EXPLORER-HCM
Research type
Research Study
Full title
A Randomized, Double blind, Placebo controlled Clinical Study to Evaluate Mavacamten (MYK-461) in Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy
IRAS ID
233373
Contact name
Perry Elliott
Contact email
Sponsor organisation
MyoKardia, Inc.
Eudract number
2017-002530-23
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
1 years, 9 months, days
Research summary
Hypertrophic cardiomyopathy (HCM) is a condition that causes abnormal thickening of the heart muscle. It is often a genetic condition and as a consequence can be passed along through families. In some cases, thickening of the wall between the two lower chambers of the heart, called the septum, blocks the flow of blood through the outlet to the left ventricle when the heart contracts. This is known as left ventricular outflow tract (LVOT) obstruction and can cause chest pain with exertion, breathlessness, and blackouts.
The genetic mutations that cause HCM affect the proteins that make up the molecular motor (the sarcomere) that allows heart muscle cells to contract. Many of these mutations appear to increase the force with which the heart beats and can also make the heart stiffer than normal.
Mavacamten (MYK-461) is a novel drug being studied that specifically affects one of the sarcomere proteins, cardiac myosin, reducing its ability to cause contraction of the heart muscle, and this affect is reversed when the study drug is stopped. This action of the drug is predicted to reduce LVOT obstruction and to potentially reduce heart thickening with chronic treatment.
EXPLORER is a double-blind, randomised study sponsored by MyoKardia, Inc. that will assess the effects and safety of mavacamten compared with placebo (1:1) in patients with symptomatic obstructive HCM. Approximately 220 patients will be enrolled for a total duration of approximately 42 weeks. The primary end-point is the effect of Mavacamten on symptoms and exercise capacity.
REC name
London - London Bridge Research Ethics Committee
REC reference
18/LO/1348
Date of REC Opinion
22 Oct 2018
REC opinion
Further Information Favourable Opinion