Efficacy and Safety of Aficamten in Children and Adolescents With Obstructive Hypertrophic Cardiomyopathy: Study Design and Rationale of CEDAR-HCM.
Juan Pablo Kaski, Paul F Kantor, Stephanie J Nakano...
https://pubmed.ncbi.nlm.nih.gov/41347307Actively Recruiting
Phase 2
Phase 3
Age: 12Years - 17Years
All Genders
ID06412666
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
Led by Cytokinetics · Updated on 2026-04-13
55
Participants Needed
38
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying aficamten in children and adolescents aged 6 to under 18 years with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), a condition causing thickened heart muscle and outflow obstruction. The study aims to evaluate the drug's effectiveness, safety, and how the body processes it in this pediatric group. Adolescents and children will be studied sequentially, starting with adolescents to establish safe and effective dosing before proceeding to younger children. The trial is a Phase 2/3 randomized, double-blind, placebo-controlled study with additional open-label extension periods for long-term assessment. Participants will be assigned randomly to receive either oral aficamten or a placebo once daily. The initial treatment period lasts 12 weeks and is double-blinded. After this, all participants receive aficamten for 52 weeks during an open-label extension, followed by a long-term extension of 144 weeks to further assess safety, tolerability, and efficacy. Doses range from 5 mg to 20 mg and are adjusted based on echocardiography findings. This staged approach ensures careful monitoring and dosing adjustments throughout the study. During the study, participants will undergo regular evaluations including heart ultrasounds to measure heart function and obstruction, blood tests for heart biomarkers, and assessments of heart failure symptoms using the New York Heart Association functional class. Researchers will monitor how aficamten affects the heart's outflow tract gradient over 12 weeks and track long-term safety over several years. The study includes multiple visits for clinical assessments, laboratory tests, and drug level measurements to understand treatment effects and ensure participant safety.
CONDITIONS
Brief Title
A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
Who Can Participate
Age: 12Years - 17Years
All Genders
Eligibility Criteria
You may qualify if you...
- Males and females aged 12 to under 18 years at screening and Day 1
- Body weight at least 45 kg for initial cohort, then at least 35 kg after safety review
- Confirmed obstructive hypertrophic cardiomyopathy by core lab echocardiography with specific wall thickness criteria
- Left ventricular ejection fraction (LVEF) of 60% or higher and Valsalva LVOT gradient of 50 mmHg or higher
- oHCM of sarcomeric origin confirmed by genetic testing or presumed without other metabolic or neuromuscular diseases
- New York Heart Association (NYHA) Class II or higher at screening
- Adequate echocardiographic acoustic windows
- Stable doses of beta blockers, verapamil, diltiazem, or disopyramide for more than 4 weeks prior to randomization
- For extension periods, completion of prior periods with LVEF 55% or higher after washout
You will not qualify if you...
- Significant valvular heart disease such as moderate/severe aortic stenosis or fixed subaortic obstruction
- Mitral regurgitation more than mild and not due to systolic anterior motion
- Fixed left-sided obstruction like subaortic membrane, aortic valve stenosis, or aortic coarctation
- 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
- Surgical myectomy or alcohol septal ablation within 6 months or planned during trial
- History of atrial fibrillation or atrial flutter
- Syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia within 3 months prior to screening
- Other significant disorders, malignancy, active infection, or conditions posing safety risk
- Use of drugs causing cardiomyopathy like anthracyclines, trastuzumab, cyclophosphamide, sunitinib, or imatinib
- Participation in other investigational drug/device trials within 1 month prior to 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
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - 12 weeks
Participants receive a daily oral dose of aficamten or placebo to assess efficacy, safety, and tolerability during a 12-week double-blind period.
Weekly visits for up to 12 weeks
Treatment
Duration - 52 weeks
Participants receive a daily oral dose of open-label aficamten to assess long-term safety, efficacy, and tolerability.
Visits scheduled periodically during the 52 weeks
Treatment
Duration - 144 weeks
Participants continue daily oral dosing of aficamten to further assess long-term safety, efficacy, and tolerability during the long-term extension period.
Visits scheduled periodically during the 144 weeks
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
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Research Team
C
Cytokinetics MD
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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