Mavacamten in nonobstructive HCM
Research type
Research Study
Full title
A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adults with Symptomatic Nonobstructive Hypertrophic Cardiomyopathy
IRAS ID
1006306
Contact name
GSM-CT Representative
Contact email
Sponsor organisation
Myokardia, Inc.
Eudract number
2021-005329-26
Research summary
The purpose of this Phase 3, double-blind, randomised, parallel-group, placebo-controlled, multicentre, international study is to find out if treatment with mavacamten for 52 weeks reduces shortness of breath and fatigue and increases exercise capacity in patients with non-obstructive hypertrophic cardiomyopathy (nHCM). Mavacamten (BMS-986427) has been approved by the United States Food and Drug Administration (US FDA) to improve functional capacity and symptoms in adults with obstructive hypertrophic cardiomyopathy (oHCM). However, mavacamten is not approved for people with nHCM, so its use in this study is considered to be investigational.
About 420 participants at about 180 sites across 21 countries in the Americas, Europe, the Middle East, and Asia are expected to participate in this study. The participation will last for a period between 52 weeks and up to 134 weeks (about 2 1/2 years). The participants have an equal chance of getting mavacamten capsules or matching placebo capsules.
• The placebo is a dummy capsule that looks like the real one but does not contain mavacamten.
• Neither the participant nor the study doctor will know which treatment the participant are receiving, except in an emergency.
The pharmaceutical company sponsoring this study is MyoKardia Inc., a wholly-owned subsidiary of Bristol Myers Squibb company.REC name
London - Fulham Research Ethics Committee
REC reference
22/LO/0906
Date of REC Opinion
27 Sep 2023
REC opinion
Further Information Favourable Opinion