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New drug aims to help kids with stiff heart muscle breathe easier

NCT ID NCT06412666

Disease control Sponsor: Cytokinetics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 19, 2026 · Updated 2 times

Summary

This study tests a medicine called aficamten in children aged 12 to 18 with a heart condition that makes it hard for blood to pump out. The goal is to see if the drug can reduce pressure inside the heart and improve symptoms like shortness of breath. About 55 participants will receive either aficamten or a placebo, and all will have the option to continue on the drug in an open-label phase.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 65 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2024

Expected to finish

Jan 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

hypertrophic cardiomyopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Pediatric Symptomatic obstructive hypertrophic cardiomyopathy

Contacts and locations

Locations

  • Alder Hey Children's Hospital

    Liverpool, United Kingdom

  • Ann & Robert H. Lurie Children's Hospital

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Azienda Ospedaliera Universitaria Meyer IRCCS

    Florence, Italy

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital at Montefiore

    The Bronx, New York, 10467, United States

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Mercy Hospital

    Kansas City, Missouri, 64108, United States

  • Children's National Hospital

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

  • Children's Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Dell Children's Hospital

    Austin, Texas, 78723, United States

  • Duke Clinical Research Institute

    Durham, North Carolina, 27701, United States

  • Evelina Children's Hospital

    London, SW3 6NP, United Kingdom

  • Great Ormond Street Hospital for Children

    London, WC1N 3BH, United Kingdom

  • Hospital Sant Joan de Deu

    Barcelona, Spain

  • Juntendo University Hospital

    Tokyo, Japan

  • Kitasato University Hospital

    Sagamihara, Japan

  • LeBonheur Children's Hospital

    Memphis, Tennessee, 38103, United States

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • Morristown Medical Center

    Morristown, New Jersey, 07960, United States

  • NHO Kagoshima Medical Center

    Kagoshima, Japan

  • NYP/Columbia University Medical Center

    New York, New York, 10027, United States

  • National Cerebral and Cardiovascular Center

    Suita, Japan

  • Nicklaus Children's Hospital

    Miami, Florida, 33155, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • The Hospital for Sick Children (SickKids)

    Toronto, Ontario, M5G 1E8, Canada

  • UT Southwestern

    Dallas, Texas, 75390, United States

  • Unidad de Cardiología Infantil; Hospital Universitario da Coruña

    A Coruña, Spain

  • University of California, Los Angeles (UCLA)

    Los Angeles, California, 90095, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198, United States

  • University of Osaka Hospital

    Osaka, Japan

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37235, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.