Aficamten Compared To Placebo In Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy
Research type
Research Study
Full title
A Phase 3, Multi-Center, Randomized, Double-Blind Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults with Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy
IRAS ID
1008323
Contact name
Rachel Melman
Contact email
Sponsor organisation
Cytokinetics, Inc.
Clinicaltrials.gov Identifier
Research summary
The purpose of this study is to compare the effects of aficamten and placebo on health status and exercise capacity in participants with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM). nHCM is typically a genetic condition in which the main pumping chamber of the heart (left ventricle) becomes abnormally thickened and stiff, which can make it harder for the ventricle to fill with enough blood to pump out. An important feature of nHCM is hypercontractility, which means that the heart pumps too vigorously causing the muscle to work harder than is necessary. Aficamten is designed to reduce excessive heart pumping function.
The safety and tolerability of aficamten at different, increasing dose levels will also be studied. The study will also measure the amount of aficamten in the blood at various times (PK – pharmacokinetics), and the effect the research medicine may have on nHCM.
About 420 people between 18 and 85 years of age are expected to participate in this study at approximately 150 sites worldwide.
Participation in this research will involve up to 13 visits to the study site. The research study will last between 46 weeks (10.5 months) to at most 82 weeks (19 months). This time estimate includes the screening period (up to 6 weeks), the treatment period (36 to 72 weeks), and the follow-up period (of 4 weeks after the last treatment).
Participants will receive either aficamten or placebo during the study treatment period (but not both) of 36 to 72 weeks (8.5 to 17 months).
There are 2 parts to the study.
• Part 1: All participants will receive the study medicine and be followed for 36 weeks (8.5 months).
• Part 2: Participants who complete Week 36 will continue with the same treatment until Week 72 or until the last participant in Part 1 completes Week 36, then all participants will have an End of Study visit 4 weeks after their last dose.REC name
South Central - Hampshire A Research Ethics Committee
REC reference
24/SC/0021
Date of REC Opinion
16 Feb 2024
REC opinion
Further Information Favourable Opinion