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

Last updated: March 31, 2026
Sponsor: Cytokinetics
Overall Status: Active - Recruiting

Phase

2/3

Condition

Circulation Disorders

Treatment

Aficamten

Placebo

Clinical Study ID

NCT06412666
CY 6023
2024-511377-30-00
  • Ages 12-17
  • All Genders

Study Summary

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).

Eligibility Criteria

Inclusion

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 afterat least 10 participants in the initial cohort have undergone dose titration upto Week 4 without observed events of LVEF < 50% at the starting dose of 5 mgqd.

  • Core laboratory confirmation of the following oHCM echocardiographic criteriaat screening:

  • Left ventricular (LV) hypertrophy with nondilated LV chamber in the absence of othercardiac 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 genetictest.

  • LVEF ≥ 60% AND Valsalva LVOT-G ≥ 50 mmHg.

  • oHCM of sarcomeric origin confirmed by genetic testing or, if unable to confirmby genetic testing, oHCM of sarcomeric origin may be presumed in the absence ofhistory of metabolic disorders, mitochondrial cardiomyopathies, neuromusculardisease, malformation syndromes, infiltrative diseases/inflammation, andendocrine disorders (such as Fabry's disease, Noonan syndrome with leftventricular 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 shouldhave 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 notrelated to compliance or safety, the Medical Monitor may review and determineeligibility.

  • LVEF ≥ 55% after washout.

  • Period 3: Long-term Extension • Completed Period 2.

Exclusion

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 tosystolic anterior motion of the mitral valve (per judgment of PrincipalInvestigator or designee).

  • Evidence of fixed left-sided obstruction (eg, subaortic membrane, aortic valvestenosis, or coarctation of the aorta).

  • History of LV systolic dysfunction (LVEF < 45%) or stress cardiomyopathy at any timeduring their clinical course.

  • History of congenital heart disease other than oHCM (may be enrolled if nothemodynamically significant in the judgement of the Principal Investigator and studyMedical Monitor).

  • Has been treated with SRT (surgical myectomy or percutaneous alcohol septalablation) within the preceding 6 months or has plans for either treatment during thetrial period.

  • History of paroxysmal or persistent atrial fibrillation or atrial flutter.

  • History of syncope, symptomatic ventricular arrhythmia, or sustained ventriculartachyarrhythmia within 3 months prior to screening.

  • History or evidence of any other clinically significant disorder, malignancy, activeinfection, other condition, or disease that, in the opinion of the PrincipalInvestigator (or designee) or the Medical Monitor, would pose a risk to participantsafety 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], andtyrosine kinase inhibitors [sunitinib and imatinib]).

  • Currently participating in another investigational device or drug trial or receivedan investigational device or drug < 1 month (or 5 half-lives for drugs, whichever islonger) prior to screening.

  • Implantable cardioverter defibrillator (ICD) implantation within 6 weeks ofscreening 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 hearttransplantation in the next 12 months.

Study Design

Total Participants: 55
Treatment Group(s): 2
Primary Treatment: Aficamten
Phase: 2/3
Study Start date:
May 29, 2024
Estimated Completion Date:
January 31, 2030

Study Description

The overall objective of the trial is to determine the efficacy, safety, and tolerability of administration of aficamten in adolescents (12 to < 18 years old) and children (6 to < 12 years old) with symptomatic oHCM. Adolescents and children will be studied in a staged approach involving established favorable pharmacodynamic and safety profiles of aficamten in adolescents followed by further pharmacokinetic modeling to inform the dosing regimen in children. Only the 12 to <18 years old cohort is currently open for enrollment.

The trial will consist of 3 periods:

  1. Period 1 is the randomized, double-blind, placebo-controlled treatment period that will assess the efficacy, safety and tolerability of aficamten in pediatric participants. Duration: 12 weeks.

  2. Period 2 is the open-label extension trial that will assess the long-term safety of aficamten in pediatric participants, and further assess efficacy and tolerability. Duration: 52 weeks.

  3. Period 3 is the long-term extension trial that will assess the long-term safety of aficamten in pediatric participants, and further assess efficacy and tolerability. Duration: 144 weeks.

