NCT06412666 · Cytokinetics
A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
(CEDAR-HCM)
What this study is about
The purpose of this study is to evaluate the effectiveness, safety and PK of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
View original scientific description
The purpose of this study is to evaluate the efficacy, safety and PK of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
Interventions
DRUG
Aficamten
Oral Tablet
DRUG
Placebo
Oral Tablet
Primary outcome measures
Change from baseline in Valsalva left ventricular outflow tract gradient (LVOT-G)
Time frame: Baseline to week 12
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Period 1: Treatment Period
- Males and females between 12 and \< 18 years of age at screening and at Day 1.
- Body weight ≥ 45 kg for the initial cohort and then body weight ≥ 35 kg after at least 10 participants in the initial cohort have undergone dose titration up to Week 4 without observed events of LVEF \< 50% at the starting dose of 5 mg qd.
- Core laboratory confirmation of the following oHCM echocardiographic criteria at screening:
- Left ventricular (LV) hypertrophy with nondilated LV chamber in the absence of other cardiac disease.
- LV end-diastolic wall thickness that meets a threshold of:
- Z-score \> 2.5 in the absence of family history OR
- Z-score \> 2 in the presence of positive family history or positive genetic test.
- LVEF ≥ 60% AND Valsalva LVOT-G ≥ 50 mmHg.
- oHCM of sarcomeric origin confirmed by genetic testing or, if unable to confirm by genetic testing, oHCM of sarcomeric origin may be presumed in the absence of history of
Where
- Phoenix, Arizona
- Los Angeles, California
- San Francisco, California
- Aurora, Colorado
- Washington D.C., District of Columbia
- Miami, Florida
- Chicago, Illinois
- Iowa City, Iowa
- New Orleans, Louisiana
- Ann Arbor, Michigan
- Detroit, Michigan
- Rochester, Minnesota
And 13 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 13, 2026 · Source of record for eligibility and locations