Musculoskeletal Diseases

All Conditions

Neoplasms

Skin and Connective Tissue Diseases

Wounds and Injuries

Nervous System Diseases

Rare Diseases

Synovitis

Giant Cell Tumors

Giant Cell Tumor of Tendon Sheath

Synovitis, Pigmented Villonodular

Joint Diseases

Neoplasms, Connective Tissue

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Tendinopathy

Muscular Diseases

Pigmented Villonodular Synovitis

Participants receive blinded treatment of 30 mg twice a week (biw) vimseltinib for 24 weeks in Part 1 and continue on 30 mg biw vimseltinib in Part 2

Participants receive blinded treatment of 30 mg twice a week (biw) matching placebo for 24 weeks in Part 1 and have option to receive 30 mg biw vimseltinib in Part 2

vimseltinib

Placebo

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.

Study of Vimseltinib for Tenosynovial Giant Cell Tumor

A Phase 3, Randomized, Placebo-controlled, Double-blind Study of Vimseltinib to Assess the Efficacy and Safety in Patients With Tenosynovial Giant Cell Tumor (MOTION)

Assessed by central read using Response Evaluation Criteria In Solid Tumors (RECIST) Version 1.1

Assessed by central read using Tumor Volume Score (TVS). TVS is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor.

Mean change from baseline in active ROM of the affected joint, relative to a reference standard at Week 25

Mean change from baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) physical function score at Week 25

Mean change from baseline in the Worst Stiffness Numeric Rating Scale (NRS) score at Week 25

Mean change from baseline in EuroQoL Visual Analogue Scale (EQ-VAS) at Week 25

Proportion of responders, with a response defined as at least a 30% improvement in the mean Brief Pain Inventory (BPI) Worst Pain NRS score without a 30% or greater increase in narcotic analgesic use at Week 25

Spots Global Cancer Trial Database for tenosynovial giant cell tumor, diffuse