Connect with a study center

  • The Hospital for Sick Children (SickKids)

    Toronto, Ontario M5G 1E8
    Canada

    Active - Recruiting

  • The Hospital for Sick Children (SickKids)

    Toronto 6167865, Ontario 6093943 M5G 1E8
    Canada

    Site Not Available

  • Careggi University Hospital

    Florence,
    Italy

    Active - Recruiting

  • Careggi University Hospital

    Florence 3176959,
    Italy

    Site Not Available

  • NHO Kagoshima Medical Center

    Kagoshima,
    Japan

    Active - Recruiting

  • NHO Kagoshima Medical Center

    Kagoshima 1860827,
    Japan

    Site Not Available

  • University of Osaka Hospital

    Osaka,
    Japan

    Active - Recruiting

  • University of Osaka Hospital

    Osaka 1853909,
    Japan

    Site Not Available

  • Kitasato University Hospital

    Sagamihara,
    Japan

    Active - Recruiting

  • Kitasato University Hospital

    Sagamihara 11611609,
    Japan

    Site Not Available

  • National Cerebral and Cardiovascular Center

    Suita,
    Japan

    Active - Recruiting

  • National Cerebral and Cardiovascular Center

    Suita 1851483,
    Japan

    Site Not Available

  • Juntendo University Hospital

    Tokyo,
    Japan

    Active - Recruiting

  • Juntendo University Hospital

    Tokyo 1850147,
    Japan

    Site Not Available

  • Unidad de Cardiología Infantil; Hospital Universitario da Coruña

    A Coruña,
    Spain

    Active - Recruiting

  • Unidad de Cardiología Infantil; Hospital Universitario da Coruña

    A Coruña 3119841,
    Spain

    Site Not Available

  • Hospital Sant Joan de Deu

    Barcelona,
    Spain

    Active - Recruiting

  • Hospital Sant Joan de Deu

    Barcelona 3128760,
    Spain

    Site Not Available

  • Alder Hey Children's Hospital

    Liverpool,
    United Kingdom

    Active - Recruiting

  • Alder Hey Children's Hospital

    Liverpool 2644210,
    United Kingdom

    Site Not Available

  • Evelina Children's Hospital

    London, SW3 6NP
    United Kingdom

    Active - Recruiting

  • Great Ormond Street Hospital for Children

    London, WC1N 3BH
    United Kingdom

    Active - Recruiting

  • Royal Brompton Hospital

    London, SW3 6NP
    United Kingdom

    Site Not Available

  • Evelina Children's Hospital

    London 2643743, SW3 6NP
    United Kingdom

    Site Not Available

  • Great Ormond Street Hospital for Children

    London 2643743, WC1N 3BH
    United Kingdom

    Active - Recruiting

  • Phoenix Children's Hospital

    Phoenix, Arizona 85016
    United States

    Active - Recruiting

  • Phoenix Children's Hospital

    Phoenix 5308655, Arizona 5551752 85016
    United States

    Site Not Available

  • Children's Hospital Los Angeles

    Los Angeles, California 90027
    United States

    Active - Recruiting

  • University of California, Los Angeles (UCLA)

    Los Angeles, California 90095
    United States

    Active - Recruiting

  • UCSF Benioff Children's Hospital

    San Francisco, California 94158
    United States

    Site Not Available

  • Children's Hospital Los Angeles

    Los Angeles 5368361, California 5332921 90027
    United States

    Site Not Available

  • University of California, Los Angeles (UCLA)

    Los Angeles 5368361, California 5332921 90095
    United States

    Active - Recruiting

  • UCSF Benioff Children's Hospital

    San Francisco 5391959, California 5332921 94158
    United States

    Site Not Available

  • Children's Hospital Colorado

    Aurora, Colorado 80045
    United States

    Active - Recruiting

  • Children's Hospital Colorado

    Aurora 5412347, Colorado 5417618 80045
    United States

    Site Not Available

  • Children's National Hospital

    Washington, District of Columbia 20010
    United States

    Site Not Available

  • Children's National Hospital

    Washington D.C., District of Columbia 20010
    United States

    Active - Recruiting

  • Children's National Hospital

    Washington D.C. 4140963, District of Columbia 4138106 20010
    United States

    Site Not Available

  • Baptist Health South Florida

    Miami, Florida 33176
    United States

    Site Not Available

  • Nicklaus Children's Hospital

    Miami, Florida 33155
    United States

    Active - Recruiting

  • Nicklaus Children's Hospital

    Miami 4164138, Florida 4155751 33155
    United States

    Site Not Available

  • Ann & Robert H. Lurie Children's Hospital

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • Ann & Robert H. Lurie Children's Hospital

