drugset / Trial / NCT06253221
A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy
RandomizedParallel-groupQuadruple-blindTreatment
Summary
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
Timeline
- Start
- 2024-04-17
- Primary completion
- 2025-11-25
- Completion
- 2031-03-28
Outcome
Met primary endpoint
paper gradient was -48.5 mm Hg in the mavacamten group and -0.5 mm Hg in the placebo group PMID 41910394 ↗
paper difference, -48.0 mm Hg; 95% confidence interval, -67.7 to -28.3; P<0.001 PMID 41910394 ↗
release “Application based on positive results of Phase 3 SCOUT-HCM trial, which met its primary endpoint” bms.com ↗
release “The SCOUT-HCM trial met its primary endpoint” bms.com ↗
Publications
- Rossano JW, Canter C, Wolf CM, Papez A, Gambra M, Bryant RM, Alejos J, McCulloch M, Sarquella Brugada G, Bock MJ, Jeewa A, Pearce FB, Desai MY, Favatella N, Javidialsaadi A, Phung V, Rano T, Zhu L, Dyme JL, Mital S; SCOUT-HCM Investigators. Mavacamten in Adolescents with Obstructive Hypertrophic Cardiomyopathy. N Engl J Med. 2026 Mar 29. doi: 10.1056/NEJMoa2601103. Online ahead of print.
- Rossano J, Canter C, Wolf C, Favatella N, Lockman J, Puli S, Javidialsaadi A, Dyme J, Crevar C, Mital S. Mavacamten in symptomatic adolescent patients with obstructive hypertrophic cardiomyopathy: design of the phase 3 SCOUT-HCM trial. Am Heart J. 2026 Feb;292:107283. doi: 10.1016/j.ahj.2025.107283. Epub 2025 Sep 30.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Mavacamten | Small molecule | 1 mg | — |
| Subject | Mavacamten | Small molecule | 15 mg | — |
Indications
No indication recorded.