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

April 8, 2026 updated by: Cytokinetics

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

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).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Interventional

Enrollment (Estimated)

55

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 1E8
        • Recruiting
        • The Hospital for Sick Children (SickKids)
        • Contact:
          • Bhavikkumar Langanecha
        • Principal Investigator:
          • Bhavikkumar Langanecha
  • Italy
      • Florence, Italy
        • Recruiting
        • Azienda Ospedaliera Universitaria Meyer IRCCS
        • Contact:
          • Iacopo Olivotto
        • Principal Investigator:
          • Iacopo Olivotto
  • Japan
      • Kagoshima, Japan
        • Recruiting
        • NHO Kagoshima Medical Center
        • Contact:
          • Yumiko Ninomiya
        • Principal Investigator:
          • Yumiko Ninomiya
      • Osaka, Japan
        • Recruiting
        • University of Osaka Hospital
        • Contact:
          • Jun Narita
        • Principal Investigator:
          • Jun Narita
      • Sagamihara, Japan
        • Recruiting
        • Kitasato University Hospital
        • Contact:
          • Yoichiro Hirata
        • Principal Investigator:
          • Yoichiro Hirata
      • Suita, Japan
        • Recruiting
        • National Cerebral and Cardiovascular Center
        • Contact:
          • Kenichi Kurosaki
        • Principal Investigator:
          • Kenichi Kurosaki
      • Tokyo, Japan
        • Recruiting
        • Juntendo University Hospital
        • Contact:
          • Hideo Fukunaga
        • Principal Investigator:
          • Hideo Fukunaga
  • Spain
      • A Coruña, Spain
        • Recruiting
        • Unidad de Cardiología Infantil; Hospital Universitario da Coruña
        • Contact:
          • Fernando R Nunez
        • Principal Investigator:
          • Fernando R Nunez
      • Barcelona, Spain
        • Recruiting
        • Hospital Sant Joan de Déu
        • Contact:
          • Georgia Sarquella Brugada
        • Principal Investigator:
          • Georgia Sarquella Brugada
  • United Kingdom
      • Liverpool, United Kingdom
        • Recruiting
        • Alder Hey Children's Hospital
        • Contact:
          • Bernadette Khodaghalian
        • Principal Investigator:
          • Bernadette Khodaghalian
      • London, United Kingdom, WC1N 3BH
        • Recruiting
        • Great Ormond Street Hospital for Children
        • Contact:
          • Juan P Kaski
        • Principal Investigator:
          • Juan P Kaski
      • London, United Kingdom, SW3 6NP
        • Recruiting
        • Evelina Children's Hospital
        • Contact:
          • Andres Rico-Armada
        • Principal Investigator:
          • Andres Rico-Armada
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Recruiting
        • Phoenix Children's Hospital
        • Contact:
          • Andrew Papez
        • Principal Investigator:
          • Andrew Papez
    • California
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Children's Hospital Los Angeles
        • Contact:
          • Paul Kantor
        • Principal Investigator:
          • Paul Kantor
      • Los Angeles, California, United States, 90095
        • Recruiting
        • University of California, Los Angeles (UCLA)
        • Contact:
          • Nancy Halnon
        • Principal Investigator:
          • Nancy Halnon
      • San Francisco, California, United States, 94158
        • Not yet recruiting
        • UCSF Benioff Children's Hospital
        • Contact:
          • Othman Aljohani
        • Principal Investigator:
          • Othman Aljohani
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • Children's Hospital Colorado
        • Contact:
          • Stephanie Nakano
        • Principal Investigator:
          • Stephanie Nakano
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Recruiting
        • Children's National Hospital
        • Contact:
          • Sairah Khan
        • Principal Investigator:
          • Sairah Khan
    • Florida
      • Miami, Florida, United States, 33155
        • Recruiting
        • Nicklaus Children's Hospital
        • Contact:
          • Ronald Kanter
        • Principal Investigator:
          • Ronald Kanter
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Ann & Robert H. Lurie Children's Hospital
        • Contact:
          • Defne Magnetta
        • Principal Investigator:
          • Defne Magnetta
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Withdrawn
        • University of Iowa
    • Louisiana
      • New Orleans, Louisiana, United States, 70018
        • Recruiting
        • Children's Hospital New Orleans
        • Contact:
          • Tamara Bradford
        • Principal Investigator:
          • Tamara Bradford
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan
        • Contact:
          • Mark Russell
        • Principal Investigator:
          • Mark Russell
      • Detroit, Michigan, United States, 48201
        • Recruiting
        • Children's Hospital of Michigan
        • Contact:
          • Swati Sehgal
        • Principal Investigator:
          • Swati Sehgal
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic
        • Contact:
          • Rebecca Ameduri
        • Principal Investigator:
          • Rebecca Ameduri
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Recruiting
        • Children's Mercy Hospital
        • Contact:
          • Brian Birnbaum
        • Principal Investigator:
          • Brian Birnbaum
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • Recruiting
        • University of Nebraska Medical Center
        • Contact:
          • Jason Cole
        • Principal Investigator:
          • Jason Cole
    • New Jersey
      • Morristown, New Jersey, United States, 07960
        • Recruiting
        • Morristown Medical Center
        • Contact:
          • Matthew Martinez
        • Principal Investigator:
          • Matthew Martinez
    • New York
      • New York, New York, United States, 10027
        • Recruiting
        • NYP/Columbia University Medical Center
        • Contact:
          • Irene Lytrivi
        • Principal Investigator:
          • Irene Lytrivi
      • The Bronx, New York, United States, 10467
        • Recruiting
        • Children's Hospital at Montefiore
        • Contact:
          • Daphne Hsu
        • Principal Investigator:
          • Daphne Hsu
    • North Carolina
      • Durham, North Carolina, United States, 27701
        • Recruiting
        • Duke Clinical Research Institute
        • Contact:
          • Kevin Hill
        • Principal Investigator:
          • Kevin Hill
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health & Science University
        • Principal Investigator:
          • Seshadri Balaji
        • Contact:
          • Seshadri Balaji
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
        • Contact:
          • Humera Ahmed
        • Principal Investigator:
          • Humera Ahmed
    • Tennessee
      • Memphis, Tennessee, United States, 38103
        • Recruiting
        • LeBonheur Children's Hospital
        • Contact:
          • Kaitlin Ryan
        • Principal Investigator:
          • Kaitlin Ryan
      • Nashville, Tennessee, United States, 37235
        • Recruiting
        • Vanderbilt University Medical Center
        • Contact:
          • Aarti Dalal
        • Principal Investigator:
          • Aarti Dalal
    • Texas
      • Austin, Texas, United States, 78723
        • Recruiting
        • Dell Children's Hospital
        • Contact:
          • Chesney Castleberry
        • Principal Investigator:
          • Chesney Castleberry
      • Dallas, Texas, United States, 75390
        • Recruiting
        • UT Southwestern
        • Contact:
          • Nathanya Baez-Hernandez
        • Principal Investigator:
          • Nathanya Baez-Hernandez
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Not yet recruiting
        • Children's Wisconsin
        • Contact:
          • Alexander Raskin
        • Principal Investigator:
          • Alexander Raskin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Aficamten
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
Oral Tablet
Placebo Comparator: Placebo
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: Placebo
Oral Tablet
Drug: Aficamten
Oral Tablet