    Chicago 4887398, Illinois 4896861 60611
    United States

    Site Not Available

  • University of Iowa

    Iowa City, Iowa 52242
    United States

    Site Not Available

  • University of Iowa

    Iowa City 4862034, Iowa 4862182 52242
    United States

    Site Not Available

  • Children's Hospital New Orleans

    New Orleans, Louisiana 70018
    United States

    Active - Recruiting

  • Children's Hospital New Orleans

    New Orleans 4335045, Louisiana 4331987 70018
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Active - Recruiting

  • Children's Hospital of Michigan

    Detroit, Michigan 48201
    United States

    Active - Recruiting

  • University of Michigan

    Ann Arbor 4984247, Michigan 5001836 48109
    United States

    Site Not Available

  • Children's Hospital of Michigan

    Detroit 4990729, Michigan 5001836 48201
    United States

    Site Not Available

  • Mayo Clinic

    Rochester, Minnesota 55905
    United States

    Active - Recruiting

  • Mayo Clinic

    Rochester 5043473, Minnesota 5037779 55905
    United States

    Site Not Available

  • Children's Mercy Hospital

    Kansas City, Missouri 64108
    United States

    Active - Recruiting

  • Children's Mercy Hospital

    Kansas City 4393217, Missouri 4398678 64108
    United States

    Site Not Available

  • University of Nebraska Medical Center

    Omaha, Nebraska 68198
    United States

    Active - Recruiting

  • University of Nebraska Medical Center

    Omaha 5074472, Nebraska 5073708 68198
    United States

    Site Not Available

  • Morristown Medical Center

    Morristown, New Jersey 07960
    United States

    Active - Recruiting

  • Morristown Medical Center

    Morristown 5101427, New Jersey 5101760 07960
    United States

    Site Not Available

  • Children's Hospital at Montefiore

    Bronx, New York 10467
    United States

    Site Not Available

  • Columbia University

    New York, New York 10027
    United States

    Site Not Available

  • NYP/Columbia University Medical Center

    New York, New York 10027
    United States

    Active - Recruiting

  • Children's Hospital at Montefiore

    The Bronx, New York 10467
    United States

    Active - Recruiting

  • NYP/Columbia University Medical Center

    New York 5128581, New York 5128638 10027
    United States

    Site Not Available

  • Children's Hospital at Montefiore

    The Bronx 5110266, New York 5128638 10467
    United States

    Site Not Available

  • Duke Clinical Research Institute

    Durham, North Carolina 27701
    United States

    Active - Recruiting

  • Duke Clinical Research Institute

    Durham 4464368, North Carolina 4482348 27701
    United States

    Site Not Available

  • Oregon Health & Science University

    Portland, Oregon 97239
    United States

    Active - Recruiting

  • Oregon Health & Science University

    Portland 5746545, Oregon 5744337 97239
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Children's Hospital of Philadelphia

    Philadelphia 4560349, Pennsylvania 6254927 19104
    United States

    Site Not Available

  • LeBonheur Children's Hospital

    Memphis, Tennessee 38103
    United States

    Active - Recruiting

  • Vanderbilt University Medical Center

    Nashville, Tennessee 37235
    United States

    Active - Recruiting

  • LeBonheur Children's Hospital

    Memphis 4641239, Tennessee 4662168 38103
    United States

    Site Not Available

  • Vanderbilt University Medical Center

    Nashville 4644585, Tennessee 4662168 37235
    United States

    Site Not Available

  • Dell Children's Hospital

    Austin, Texas 78723
    United States

    Active - Recruiting

  • UT Southwestern

    Dallas, Texas 75390
    United States

    Active - Recruiting

  • Dell Children's Hospital

    Austin 4671654, Texas 4736286 78723
    United States

    Site Not Available

  • UT Southwestern

    Dallas 4684888, Texas 4736286 75390
    United States

    Site Not Available

  • Children's Wisconsin

    Milwaukee, Wisconsin 53226
    United States

    Site Not Available

  • Children's Wisconsin

    Milwaukee 5263045, Wisconsin 5279468 53226
    United States

    Site Not Available

Map preview placeholder

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.