metastatic disease.
• Cohort A: must have an FGFR3 mutation or FGFR1-4 fusion/rearrangement.
• Cohort B: all other patients with UC (including patients with other FGFR or non-FGFR genetic aberrations and patients with wild-type \[non-mutated\] tumors)
• Unfit for or intolerant to standard platinum-based chemotherapy.
• Have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 1.
• Adequate organ function.
• Have a measurable disease per RECIST 1.1

You may not qualify if:

• Have received prior therapy with anti-PD-1, anti-PD-L1/L2 agent or FGFR inhibitor.
• History and/or current evidence of any of the following disorders:
• Non-tumor related alteration of the calcium-phosphorus homeostasis that is considered clinically significant in the opinion of the Investigator.
• Ectopic mineralization/calcification considered clinically significant in the opinion of the Investigator.
• Retinal or corneal disorder considered clinically significant in the opinion of the Investigator.
• Has received a live vaccine within 30 days prior to the first dose of study drug.
• Have an active autoimmune disease that has required systemic treatment in the past 2 years.
• Have a history of (noninfectious) pneumonitis that required steroids or has current pneumonitis.
• Have had an allogenic tissue/ organ transplant.
• Has known human immunodeficiency virus (HIV) and/or history of Hepatitis B or C infections, or known to be positive for Hepatitis B antigen (HBsAg)/ Hepatitis B virus (HBV) DNA or Hepatitis C Antibody or RNA.
• Have known active central nervous system metastases and/or carcinomatous meningitis.
• Has a diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy.
• Has severe hypersensitivity (≥Grade 3) to pembrolizumab and/or any of its excipients.

Sponsors & Collaborators

• Taiho Oncology, Inc.: lead
• Merck Sharp & Dohme LLC: collaborator

Study Sites (18)

UCSF Helen Diller Family Comprehensive Cancer Center

San Francisco, California, 94158, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Henry Ford Hospital

Detroit, Michigan, 48202, United States

Location

Comprehensive Care Centers of Nevada

Las Vegas, Nevada, 89148, United States

Location

ICANS - Institut de cancérologie de Strasbourg Europe

Strasbourg, Bas-Rhin, 67200, France

Location

Institut Paoli Calmettes - Hôpital de jour

Marseille, Bouches-du-Rhône, 13273, France

Location

Centre Georges-François Leclerc

Dijon, Côte d'Or, 21079, France

Location

Centre Leon Berard - departement d'oncologie medicale

Lyon, Rhone, 69373, France

Location

Centre Regional de Lutte Contre le Cancer de Lorraine

Vandœuvre-lès-Nancy, 54500, France

Location

Institut De Cancerologie Gustave Roussy

Villejuif, 94805, France

Location

ALTHAIA, Xarxa Assistencial Universitària de Manresa

Manresa, Brcelona, 8243, Spain

Location

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hospital de La Santa Creu i Sant Pau

Barcelona, 8025, Spain

Location

Hospital Universitario Vall d'Hebrón

Barcelona, 8035, Spain

Location

Hospital Universitario Reina Sofia

Córdoba, 14004, Spain

Location

Hospital Universitario HMN Sanchinarro

Madrid, 28050, Spain

Location

Hospital Universitario Marqués de Valdecilla

Santander, 39008, Spain

Location

Hospital la Fe

Valencia, 46026, Spain

Location

MeSH Terms

Interventions

futibatinib; pembrolizumab

Results Point of Contact

• Title: Taiho
• Organization: Taiho Oncology, Inc

medicalinformation@taihooncology.com

844-878-2446

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 15, 2020
• First Posted: October 26, 2020
• Study Start: January 7, 2021
• Primary Completion: March 8, 2024
• Study Completion: September 16, 2025
• Last Updated: December 31, 2025
• Results First Posted: December 31, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

ES (8); FR (6); US (4)