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in Valsalva left ventricular outflow tract gradient (LVOT-G)
Time Frame: Baseline to week 12
Baseline to week 12

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in resting LVOT-G
Time Frame: Baseline to week 12
Baseline to week 12
Change in values of NT-proBNP
Time Frame: Baseline to week 12
Baseline to week 12
Change in values of hs-cTnI
Time Frame: Baseline to week 12
Baseline to week 12
Change in New York Heart Association (NYHA) Functional Class
Time Frame: Baseline to week 12
Baseline to week 12
Proportion of patients with ≥1 class improvement in NYHA Functional Class
Time Frame: Baseline to week 12
Baseline to week 12
Maximum observed concentration (Cmax), tmax, AUCtau, and Ctrough of aficamten
Time Frame: Week 8 or Week 12
A voluntary intensive PK substudy may occur at Week 8 or Week 12
Week 8 or Week 12
Trough observed plasma concentration (Ctrough) and C2h postdose of aficamten
Time Frame: Baseline to week 12
Baseline to week 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cytokinetics

Investigators

  • Study Director: Cytokinetics MD, Cytokinetics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 29, 2024

Primary Completion (Estimated)

January 1, 2030

Study Completion (Estimated)

January 1, 2030

Study Registration Dates

First Submitted

May 9, 2024

First Submitted That Met QC Criteria

May 9, 2024

First Posted (Actual)

May 14, 2024

Study Record Updates

Last Update Posted (Actual)

April 13, 2026

Last Update Submitted That Met QC Criteria

April 8, 2026

Last Verified

May 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CY 6023
  • 2024-511377-30-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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