A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
Start Date
5/29/2024
Completion Date
1/1/2030
Summary
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).
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.
Eligibility Criteria
Age Range: 12 years to 17 years
Interventions
Aficamten
Placebo
Conditions
Locations
Phoenix Children's Hospital
Phoenix, Arizona 85016
United States
Children's Hospital Los Angeles
Los Angeles, California 90027
United States
University of California, Los Angeles (UCLA)
Los Angeles, California 90095
United States
UCSF Benioff Children's Hospital
San Francisco, California 94158
United States
Children's Hospital Colorado
Aurora, Colorado 80045
United States
Children's National Hospital
Washington D.C., District of Columbia 20010
United States
Nicklaus Children's Hospital
Miami, Florida 33155
United States
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois 60611
United States
University of Iowa
Iowa City, Iowa 52242
United States
Children's Hospital New Orleans
New Orleans, Louisiana 70018
United States
University of Michigan
Ann Arbor, Michigan 48109
United States
Children's Hospital of Michigan
Detroit, Michigan 48201
United States
Mayo Clinic
Rochester, Minnesota 55905
United States
Children's Mercy Hospital
Kansas City, Missouri 64108
United States
University of Nebraska Medical Center
Omaha, Nebraska 68198
United States
Morristown Medical Center
Morristown, New Jersey 07960
United States
NYP/Columbia University Medical Center
New York, New York 10027
United States
Children's Hospital at Montefiore
The Bronx, New York 10467
United States
Duke Clinical Research Institute
Durham, North Carolina 27701
United States
Oregon Health & Science University
Portland, Oregon 97239
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
LeBonheur Children's Hospital
Memphis, Tennessee 38103
United States
Vanderbilt University Medical Center
Nashville, Tennessee 37235
United States
Dell Children's Hospital
Austin, Texas 78723
United States
UT Southwestern
Dallas, Texas 75390
United States
Children's Wisconsin
Milwaukee, Wisconsin 53226
United States
The Hospital for Sick Children (SickKids)
Toronto, Ontario M5G 1E8
Canada
Azienda Ospedaliera Universitaria Meyer IRCCS
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, SW3 6NP
United Kingdom
Great Ormond Street Hospital for Children
London, WC1N 3BH
United Kingdom