New Drug: Vimseltinib for Tenosynovial Giant Cell Tumor
Study
Multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
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Patients: Adults (≥18 years) with symptomatic TGCT not amenable to surgery
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Treatment: Vimseltinib 30 mg orally twice weekly (n=83) vs. placebo (n=40) for 24 weeks
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Efficacy
ORR: 40% (33/83) vs. 0% (0/40) (vimseltinib vs. placebo), p<0.0001
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mDOR: Not reached in vimseltinib group
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Improvements in functional outcomes: Active range of motion, PROMIS-PF, stiffness, EQ-VAS, BPI worst pain, all significantly improved with vimseltinib
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Safety
Grade ≥3 AEs: Increased blood creatine phosphokinase (10%), hypertension (5%), AST increase (5%)
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Serious TEAEs: 1 case of subcutaneous abscess
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No hepatotoxicity or drug-induced liver injury observed
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Gelderblom H,Bhadri V,Stacchiotti S Vimseltinib versus placebo for tenosynovial giant cell tumour (MOTION): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Reviewed by Ulas D. Bayraktar, MD on Mar 14, 2025
