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Futibatinib in Patients With Specific FGFR Aberrations

Phase 2
Completed
Conditions
Advanced or Metastatic Gastric or Gastroesophageal Cancer
Advanced or Metastatic Solid Tumor
Myeloid or Lymphoid Neoplasms (MLN)
Interventions
Registration Number
NCT04189445
Lead Sponsor
Taiho Oncology, Inc.
Brief Summary

The purpose of this study is to evaluate the efficacy and safety of futibatinib in patients with FGFR aberrations in 3 distinct cohorts. Patients will be enrolled into one of 3 cohorts: patients with advanced, metastatic or locally-advanced solid tumors harboring FGFR1-4 rearrangements (excluding primary brain tumors and intrahepatic cholangiocarcinoma \[iCCA\]); patients with gastric or gastro-esophageal junction (GEJ) cancer harboring FGFR2 amplification; and patients with myeloid or lymphoid neoplasms with FGFR1 rearrangements.

Detailed Description

Study TAS-120-202 is an open-label, multinational, 3-arm Phase 2 study evaluating the efficacy, safety, tolerability, PK, and pharmacodynamics of futibatinib in patients with FGFR aberrations. Eligible patients will be assigned to 1 of 3 treatment cohorts based on diagnosis and FGFR gene aberration status.

Patients will receive futibatinib at an oral dose of 20 mg once a day on a continuous 28-day cycle.

The study will enroll approximately:

* Cohort A: 60 patients with locally advanced, advanced, or metastatic solid tumor harboring FGFR rearrangements other than primary brain tumor or iCCA;

* Cohort B: 35 patients with locally-advanced, advanced, or metastatic gastric cancer or gastro-esophageal junction (GEJ) with FGFR2 amplification;

* Cohort C: 20 patients with myeloid or lymphoid neoplasms (MLN) with FGFR1 rearrangements

Treatment in all cohorts will continue until disease progression, unacceptable toxicity, or any other of the criteria for treatment discontinuation is met. For patients who discontinue treatment for reasons other than disease progression, tumor assessments should be continued until radiologic disease progression is documented or until initiation of subsequent new anticancer therapy (whichever occurs first).

Patients will be followed for survival every 12 weeks (±2 weeks) until survival events (deaths) have been reported for 75% of enrolled patients or the study is terminated early by the Sponsor.

Additional cohorts may be added in the future in case of new emerging efficacy data.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
115
Inclusion Criteria
  1. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1

  2. Known FGFR aberration status and tumor type that meet all of the criteria for 1 of the following cohorts:

    a. Cohort A

    i. Histologically-confirmed, locally-advanced, advanced, or metastatic solid tumors harboring a FGFR1-4

ii. Measurable disease per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1

iii. Had disease progression/recurrence after standard treatment for their cancer

b. Cohort B

i. Histologically-confirmed, locally-advanced, advanced, or metastatic gastric or gastroesophageal junction cancer harboring a FGFR2 amplification.

ii. Measurable disease per RECIST 1.1

iii. Received at least 2 prior systemic regimens for advanced/metastatic disease

iv. Experienced disease progression/recurrence during or after the most recent prior systemic treatment for advanced/metastatic gastric cancer or GEJ cancer

c. Cohort C

i. Confirmed myeloid or lymphoid neoplasms as defined by WHO criteria with a FGFR1 rearrangement

ii. Not a candidate for hematological stem cell transplant (HSCT) or relapsed after HSCT and donor lymphocyte infusion, and progressed and not a candidate for other therapies

Exclusion Criteria
  1. History and/or current evidence of any of the following disorders:

    1. Non-tumor related alteration of the calcium-phosphorus homeostasis that is considered clinically significant in the opinion of the Investigator
    2. Ectopic mineralization/calcification including, but not limited to, soft tissue, kidneys, intestine, or myocardia and lung, considered clinically significant in the opinion of the Investigator
    3. Retinal or corneal disorder confirmed by retinal/corneal examination and considered clinically significant in the opinion of the Investigator.
  2. Prior treatment with an FGFR inhibitor

  3. Brain metastases that are untreated or clinically or radiologically unstable (that is, have been stable for <1 month)

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Futibatinib (Cohort A)FutibatinibAdvanced or metastatic solid tumors harboring FGFR1-4 rearrangements
Futibatinib (Cohort C)FutibatinibMyeloid or lymphoid neoplasm harboring FGFR1 rearrangement
Futibatinib (Cohort B)FutibatinibAdvanced or metastatic solid gastric or GEJ cancer harboring FGFR2 amplification
Primary Outcome Measures
NameTimeMethod
Objective response rate (ORR) in Cohorts A and BApproximately 6 months

ORR, defined as the proportion of patients experiencing a best overall response of partial response (PR) or complete response (CR) (per Response Evaluation Criteria in Solid Tumors, RECIST version 1.1), based on independent central review of radiological images.

