Study of Vimseltinib for Tenosynovial Giant Cell Tumor

Last updated: February 20, 2025
Sponsor: Deciphera Pharmaceuticals, LLC
Overall Status: Active - Not Recruiting

Phase

3

Condition

Neoplasms

Soft Tissue Infections

Arthritis And Arthritic Pain

Treatment

Vimseltinib

Placebo

vimseltinib

Clinical Study ID

NCT05059262
DCC-3014-03-001
2024-513624-42-00
  • Ages > 18
  • All Genders

Study Summary

This is a multicenter Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug called vimseltinib for the treatment of tenosynovial giant cell tumor (TGCT) in cases where surgical removal of the tumor is not an option.

The study consists of two parts. In Part 1, eligible study participants will be assigned to receive either vimseltinib or matching placebo for 24 weeks. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. MRI scans will be used to evaluate the response of the tumors to the treatment. Participants assigned to placebo in Part 1 will have the option to receive vimseltinib for Part 2. Part 2 is a long-term treatment phase in which all participants receive open-label vimseltinib.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Patients ≥18 years of age

  2. TGCT for which surgical resection is not an option (tumor biopsy to confirmdiagnosis required if no histology/pathology available at screening)

  3. Symptomatic disease as defined as at least moderate pain or at least moderatestiffness (defined as a score of 4 or more, with 10 describing the worst condition)within the screening period and documented in the medical record

  4. Participants should complete 14 consecutive days of questionnaires during thescreening period and must meet minimum requirements as outlined in study protocol

  5. Must have stable analgesic regimen, as judged by the investigator, for at least 2weeks prior to first dose of study drug

  6. Must have measurable disease, as per RECIST Version 1.1, with at least one lesionhaving a minimum size of 2cm

  7. Adequate organ and bone marrow function

  8. If a female of childbearing potential, must have a negative pregnancy test prior toenrollment and agree to follow the contraception requirements

  9. Must provide signed consent to participate in the study and is willing to complywith study-specific procedures

  10. Willing and able to complete the patient-reported outcome (PRO) assessments on anelectronic device

Exclusion

Exclusion Criteria:

  1. Previous use of systemic therapy (investigational or approved) targetingcolony-stimulating factor 1 (CSF1) or colony-stimulating factor 1 receptor (CSF1R);previous therapy with imatinib and nilotinib is allowed

  2. Received therapy for TGCT, including investigational therapy during the screeningperiod. Participated in a non-TGCT investigational drug study within 30 days ofscreening.

  3. Known metastatic TGCT or other active cancer that requires concurrent treatment (exceptions will be considered on a case-by-case basis)

  4. QT interval corrected by Fridericia's formula (QTcF) >450 ms in males or >470 ms infemales or history of long QT syndrome

  5. Concurrent treatment with any study-prohibited medications

  6. Major surgery within 14 days of the first dose of study drug

  7. Any clinically significant comorbidities

  8. Active liver or biliary disease including nonalcoholic steatohepatitis (NASH) orcirrhosis

  9. Malabsorption syndrome or other illness that could affect oral absorption

  10. Known active human immunodeficiency virus (HIV), acute or chronic hepatitis B, acuteor chronic hepatitis C, or known active mycobacterium tuberculosis infection

  11. If female, the participant is pregnant or breastfeeding

  12. Known allergy or hypersensitivity to any component of the study drug

  13. Contraindication to MRI

Study Design

Total Participants: 123
Treatment Group(s): 3
Primary Treatment: Vimseltinib
Phase: 3
Study Start date:
October 14, 2021
Estimated Completion Date:
July 31, 2026

Connect with a study center

  • Chris O'Brien Lifehouse

    Camperdown,
    Australia

    Site Not Available

  • Princess Margaret Hospital

    Montreal,
    Canada

    Site Not Available

  • McGill University

    Montréal,
    Canada

    Site Not Available

  • Princess Margaret Hospital

    Toronto,
    Canada

    Site Not Available

  • Institut Bergonié

    Bordeaux,
    France

    Site Not Available

  • Centre Léon Bérard

    Lyon,
    France

    Site Not Available

  • Institut Gustave Roussy

    Villejuif,
    France

    Site Not Available

  • Helios Klinikum Berlin-Buch

    Berlin,
    Germany

    Site Not Available

  • University Hospital Essen (Universitätsklinikum Essen)

    Essen,
    Germany

    Site Not Available

  • Prince of Wales Hospital

    Hong Kong,
    Hong Kong

    Site Not Available

  • Istituto Ortopedico Rizzoli

    Bologna,
    Italy

    Site Not Available

  • Fondazione IRCCS Istituto Nazionale dei Tumori

    Milan,
    Italy

    Site Not Available

  • Istituto Nazionale Tumori IRCCS "Fondazione G. Pascale"

    Naples,
    Italy

    Site Not Available

  • Istituto Oncologico Veneto

    Padua,
    Italy

    Site Not Available

  • Istituto Nazionale Tumori Regina Elena

    Rome,
    Italy

    Site Not Available

  • Seoul National University Hospital

    Seoul,
    Korea, Republic of

    Site Not Available

  • Leiden University Medical Center

    Leiden,
    Netherlands

    Site Not Available

  • Oslo University Hospital

    Oslo,
    Norway

    Site Not Available

  • Klinika Nowotworów Tkanek Miękkich, Kości i Czerniaków Narodowy Instytut Onkologii im. Marii Skłodowskiej-Curie - Państwowy Instytut Badawczy

    Warsaw,
    Poland

    Site Not Available

  • Hospital Universitario Vall d'Hebron

    Barcelona,
    Spain

    Site Not Available

  • Hospital de la Santa Creu i Sant Pau

    Barcelona,
    Spain

    Site Not Available

  • Fundacion Jimenez Diaz

    Madrid,
    Spain

    Site Not Available

  • Hospital Clinico San Carlos

    Madrid,
    Spain

    Site Not Available

  • Universitäts-Kinderspital beider Basel (UKBB)

    Basel,
    Switzerland

    Site Not Available

  • Cancer & Haematology Centre, The Churchill Hospital - Oxford University Hospitals NHS Foundation Trust

    London,
    United Kingdom

    Site Not Available

  • University College London Hospitals

    London,
    United Kingdom

    Site Not Available

  • City of Hope

    Duarte, California 91010
    United States

    Site Not Available

  • UC Davis Comprehensive Cancer Center

    Sacramento, California 95817
    United States

    Site Not Available

  • University of Colorado

    Aurora, Colorado 80045
    United States

    Site Not Available

  • University of Kansas

    Kansas City, Kansas 66160
    United States

    Site Not Available

  • Dana Farber Cancer Institute

    Boston, Massachusetts 02215
    United States

    Site Not Available

  • Mayo Clinic Rochester

    Rochester, Minnesota 55902
    United States

    Site Not Available

  • Memorial Sloan-Kettering Cancer Center

    New York, New York 10016
    United States

    Site Not Available

  • Duke Sarcoma Research

    Durham, North Carolina 27710
    United States

    Site Not Available

  • Ohio State University

    Columbus, Ohio 43210
    United States

    Site Not Available

  • University of Texas MD Anderson Cancer Center

    Houston, Texas 77030
    United States

    Site Not Available

  • Seattle Cancer Care Alliance

    Seattle, Washington 98109
    United States

    Site Not Available

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