Study
| Multicentre, randomised, double-blind, placebo-controlled, phase 3 trial |
| Patients: Adults (≥18 years) with symptomatic TGCT not amenable to surgery |
| Treatment: Vimseltinib 30 mg orally twice weekly (n=83) vs. placebo (n=40) for 24 weeks |
Efficacy
| ORR: 40% (33/83) vs. 0% (0/40) (vimseltinib vs. placebo), p<0.0001 |
| mDOR: Not reached in vimseltinib group |
| Improvements in functional outcomes: Active range of motion, PROMIS-PF, stiffness, EQ-VAS, BPI worst pain, all significantly improved with vimseltinib |
Safety
| Grade ≥3 AEs: Increased blood creatine phosphokinase (10%), hypertension (5%), AST increase (5%) |
| Serious TEAEs: 1 case of subcutaneous abscess |
| No hepatotoxicity or drug-induced liver injury observed |
Lancet 2024;403:2709-19
http://doi.org/10.1016/S0140-6736(24)00885-7
Reviewed by Ulas D. Bayraktar, MD on Mar 14, 2025
