A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
NCT ID: NCT06412666
Last Updated: 2025-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2/PHASE3
55 participants
INTERVENTIONAL
2024-05-29
2030-01-31
Brief Summary
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Detailed Description
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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.
Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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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.
Aficamten
Oral Tablet
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.
Aficamten
Oral Tablet
Placebo
Oral Tablet
Interventions
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Aficamten
Oral Tablet
Placebo
Oral Tablet
Eligibility Criteria
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Inclusion Criteria
* 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
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.
12 Years
17 Years
ALL
No
Sponsors
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Cytokinetics
INDUSTRY
Responsible Party
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Principal Investigators
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Cytokinetics MD
Role: STUDY_DIRECTOR
Cytokinetics
Locations
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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
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
LeBonheur Children's Hospital
Memphis, Tennessee, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Dell Children's Hospital
Austin, Texas, United States
UT Southwestern
Dallas, Texas, United States
Children's Wisconsin
Milwaukee, Wisconsin, United States
The Hospital for Sick Children (SickKids)
Toronto, Ontario, Canada
Careggi University Hospital
Florence, , Italy
NHO Kagoshima Medical Center
Kagoshima, , Japan
University of Osaka Hospital
Osaka, , Japan
Kitasato University Hospital
Sagamihara, , Japan
National Cerebral and Cardiovascular Center
Suita, , Japan
Juntendo University Hospital
Tokyo, , Japan
Unidad de Cardiología Infantil; Hospital Universitario da Coruña
A Coruña, , Spain
Hospital Sant Joan de Deu
Barcelona, , Spain
Alder Hey Children's Hospital
Liverpool, , United Kingdom
Evelina Children's Hospital
London, , United Kingdom
Great Ormond Street Hospital for Children
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Andrew Papez
Role: primary
Paul Kantor
Role: primary
Nancy Halnon
Role: primary
Othman Aljohani
Role: primary
Stephanie Nakano
Role: primary
Sairah Khan
Role: primary
Ronald Kanter
Role: primary
Defne Magnetta
Role: primary
Gary Beasley
Role: primary
Tamara Bradford
Role: primary
Mark Russell
Role: primary
Swati Sehgal
Role: primary
Rebecca Ameduri
Role: primary
Brian Birnbaum
Role: primary
Jason Cole
Role: primary
Matthew Martinez
Role: primary
Irene Lytrivi
Role: primary
Daphne Hsu
Role: primary
Kevin Hill
Role: primary
Seshadri Balaji
Role: primary
Humera Ahmed
Role: primary
Kaitlin Ryan
Role: primary
Aarti Dalal
Role: primary
Chesney Castleberry
Role: primary
Nathanya Baez-Hernandez
Role: primary
Alexander Raskin
Role: primary
Bhavikkumar Langanecha
Role: primary
Iacopo Olivotto
Role: primary
Yumiko Ninomiya
Role: primary
Jun Narita
Role: primary
Yoichiro Hirata
Role: primary
Kenichi Kurosaki
Role: primary
Hideo Fukunaga
Role: primary
Fernando R Nunez
Role: primary
Georgia Sarquella Brugada
Role: primary
Bernadette Khodaghalian
Role: primary
Andres Rico-Armada
Role: primary
Juan P Kaski
Role: primary
Other Identifiers
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2024-511377-30-00
Identifier Type: CTIS
Identifier Source: secondary_id
CY 6023
Identifier Type: -
Identifier Source: org_study_id