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PediatricPhase 2/3CEDAR-HCMNCT06412666

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

A Phase 2/3 study testing Aficamten in people with Pediatric and Symptomatic Obstructive Hypertrophic Cardiomyopathy, run by Cytokinetics at 36 sites in 6 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Read more about this study

Who is it for?
Ages 12 to 17 years
What is being tested?
Aficamten
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
65 participants
Where
36 sitesin 6 countries
Study dates
29 May 2024to Jan 2030
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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:
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  • 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

Who cannot join

  • 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).
Show all 17Show fewer
  • 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.

Trial locations and contacts

36 sites in 6 countries.

36 sites in 6 countries

United States24 sites
  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Dell Children's Hospital

    Austin, Texas, 78723

  • Ann & Robert H. Lurie Children's Hospital

    Chicago, Illinois, 60611

  • UT Southwestern

    Dallas, Texas, 75390

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201

  • Duke Clinical Research Institute

    Durham, North Carolina, 27701

  • Children's Mercy Hospital

    Kansas City, Missouri, 64108

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027

  • University of California, Los Angeles (UCLA)

    Los Angeles, California, 90095

  • LeBonheur Children's Hospital

    Memphis, Tennessee, 38103

  • Nicklaus Children's Hospital

    Miami, Florida, 33155

  • Children's Wisconsin

    Milwaukee, Wisconsin, 53226

  • Morristown Medical Center

    Morristown, New Jersey, 07960

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37235

  • NYP/Columbia University Medical Center

    New York, New York, 10027

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • Children's Hospital at Montefiore

    The Bronx, New York, 10467

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010

Other trials in United States

Japan5 sites
  • NHO Kagoshima Medical Center

    Kagoshima

  • University of Osaka Hospital

    Osaka

  • Kitasato University Hospital

    Sagamihara

  • National Cerebral and Cardiovascular Center

    Suita

  • Juntendo University Hospital

    Tokyo

Other trials in Japan

United Kingdom3 sites
  • Alder Hey Children's Hospital

    Liverpool

  • Evelina Children's Hospital

    London, SW3 6NP

  • Great Ormond Street Hospital for Children

    London, WC1N 3BH

Other trials in United Kingdom

Spain2 sites
  • Unidad de Cardiología Infantil; Hospital Universitario da Coruña

    A Coruña

  • Hospital Sant Joan de Deu

    Barcelona

Other trials in Spain

Canada1 site
  • The Hospital for Sick Children (SickKids)

    Toronto, Ontario, M5G 1E8

Other trials in Canada

Italy1 site
  • Azienda Ospedaliera Universitaria Meyer IRCCS

    Florence

Other trials in Italy

About this study

Official title: 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 pharmacokinetics of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

Read the 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.

The 12 to <18 years old cohort has enrolled all participants and is no longer recruiting, the 6 to < 12 year old cohort is not yet recruiting.

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.
  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.
  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.
Being tested
Aficamten, Placebo
Keywords
CK-3773274, CK-274, Aficamten, Symptomatic Obstructive Hypertrophic Cardiomyopathy, oHCM, CEDAR, CEDAR-HCM, CY 6023

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AficamtenDrug

    Oral Tablet

  • PlaceboDrug

    Oral Tablet

Study groups in NCT06412666
Study groupRoleWhat they receive
AficamtenExperimentalParticipants 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.
Receives: Drug: Aficamten
PlaceboPlacebo comparatorParticipants 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.
Receives: Drug: Aficamten; Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: Baseline to week 12 (Period 1)

Secondary outcomes (16)
  1. Change from baseline in resting LVOT-G

    Measured: Baseline to week 12 (Period 1)

  2. Trough observed plasma concentration (Ctrough) and concentration 2 hours postdose of aficamten

    Measured: Baseline to week 12 (Period 1)

  3. Change in values of N-terminal prohormone brain natriuretic peptide (NT-proBNP)

    Measured: Baseline to week 12 (Period 1)

  4. Change in values of high sensitivity cardiac troponin I (hs-cTnI)

    Measured: Baseline to week 12 (Period 1)

  5. Change in New York Heart Association (NYHA) Functional Class

    Measured: Baseline to week 12 (Period 1), week 14 to week 66 (Period 2), and up to week 210 (Period 3)

  6. Change in peak LVOT-G

    Change in peak LVOT-G at rest and with valsalva provocation will be assessed at 12 week intervals during Period 2 and 24 week intervals during Period 3

    Measured: Week 14 to week 66 (Period 2), and up to week 210 (Period 3)

  7. Proportion of participants with ≥1 class improvement in NYHA Functional Class

    Measured: Baseline to week 12 (Period 1), week 14 to week 66 (Period 2), and up to week 210 (Period 3)

  8. Number of participants that have drug interruption or early discontinuation

    Measured: Up to week 210

  9. Number of participants with appropriate implantable cardioverter defibrillator (ICD) discharges

    Measured: Up to week 210

  10. Number of participants with left ventricular ejection fraction (LVEF) < 50%

    Measured: Baseline to week 12 (Period 1), week 14 to week 66 (Period 2), and up to week 210 (Period 3)

  11. Number of participants with clinically significant changes in vital signs, 12-lead electrocardiogram (ECGs) and safety laboratory parameters

    Measured: Up to week 210

  12. Proportion of participants with Valsalva LVOT-G < 50 mmHg

    Measured: Week 14 to week 66 (Period 2), and up to week 210 (Period 3)

  13. Proportion of participants with Valsalva LVOT-G < 30 mmHg

    Measured: Week 14 to week 66 (Period 2), and up to week 210 (Period 3)

  14. Time to first resting LVOT-G < 30 mmHg through last follow-up

    Measured: Time to the following event through last follow-up, up to week 210

  15. Time to first valsalva LVOT-G < 50 mmHg through last follow-up

    Measured: Time to event through last follow-up, up to week 210

  16. Time to first valsalva LVOT-G < 30 mmHg through last follow-up

    Measured: Time to event through last follow-up, up to week 210

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 May 2024
  2. Study start29 May 2024
  3. Primary completionJan 2030 (estimated)
  4. Study completionJan 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Investigator, Outcomes assessor
Enrolment
65 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06412666
Sponsor's protocol ID
CY 6023
Other IDs
2024-511377-30-00
Acronym
CEDAR-HCM
Status last verified
Aug 2026
First submitted
9 May 2024
Last updated
18 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 41347307

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Aficamten in people with Pediatric and Symptomatic Obstructive Hypertrophic Cardiomyopathy, run by Cytokinetics at 36 sites in 6 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 18 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 18 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 36 sites in United States, Japan, United Kingdom, Spain, Canada and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from baseline in Valsalva left ventricular outflow tract gradient (LVOT-G) (time frame: Baseline to week 12 (Period 1)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06412666, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Aug 2026.

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