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

Cytokinetics

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).

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 randomi…

Eligibility

Age range: 12–17 years
Sex: All
Healthy volunteers: No

Detailed criteria

Inclusion Criteria: * Period 1: Treatment Period * 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 wal…

Interventions

Drug
Aficamten
Oral Tablet
Drug
Placebo
Oral Tablet

Locations (38)

Phoenix Children's Hospital
Phoenix, Arizona
Children's Hospital Los Angeles
Los Angeles, California
University of California, Los Angeles (UCLA)
Los Angeles, California
UCSF Benioff Children's Hospital
San Francisco, California
Children's Hospital Colorado
Aurora, Colorado
Children's National Hospital
Washington D.C., District of Columbia