CEDAR-HCM
Research type
Research Study
Full title
A Phase 2/3 Multicenter, Randomized, Double-Blind, Placebo-Controlled And Open-Label Extension Trial To Evaluate The Efficacy And Safety Of Aficamten In A Pediatric Population With Symptomatic Obstructive Hypertrophic Cardiomyopathy
IRAS ID
1010004
Contact name
Lynsey Kennedy
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 the level of obstruction that limits blood flow from the heart in young participants with Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM). The safety and tolerability of aficamten at different, increasing dose levels will also be studied. The effectiveness of aficamten and placebo will be compared by measuring the level of obstruction that limits blood flow from the heart with an echocardiogram. The study will also measure the amount of aficamten in the blood at various times (PK – pharmacokinetics), and the effect the study drug may have on oHCM.
About 40 participants between 12 and less than 18 years of age will take part in this study at about 20 to 30 sites worldwide. This study may be opened to about 8-10 younger participants (age 6 to less than age 12) at a later date, depending upon the results from this and other research studies.
The study is 2 periods; 12 weeks of randomised, double-blinded treatment where some participants will take a placebo and then 52 weeks (1 year) of open label treatment where all participants will take the study drug.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
24/EM/0201
Date of REC Opinion
5 Nov 2024
REC opinion
Further Information Favourable Opinion