Mavacamten Impact on Myocardial Structure in Obstructive Hypertrophic Cardiomyopathy
Research type
Research Study
Full title
A Phase 3b/4, Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy to Assess the Impact on Myocardial Structure with Cardiac Magnetic Resonance Imaging (CMR)
IRAS ID
1008133
Contact name
GSM-CT Representative
Contact email
Sponsor organisation
MyoKardia, Inc., a wholly owned subsidiary of Bristol-Myers Squibb Company
Eudract number
2022-502316-36
Research summary
Hypertrophic cardiomyopathy (HCM) is a disease in which the heart muscle becomes thickened (hypertrophied). The thickened heart muscle can make it harder for the heart to pump blood.
Hypertrophic cardiomyopathy often goes undiagnosed because many people with the disease have few, if any, symptoms. However, in a small number of people with HCM, the thickened heart muscle can cause shortness of breath, chest pain or changes in the heart's electrical system, resulting in life-threatening irregular heart rhythms (arrhythmias) or sudden death.
When HCM causes the heart muscle to get so thick that it blocks or reduces blood flow from the heart to the rest of the body, it’s called obstructive HCM (oHCM).
Mavacamten is a novel, well tolerated and effective therapy that aims to reduce myocardial hypercontractility, thought to be one of the key pathophysiological mechanisms in oHCM. Mavacamten has the potential to slow or halt disease progression and improve patient’s functional status and quality of life.9 Therefore, this appraisal should be considered a priority.
The safety, efficacy, and tolerability results of Mavacamten have been carefully investigated in nonclinical and clinical studies. Overall, Mavacamten appears to improve functional capacity and symptoms in patients with oHCM and is generally well tolerated with little evidence for off-target toxicity. The benefit-risk profile for Mavacamten remains favourable.
This is a Phase 3b/4 study where participants will be either receiving mavacamten or placebo by chance (randomised study) and the study doctor, study team and the participants will be unaware of the kind of treatment received (double-blind study) for the first 48 weeks.
Starting at week 48 through week 96, all participants will receive mavacamten (active treatment).REC name
Wales REC 1
REC reference
23/WA/0261
Date of REC Opinion
3 Nov 2023
REC opinion
Further Information Favourable Opinion