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

NCT06412666 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: CY 60232024-511377-30-00
• Study Type: interventional
• Target: 55
• Locations: 6 countries
• Sites: 38

Brief 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).

Conditions

Pediatric; Symptomatic Obstructive Hypertrophic Cardiomyopathy

Keywords

CK-3773274; CK-274; Aficamten; Symptomatic Obstructive Hypertrophic Cardiomyopathy; oHCM; CEDAR; CEDAR-HCM; CY 6023

Outcome Measures

Primary Outcomes (1)

• Change from baseline in Valsalva left ventricular outflow tract gradient (LVOT-G)

  Baseline to week 12

Secondary Outcomes (7)

• Change from baseline in resting LVOT-G

  Baseline to week 12

• Change in values of NT-proBNP

  Baseline to week 12

• Change in values of hs-cTnI

  Baseline to week 12

• Change in New York Heart Association (NYHA) Functional Class

  Baseline to week 12

• Proportion of patients with ≥1 class improvement in NYHA Functional Class

  Baseline to week 12

Study Arms (2)

Aficamten

EXPERIMENTAL

Participants in this arm will receive a single daily oral dose of aficamten with dose levels (5 mg to 20 mg) guided by echocardiography assessments, for 12 weeks during the double-blinded period, for another 52 weeks during the open-label extension period, and for an additional 144 weeks during the long-term extension period.

Drug: Aficamten

Placebo

PLACEBO COMPARATOR

Participants in this arm will receive a single daily oral dose of placebo for 12 weeks during the double-blinded period and then will receive aficamten for 52 weeks during the open-label extension period, followed by an additional 144 weeks of aficamten during the long-term extension period.

Drug: Aficamten; Drug: Placebo

Interventions

Aficamten DRUG

Oral Tablet

Aficamten; Placebo

Placebo DRUG

Oral Tablet

Placebo

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Cytokinetics: lead

Study Sites (38)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

RECRUITING

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

University of California, Los Angeles (UCLA)

Los Angeles, California, 90095, United States

RECRUITING

UCSF Benioff Children's Hospital

San Francisco, California, 94158, United States

NOT YET RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

Children's National Hospital

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

RECRUITING

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

RECRUITING

Ann & Robert H. Lurie Children's Hospital

Chicago, Illinois, 60611, United States

RECRUITING

University of Iowa

Iowa City, Iowa, 52242, United States

WITHDRAWN

Children's Hospital New Orleans

New Orleans, Louisiana, 70018, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

RECRUITING

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

RECRUITING

Morristown Medical Center

Morristown, New Jersey, 07960, United States

RECRUITING

NYP/Columbia University Medical Center

New York, New York, 10027, United States

RECRUITING

Children's Hospital at Montefiore

The Bronx, New York, 10467, United States

RECRUITING

Duke Clinical Research Institute

Durham, North Carolina, 27701, United States

RECRUITING

Oregon Health & Science University

Portland, Oregon, 97239, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

LeBonheur Children's Hospital

Memphis, Tennessee, 38103, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37235, United States

RECRUITING

Dell Children's Hospital

Austin, Texas, 78723, United States

RECRUITING

UT Southwestern

Dallas, Texas, 75390, United States

RECRUITING

Children's Wisconsin

Milwaukee, Wisconsin, 53226, United States

NOT YET RECRUITING

The Hospital for Sick Children (SickKids)

Toronto, Ontario, M5G 1E8, Canada

RECRUITING

Azienda Ospedaliera Universitaria Meyer IRCCS

Florence, Italy

RECRUITING

NHO Kagoshima Medical Center

Kagoshima, Japan

RECRUITING

University of Osaka Hospital

Osaka, Japan

RECRUITING

Kitasato University Hospital

Sagamihara, Japan

RECRUITING

National Cerebral and Cardiovascular Center

Suita, Japan

RECRUITING

Juntendo University Hospital

Tokyo, Japan

RECRUITING

Unidad de Cardiología Infantil; Hospital Universitario da Coruña

A Coruña, Spain

RECRUITING

Hospital Sant Joan de Deu

Barcelona, Spain

RECRUITING

Alder Hey Children's Hospital

Liverpool, United Kingdom

RECRUITING

Evelina Children's Hospital

London, SW3 6NP, United Kingdom

RECRUITING

Great Ormond Street Hospital for Children

London, WC1N 3BH, United Kingdom

RECRUITING

Related Publications (1)

• Kaski JP, Kantor PF, Nakano SJ, Olivotto I, Russell MW, Godown J, Chiu M, German P, Heitner SB, Jacoby DL, Kupfer S, Lutz J, Maharao N, Malik FI, Melloni C, Nieto Morales PF, Simkins T, Wei J, Ho CY; CEDAR-HCM Investigators. Efficacy and Safety of Aficamten in Children and Adolescents With Obstructive Hypertrophic Cardiomyopathy: Study Design and Rationale of CEDAR-HCM. Circ Heart Fail. 2026 Feb;19(2):e013418. doi: 10.1161/CIRCHEARTFAILURE.125.013418. Epub 2025 Dec 5.

  PMID: 41347307 DERIVED

Study Officials

• Cytokinetics MD

  Cytokinetics

  STUDY DIRECTOR

Central Study Contacts

Cytokinetics MD

CONTACT

6506242929; medicalaffairs@cytokinetics.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 9, 2024
• First Posted: May 14, 2024
• Study Start: May 29, 2024
• Primary Completion (Estimated): January 1, 2030
• Study Completion (Estimated): January 1, 2030
• Last Updated: April 13, 2026

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

ES (2); IT (1); GB (3); JP (5); CA (1); US (26)