Clinical Trials in Canada / NCT06412666
Active, not recruiting Phase 2/3

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

NCT06412666 · tracked via the Priya Life Science Canada tracker
Phase
Phase 2/3
Started
2024-05-29
Last updated
2026-08-18

Condition(s) studied

PediatricSymptomatic Obstructive Hypertrophic Cardiomyopathy

Investigational drug(s) / intervention(s)

Aficamten →Placebo

Aficamten: Oral Tablet

Placebo: Oral Tablet

Study summary

The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

Eligibility

Sex
ALL
Min age
12 Years
Max age
17 Years
Healthy volunteers
No
Inclusion Criteria: * Period 1: Treatment Period * Males and females between 12 and \< 18 years of age at screening and at Day 1. * Body weight ≥ 35 kg * Diagnosed with oHCM based on the following at screening: * Left ventricular (LV) hypertrophy with nondilated LV chamber in the absence of other cardiac disease. * Core laboratory confirmation of 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. * Core laboratory confirmation of 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 metabolic disorders, mitochondrial cardiomyopathies, neuromuscular disease, malformation syndromes, infiltrative diseases/inflammation, and endocrine disorders (such as Fabry's disease, Noonan syndrome with left ventricular hypertrophy, and amyloid-cardiomyopathy). * New York Heart Association (NYHA) Class ≥ II at screening. * Adequate acoustic windows for echocardiography. * Participants on beta blockers, verapamil, diltiazem, or disopyramide should have been on stable doses for more than 4 weeks prior to randomization. * Period 2: Open-Label Extension * Completed Period 1. If unable to complete Period 1 due to circumstances not related to compliance or safety, the Medical Monitor may review and determine eligibility. * LVEF ≥ 55% after washout * Period 3: Long-term Extension * Completed Period 2 Exclusion Criteria: * Period 1: Treatment Period Any of the following criteria will exclude potential participants from the trial: * Significant valvular heart disease. * Moderate or severe valvular aortic stenosis or fixed subaortic obstruction. * Mitral regurgitation that is greater than mild in severity and not due to systolic anterior motion of the mitral valve (per judgment of Principal Investigator or designee). * Evidence of fixed left-sided obstruction (eg, subaortic membrane, aortic valve stenosis, or coarctation of the aorta). * History of LV systolic dysfunction (LVEF \< 45%) or stress cardiomyopathy at any time during their clinical course. * History of congenital heart disease other than oHCM (may be enrolled if not hemodynamically significant in the judgement of the Principal Investigator and study Medical Monitor). * Has been treated with SRT (surgical myectomy or percutaneous alcohol septal ablation) within the preceding 6 months or has plans for either treatment during the trial period. * History of paroxysmal or persistent atrial fibrillation or atrial flutter. * History of syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia within 3 months prior to screening. * History or evidence of any other clinically significant disorder, malignancy, active infection, other condition, or disease that, in the opinion of the Principal Investigator (or designee) or the Medical Monitor, would pose a risk to participant safety or interfere with the trial evaluation, procedures, or completion. * Current or previous use of drugs known to cause cardiomyopathy (eg, anthracyclines, monoclonal antibodies \[trastuzumab\], alkylating agents \[cyclophosphamide\], and tyrosine kinase inhibitors \[sunitinib and imatinib\]). * Currently participating in another investigational device or drug trial or received an investigational device or drug \< 1 month (or 5 half-lives for drugs, whichever is longer) prior to screening. * Implantable cardioverter defibrillator (ICD) implantation within 6 weeks of screening or planned ICD implantation during the trial period. * Has received prior treatment with aficamten or mavacamten. * Currently listed for heart transplantation or anticipated to be listed for heart transplantation in the next 12 months.

Primary outcome measure(s)

Trial sites (36)

FacilityCityRegionStatus
Phoenix Children's Hospital Phoenix Arizona
Arkansas Children's Hospital Little Rock Arkansas
Children's Hospital Los Angeles Los Angeles California
University of California, Los Angeles (UCLA) Los Angeles California
Children's Hospital Colorado Aurora Colorado
Children's National Hospital Washington D.C. District of Columbia
Nicklaus Children's Hospital Miami Florida
Ann & Robert H. Lurie Children's Hospital Chicago Illinois
University of Michigan Ann Arbor Michigan
Children's Hospital of Michigan Detroit Michigan
Mayo Clinic Rochester Minnesota
Children's Mercy Hospital Kansas City Missouri
University of Nebraska Medical Center Omaha Nebraska
Morristown Medical Center Morristown New Jersey
NYP/Columbia University Medical Center New York New York
Children's Hospital at Montefiore The Bronx New York
Duke Clinical Research Institute Durham North Carolina
Oregon Health & Science University Portland Oregon
Children's Hospital of Philadelphia Philadelphia Pennsylvania
LeBonheur Children's Hospital Memphis Tennessee
Vanderbilt University Medical Center Nashville Tennessee
Dell Children's Hospital Austin Texas
UT Southwestern Dallas Texas
Children's Wisconsin Milwaukee Wisconsin
The Hospital for Sick Children (SickKids) Toronto Ontario
Azienda Ospedaliera Universitaria Meyer IRCCS Florence Italy
NHO Kagoshima Medical Center Kagoshima Japan
University of Osaka Hospital Osaka Japan
Kitasato University Hospital Sagamihara Japan
National Cerebral and Cardiovascular Center Suita Japan
Juntendo University Hospital Tokyo Japan
Unidad de Cardiología Infantil; Hospital Universitario da Coruña A Coruña Spain
Hospital Sant Joan de Deu Barcelona Spain
Alder Hey Children's Hospital Liverpool United Kingdom
Evelina Children's Hospital London United Kingdom
Great Ormond Street Hospital for Children London United Kingdom

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06412666 on ClinicalTrials.gov ↗ ← All trials in Canada