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Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 2 / Phase 3INTERVENTIONAL

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

A Phase 2/3 Multicenter, Randomized, Double-Blind, Placebo-Controlled and Open-Label Extension Trial to Evaluate the Efficacy and Safety of Aficamten in a Pediatric Population With Symptomatic Obstructive Hypertrophic Cardiomyopathy

A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM). (NCT06412666) is a Phase 2 / Phase 3 interventional studying Pediatric and Symptomatic Obstructive Hypertrophic Cardiomyopathy, sponsored by Cytokinetics. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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).

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Pediatric and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

Target enrollment of 55 participants puts this in the typical range for a Phase 2-style efficacy study or a moderate Phase 3 trial in a focused Pediatric subpopulation. At this scale, the study has enough statistical power to detect a clear treatment effect but is not the largest cohort in the field.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 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. Who Should NOT Join This Trial: - Period 1: Treatment Period Any of the following criteria will exclude potential participants from the trial: - Significant valvular heart disease. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
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 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.

Treatments Being Tested

DRUG

Aficamten

Oral Tablet

DRUG

Placebo

Oral Tablet

Locations (20)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Phoenix Children's Hospital
Phoenix, Arizona, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
University of California, Los Angeles (UCLA)
Los Angeles, California, United States
UCSF Benioff Children's Hospital
San Francisco, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, United States
University of Iowa
Iowa City, Iowa, United States
Children's Hospital New Orleans
New Orleans, Louisiana, United States
University of Michigan
Ann Arbor, Michigan, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
Morristown Medical Center
Morristown, New Jersey, United States
NYP/Columbia University Medical Center
New York, New York, United States
Children's Hospital at Montefiore
The Bronx, New York, United States
Duke Clinical Research Institute
Durham, North Carolina, United States
Oregon Health & Science University
Portland, Oregon, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT06412666), the sponsor (Cytokinetics), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT06412666 clinical trial studying?

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). The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT06412666?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT06412666?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT06412666. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT06412666. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.