What is aficamten studying for pediatric obstructive hypertrophic cardiomyopathy?

Aficamten is being studied to assess its effects on heart function, safety, and how it is processed in children and adolescents with symptomatic obstructive hypertrophic cardiomyopathy. This research aims to better understand potential treatment options for this condition in younger patients and is sponsored by Cytokinetics.

Who may be eligible for this pediatric obstructive hypertrophic cardiomyopathy clinical trial?

Children and adolescents aged 12 to under 18 years with symptomatic obstructive hypertrophic cardiomyopathy and specific heart function criteria may be eligible. Participants must have stable heart medication doses when applicable and no significant other heart conditions. Please review eligibility criteria for more details.

Is there any cost to participate in this pediatric obstructive hypertrophic cardiomyopathy clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. Personal travel expenses may vary and are not guaranteed to be covered. Participants can expect to attend scheduled visits and assessments during the study.

Actively Recruiting

Phase 2

Phase 3

Age: 12Years - 17Years

All Genders

ID06412666

Study of Aficamten to Treat Symptomatic Obstructive Hypertrophic Cardiomyopathy in Children and Adolescents Aged 12 to Under 18 Years

Led by Cytokinetics · Updated on 2026-04-13

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are studying aficamten in children and adolescents aged 6 to under 18 years with symptomatic obstructive hypertrophic cardiomyopathy oHCM, a condition causing thickened heart muscle and outflow obstruction. The study aims to evaluate the drugs effectiveness, safety, and how the body processes it in this pediatric group. Adolescents and children will be studied sequentially, starting with adolescents to establish safe and effective dosing before proceeding to younger children. The trial is a Phase 23 randomized, double-blind, placebo-controlled study with additional open-label extension periods for long-term assessment. Participants will be assigned randomly to receive either oral aficamten or a placebo once daily. The initial treatment period lasts 12 weeks and is double-blinded. After this, all participants receive aficamten for 52 weeks during an open-label extension, followed by a long-term extension of 144 weeks to further assess safety, tolerability, and efficacy. Doses range from 5 mg to 20 mg and are adjusted based on echocardiography findings. This staged approach ensures careful monitoring and dosing adjustments throughout the study. During the study, participants will undergo regular evaluations including heart ultrasounds to measure heart function and obstruction, blood tests for heart biomarkers, and assessments of heart failure symptoms using the New York Heart Association functional class. Researchers will monitor how aficamten affects the hearts outflow tract gradient over 12 weeks and track long-term safety over several years. The study includes multiple visits for clinical assessments, laboratory tests, and drug level measurements to understand treatment effects and ensure participant safety.

CONDITIONS

Brief Title

A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).

Who Can Participate

Age: 12Years - 17Years

All Genders

Eligibility Criteria

You may qualify if you...

Males and females aged 12 to under 18 years at screening and Day 1
Body weight at least 45 kg for initial cohort, then at least 35 kg after safety review
Confirmed obstructive hypertrophic cardiomyopathy by core lab echocardiography with specific wall thickness criteria
Left ventricular ejection fraction (LVEF) of 60% or higher and Valsalva LVOT gradient of 50 mmHg or higher
oHCM of sarcomeric origin confirmed by genetic testing or presumed without other metabolic or neuromuscular diseases
New York Heart Association (NYHA) Class II or higher at screening
Adequate echocardiographic acoustic windows
Stable doses of beta blockers, verapamil, diltiazem, or disopyramide for more than 4 weeks prior to randomization
For extension periods, completion of prior periods with LVEF 55% or higher after washout

You will not qualify if you...

Significant valvular heart disease such as moderate/severe aortic stenosis or fixed subaortic obstruction
Mitral regurgitation more than mild and not due to systolic anterior motion
Fixed left-sided obstruction like subaortic membrane, aortic valve stenosis, or aortic coarctation
History of left ventricular systolic dysfunction with LVEF below 45% or stress cardiomyopathy
History of congenital heart disease other than oHCM unless not hemodynamically significant
Surgical myectomy or alcohol septal ablation within 6 months or planned during trial
History of atrial fibrillation or atrial flutter
Syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia within 3 months prior to screening
Other significant disorders, malignancy, active infection, or conditions posing safety risk
Use of drugs causing cardiomyopathy like anthracyclines, trastuzumab, cyclophosphamide, sunitinib, or imatinib
Participation in other investigational drug/device trials within 1 month prior to screening
Implantable cardioverter defibrillator implantation within 6 weeks or planned during trial
Prior treatment with aficamten or mavacamten
Listed or expected to be listed for heart transplantation within 12 months

Research Team

Cytokinetics MD

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