Complete response (CR) rate in Cohort CApproximately 6 months

CR rate, defined as the proportion of patients who achieved a CR (per response criteria for MLN) based on investigators' assessment of imaging, peripheral blood, and bone marrow.

Secondary Outcome Measures
NameTimeMethod
Duration of CR in Cohort CApproximately 6 months

Duration of CR, defined as the time from the first documentation of CR to the first documentation of recurrence or death due to any cause, whichever occurs first.

Disease control rate (DCR) in Cohort A and BApproximately 6 months

DCR, defined as the proportion of patients experiencing a best overall response of stable response (SD), PR, or complete response (CR) (per RECIST 1.1).

ORR based on investigator assessment ORR in Cohorts A and BApproximately 6 months

ORR, defined as the proportion of patients experiencing a best overall response of partial response (PR) or complete response (CR) (per RECIST 1.1), based on investigator assessment.

Overall Survival (OS) in Cohorts A, B and CApproximately 12 months

OS, defined as the time from the date of first dose to the death date.

Duration of CR+CRi in Cohort CApproximately 6 months

Duration of CR+CRi, defined as the time from the first documentation of CR or CRi to the first documentation of disease relapse or death due to any cause, whichever occurs first.

Partial cytogenetic response (PCyR) rate in Cohort CApproximately 6 months

PCyR rate, defined as the proportion of patients who achieved a PCyR

Duration of Response (DOR) in Cohorts A, B and CApproximately 6 months

DOR, defined as the time from the first documentation of response (CR or PR in Cohorts A and B; CR, PR, or CRi in Cohort C) to the first documentation of objective tumor progression or death due to any cause, whichever occurs first.

Complete cytogenetic response (CCyR) rate in Cohort C.Approximately 6 months

CCyR rate, defined as the proportion of patients who achieved a CCyR

To assess the safety and tolerability in Cohorts A, B and CApproximately 6 months

Safety based on reported AEs will be graded according to the National Cancer Institute- Common Terminology Criteria for Adverse events (NCI-CTCAE), Version 5.0.

Progression- free survival (PFS) in Cohorts A, B and CApproximately 6 months

PFS, defined as the time from first dose of the study therapy to the date of death (any cause) or disease progression, whichever occurs first.

CR+CRi rate in Cohort CApproximately 6 months

CR+CRi rate, defined as the proportion of patients who achieved a CR or CRi

Relapse-free survival (RFS) in Cohort CApproximately 6 months

RFS, defined as the time from first documentation of CR to the date of death (any cause) or disease relapse or death due to any cause, whichever occurs first

Event-free survival (EFS) in Cohort CApproximately 6 months

EFS, defined as the time from first dose of study therapy to treatment failure, disease relapse after CR, or patient death due to any cause, whichever occurs first

Trial Locations

Locations (61)

