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 (Cytokinetics)

Research summary

Approximately 420 eligible participants will be randomized in a 1:1 ratio to aficamten or placebo. Doses of 5, 10, 15, or 20 mg aficamten or matching placebo will be administered in an escalating manner using echocardiography to guide dose titration. Randomization will be stratified by persistent atrial fibrillation and presence of intracavitary obstruction. The study will consist of 2 parts: Part 1 will be comprised of Day 1 to Week 36 and Part 2 will be comprised of Week 36 to Week 72. All participants will complete Part 1. At the end of Part 1, participants will continue into Part 2 until the last randomized participant has completed Part 1 (Week 36). At that point, all remaining active study participants in Part 2 will have an end of treatment visit followed by an end of study visit as defined in the schedule of activities. Enrollment of participants with intracavitary obstruction will be limited to approximately < 10% of total enrollment. Enrollment will be limited so that approximately < 10% of participants have persistent or permanent atrial fibrillation, and approximately < 10% of participants have a body mass index in the range of > 35 to < 40 kg/m 2. Participants completing the study will have an option to roll over into a long-term open label extension study. A cardiovascular magnetic resonance imaging substudy will be conducted in up to 100 participants who provide informed consent. A pharmacokinetic (PK) substudy will be conducted in up to 30 participants who provide informed consent.

Principal Investigator

Dr Betty Raman

Contact us

Email: cvm_nurses@cardiov.ox.ac.uk

IRAS number

1008323