Actively Recruiting
A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
Led by Cytokinetics · Updated on 2026-04-13
55
Participants Needed
38
Research Sites
291 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males and females between 12 and under 18 years of age at screening and at Day 1
- Body weight at least 45 kg for the initial cohort; at least 35 kg after 10 participants have completed dose titration without low LVEF events
- Core lab confirmed obstructive hypertrophic cardiomyopathy with specified left ventricular hypertrophy criteria
- Left ventricular ejection fraction (LVEF) of at least 60% and Valsalva left ventricular outflow tract gradient (LVOT-G) of at least 50 mmHg
- Sarcomeric origin of oHCM confirmed by genetic testing or presumed without metabolic, neuromuscular, malformation, infiltrative, or endocrine disorders
- New York Heart Association Class II or higher at screening
- Adequate acoustic windows for echocardiography
- Stable doses of beta blockers, verapamil, diltiazem, or disopyramide for more than 4 weeks prior to randomization
- For open-label extension: completion of Period 1 and LVEF at least 55% after washout
- For long-term extension: completion of Period 2
You will not qualify if you...
- Moderate or severe valvular aortic stenosis or fixed subaortic obstruction
- Mitral regurgitation greater than mild and not due to systolic anterior motion of the mitral valve
- Fixed left-sided obstruction such as subaortic membrane, aortic valve stenosis, or coarctation of the aorta
- History of left ventricular systolic dysfunction with LVEF below 45% or stress cardiomyopathy
- History of congenital heart disease other than oHCM, unless not hemodynamically significant
- Treatment with surgical myectomy or alcohol septal ablation within 6 months or planned during trial
- History of paroxysmal or persistent atrial fibrillation or atrial flutter
- History of syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia within 3 months
- Any clinically significant disorder, active infection, malignancy, or condition posing risk or interfering with trial
- Current or prior use of drugs known to cause cardiomyopathy such as anthracyclines, trastuzumab, cyclophosphamide, sunitinib, or imatinib
- Participation in another investigational drug or device trial within 1 month or 5 half-lives of screening
- Implantable cardioverter defibrillator implantation within 6 weeks or planned during trial
- Prior treatment with aficamten or mavacamten
- Listed or expected to be listed for heart transplantation within 12 months
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 38 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
3
University of California, Los Angeles (UCLA)
Los Angeles, California, United States, 90095
Actively Recruiting
4
UCSF Benioff Children's Hospital
San Francisco, California, United States, 94158
Not Yet Recruiting
5
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
6
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
7
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
8
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, United States, 60611
Actively Recruiting
9
University of Iowa
Iowa City, Iowa, United States, 52242
Withdrawn
10
Children's Hospital New Orleans
New Orleans, Louisiana, United States, 70018
Actively Recruiting
11
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
12
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
Actively Recruiting
13
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
14
Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
Actively Recruiting
15
University of Nebraska Medical Center
Omaha, Nebraska, United States, 68198
Actively Recruiting
16
Morristown Medical Center
Morristown, New Jersey, United States, 07960
Actively Recruiting
17
NYP/Columbia University Medical Center
New York, New York, United States, 10027
Actively Recruiting
18
Children's Hospital at Montefiore
The Bronx, New York, United States, 10467
Actively Recruiting
19
Duke Clinical Research Institute
Durham, North Carolina, United States, 27701
Actively Recruiting
20
Oregon Health & Science University
Portland, Oregon, United States, 97239
Actively Recruiting
21
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
22
LeBonheur Children's Hospital
Memphis, Tennessee, United States, 38103
Actively Recruiting
23
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37235
Actively Recruiting
24
Dell Children's Hospital
Austin, Texas, United States, 78723
Actively Recruiting
25
UT Southwestern
Dallas, Texas, United States, 75390
Actively Recruiting
26
Children's Wisconsin
Milwaukee, Wisconsin, United States, 53226
Not Yet Recruiting
27
The Hospital for Sick Children (SickKids)
Toronto, Ontario, Canada, M5G 1E8
Actively Recruiting
28
Azienda Ospedaliera Universitaria Meyer IRCCS
Florence, Italy
Actively Recruiting
29
NHO Kagoshima Medical Center
Kagoshima, Japan
Actively Recruiting
30
University of Osaka Hospital
Osaka, Japan
Actively Recruiting
31
Kitasato University Hospital
Sagamihara, Japan
Actively Recruiting
32
National Cerebral and Cardiovascular Center
Suita, Japan
Actively Recruiting
33
Juntendo University Hospital
Tokyo, Japan
Actively Recruiting
34
Unidad de Cardiología Infantil; Hospital Universitario da Coruña
A Coruña, Spain
Actively Recruiting
35
Hospital Sant Joan de Deu
Barcelona, Spain
Actively Recruiting
36
Alder Hey Children's Hospital
Liverpool, United Kingdom
Actively Recruiting
37
Evelina Children's Hospital
London, United Kingdom, SW3 6NP
Actively Recruiting
38
Great Ormond Street Hospital for Children
London, United Kingdom, WC1N 3BH
Actively Recruiting
Research Team
C
Cytokinetics MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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