National Cancer Center Hospital East

🇯🇵

Kashiwa-shi, Chiba-Ken, Japan

Mercy Clinic Oncology and Hematology - Coletta

🇺🇸

Oklahoma City, Oklahoma, United States

Ospedale Sacro Cuore Don Calabria

🇮🇹

Negrar, Verona, Italy

Centre Georges François Leclerc

🇫🇷

Dijon, Côte-d'Or, France

Georgetown University - Lombardi Comprehensive Cancer Center

🇺🇸

Washington, District of Columbia, United States

Istituto Scientifico Romagnolo Per Lo Studio e La Cura Dei Tumori

🇮🇹

Meldola, Forli - Cesena, Italy

Centre Léon Bérard

🇫🇷

Lyon, Rhone, France

Centre Hospitalier Lyon Sud

🇫🇷

Pierre Benite cedex, Rhone, France

Universitaetsklinikum Heidelberg

🇩🇪

Heidelberg, Baden Wuerttemberg, Germany

Azienda Ospedaliera Universitaria Careggi

🇮🇹

Florence, Italy

Aichi Cancer Center Hospital

🇯🇵

Nagoya-shi, Aichi-Ken, Japan

Centre Antoine Lacassagne

🇫🇷

Nice, Alpes Maritimes, France

Universitaetsklinikum Freiburg

🇩🇪

Freiburg, Baden Wuerttemberg, Germany

Universitaetsklinikum Koeln

🇩🇪

Koeln, Nordrhein Westfalen, Germany

The University of Hong Kong

🇭🇰

Hong Kong, Hong Kong

Seoul National University Hospital

🇰🇷

Seul, Korea, Republic of

Hôpital Saint-Louis

🇫🇷

Paris Cedex 10, Paris, France

Osaka University Hospital

🇯🇵

Suita-shi, Osaka-Fu, Japan

Institut Gustave Roussy

🇫🇷

Villejuif, Val De Marne, France

Banner MD Anderson Cancer Center

🇺🇸

Gilbert, Arizona, United States

National Cancer Center Hospital

🇯🇵

Chuo-ku, Tokyo-To, Japan

Samsung Medical Center

🇰🇷

Seul, Korea, Republic of

Fundação Champalimaud

🇵🇹

Lisboa, Portugal

Centro Hospitalar do Porto, E.P.E - Hospital de Santo Antonio

🇵🇹

Porto, Portugal

Beth Israel Deaconess Medical Center

🇺🇸

Boston, Massachusetts, United States

UCLA Medical Center

🇺🇸

Los Angeles, California, United States

University of Maryland

🇺🇸

Baltimore, Maryland, United States

University of Chicago Comprehensive Cancer Center

🇺🇸

Chicago, Illinois, United States

Henry Ford Hospital

🇺🇸

Woodhaven, Michigan, United States

Houston Methodist Cancer Center

🇺🇸

Houston, Texas, United States

Institut Jules Bordet

🇧🇪

Bruxelles, Belgium

The University of Texas M. D. Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Centre Paul Strauss

🇫🇷

Strasbourg, Bas Rhin, France

Institut Bergonié

🇫🇷

Bordeaux, Gironde, France

IEO Istituto Europeo di Oncologia

🇮🇹

Milano, Italy

Azienda Ospedaliera Universitaria Integrata Verona (Ospedale Borgo Trento)

🇮🇹

Verona, Italy

NHO Shikoku Cancer Center

🇯🇵

Matsuyama-shi, Ehime-Ken, Japan

Hokkaido University Hospital

🇯🇵

Sapporo-shi, Hokkaido, Japan

Seoul National University Bundang Hospital

🇰🇷

Seongnam, Gyeonggi-do, Korea, Republic of

Asan Medical Center

🇰🇷

Seoul, Korea, Republic of

Hospital Universitario Virgen Macarena

🇪🇸

Sevilla, Spain

Severance Hospital, Yonsei University Health System

🇰🇷

Seul, Korea, Republic of

Antoni van Leeuwenhoek

🇳🇱

Amsterdam, Netherlands

National University Cancer Institute

🇸🇬

Singapore, Singapore

Erasmus Medisch Centrum

🇳🇱

Rotterdam, Netherlands

Centro Hospitalar de Lisboa Central, E.P.E. - Hospital de Santo António dos Capuchos

🇵🇹

Lisboa, Portugal

Clinica Universidad de Navarra

🇪🇸

Pamplona, Spain

Hospital Universitari Vall d'Hebron

🇪🇸

Barcelona, Spain

Karolinska universitetssjukhuset - Solna

🇸🇪

Solna, Sweden

Instituto Valenciano de Oncologia IVO

🇪🇸

Valencia, Spain

Akademiska Sjukhuset

🇸🇪

Uppsala, Sweden

Hospital Clinico Universitario de Valencia

🇪🇸

Valencia, Spain

Hospital Universitari i Politecnic La Fe

🇪🇸

Valencia, Spain

Acibadem Adana Hospital

🇹🇷

Adana, Turkey

Acibadem Maslak Hospital

🇹🇷

Istanbul, Turkey

Namik Kemal University

🇹🇷

Tekirdağ, Turkey

Sarah Cannon Research Institute UK

🇬🇧

London, Greater London, United Kingdom

Fox Chase Cancer Center

🇺🇸

Philadelphia, Pennsylvania, United States

Hospital Universitario Ramon y Cajal

🇪🇸

Madrid, Spain

Hospital Universitario HM Madrid Sanchinarro

🇪🇸

Madrid, Spain

Hong Kong United Oncology Centre

🇭🇰

Jordon, Hong Kong

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