Study of Vimseltinib for Tenosynovial Giant Cell Tumor

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis, Giant Cell Tumor of Tendon Sheath, Tenosynovial Giant Cell Tumor, Diffuse, Tenosynovial Giant Cell Tumor, Localized

In brief

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.

Key facts

Study ID

NCT05059262

Run by

Deciphera Pharmaceuticals, LLC

People needed

123

Starts

2021-10-14

Expected to finish

2028-07-01

Last updated by the study team

2026-03-20

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients ≥18 years of age
• TGCT for which surgical resection is not an option (tumor biopsy to confirm diagnosis required if no histology/pathology available at screening)
• Symptomatic disease as defined as at least moderate pain or at least moderate stiffness (defined as a score of 4 or more, with 10 describing the worst condition) within the screening period and documented in the medical record
• Participants should complete 14 consecutive days of questionnaires during the screening period and must meet minimum requirements as outlined in study protocol
• Must have stable analgesic regimen, as judged by the investigator, for at least 2 weeks prior to first dose of study drug
• Must have measurable disease, as per RECIST Version 1.1, with at least one lesion having a minimum size of 2cm
• Adequate organ and bone marrow function
• If a female of childbearing potential, must have a negative pregnancy test prior to enrollment and agree to follow the contraception requirements
• Must provide signed consent to participate in the study and is willing to comply with study-specific procedures
• Willing and able to complete the patient-reported outcome (PRO) assessments on an electronic device

You may not qualify if…

• Previous use of systemic therapy (investigational or approved) targeting colony-stimulating factor 1 (CSF1) or colony-stimulating factor 1 receptor (CSF1R); previous therapy with imatinib and nilotinib is allowed
• Received therapy for TGCT, including investigational therapy during the screening period. Participated in a non-TGCT investigational drug study within 30 days of screening.
• Known metastatic TGCT or other active cancer that requires concurrent treatment (exceptions will be considered on a case-by-case basis)
• QT interval corrected by Fridericia's formula (QTcF) >450 ms in males or >470 ms in females or history of long QT syndrome
• Concurrent treatment with any study-prohibited medications
• Major surgery within 14 days of the first dose of study drug
• Any clinically significant comorbidities
• Active liver or biliary disease including nonalcoholic steatohepatitis (NASH) or cirrhosis
• Malabsorption syndrome or other illness that could affect oral absorption
• Known active human immunodeficiency virus (HIV), acute or chronic hepatitis B, acute or chronic hepatitis C, or known active mycobacterium tuberculosis infection
• If female, the participant is pregnant or breastfeeding
• Known allergy or hypersensitivity to any component of the study drug
• Contraindication to MRI

Where it is running

• City of Hope — Duarte, California, United States
• UC Davis Comprehensive Cancer Center — Sacramento, California, United States
• University of Colorado — Aurora, Colorado, United States
• University of Kansas — Kansas City, Kansas, United States
• Dana Farber Cancer Institute — Boston, Massachusetts, United States
• Mayo Clinic Rochester — Rochester, Minnesota, United States
• Memorial Sloan-Kettering Cancer Center — New York, New York, United States
• Duke Sarcoma Research — Durham, North Carolina, United States
• Ohio State University — Columbus, Ohio, United States
• University of Texas MD Anderson Cancer Center — Houston, Texas, United States
• Seattle Cancer Care Alliance — Seattle, Washington, United States
• Chris O'Brien Lifehouse — Camperdown, Australia
• McGill University — Montreal, Canada
• Princess Margaret Hospital — Toronto, Canada
• Institut Bergonié — Bordeaux, France
• Centre Léon Bérard — Lyon, France
• Institut Gustave Roussy — Villejuif, France
• Helios Klinikum Berlin-Buch — Berlin, Germany
• University Hospital Essen (Universitätsklinikum Essen) — Essen, Germany
• Prince of Wales Hospital — Hong Kong, Hong Kong
• Istituto Ortopedico Rizzoli — Bologna, Italy
• Fondazione IRCCS Istituto Nazionale dei Tumori — Milan, Italy
• Istituto Nazionale Tumori IRCCS "Fondazione G. Pascale" — Naples, Italy
• Istituto Oncologico Veneto — Padua, Italy
• Istituto Nazionale Tumori Regina Elena — Rome, Italy

Full record on ClinicalTrials.gov

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