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

Status: Recruiting
Location: See all (38) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
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).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 17
Healthy Volunteers: f
View:

• 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 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.

Locations
United States
Arizona
Phoenix Children's Hospital
NOT_YET_RECRUITING
Phoenix
California
Children's Hospital Los Angeles
RECRUITING
Los Angeles
University of California, Los Angeles (UCLA)
RECRUITING
Los Angeles
UCSF Benioff Children's Hospital
NOT_YET_RECRUITING
San Francisco
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Washington, D.c.
Children's National Hospital
RECRUITING
Washington D.c.
Florida
Nicklaus Children's Hospital
NOT_YET_RECRUITING
Miami
Iowa
University of Iowa
RECRUITING
Iowa City
Illinois
Ann & Robert H. Lurie Children's Hospital
RECRUITING
Chicago
Louisiana
Children's Hospital New Orleans
RECRUITING
New Orleans
Michigan
University of Michigan
RECRUITING
Ann Arbor
Children's Hospital of Michigan
RECRUITING
Detroit
Minnesota
Mayo Clinic
RECRUITING
Rochester
Missouri
Children's Mercy Hospital
RECRUITING
Kansas City
North Carolina
Duke Clinical Research Institute
NOT_YET_RECRUITING
Durham
Nebraska
University of Nebraska Medical Center
RECRUITING
Omaha
New Jersey
Morristown Medical Center
RECRUITING
Morristown
New York
NYP/Columbia University Medical Center
RECRUITING
New York
Children's Hospital at Montefiore
NOT_YET_RECRUITING
The Bronx
Oregon
Oregon Health & Science University
RECRUITING
Portland
Pennsylvania
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Tennessee
LeBonheur Children's Hospital
RECRUITING
Memphis
Vanderbilt University Medical Center
RECRUITING
Nashville
Texas
Dell Children's Hospital
NOT_YET_RECRUITING
Austin
UT Southwestern
NOT_YET_RECRUITING
Dallas
Wisconsin
Children's Wisconsin
NOT_YET_RECRUITING
Milwaukee
Other Locations
Canada
The Hospital for Sick Children (SickKids)
NOT_YET_RECRUITING
Toronto
Italy
Careggi University Hospital
RECRUITING
Florence
Japan
NHO Kagoshima Medical Center
RECRUITING
Kagoshima
University of Osaka Hospital
NOT_YET_RECRUITING
Osaka
Kitasato University Hospital
RECRUITING
Sagamihara
National Cerebral and Cardiovascular Center
NOT_YET_RECRUITING
Suita
Juntendo University Hospital
NOT_YET_RECRUITING
Tokyo
Spain
Unidad de Cardiología Infantil; Hospital Universitario da Coruña
RECRUITING
A Coruña
Hospital Sant Joan de Deu
NOT_YET_RECRUITING
Barcelona
United Kingdom
Alder Hey Children's Hospital
RECRUITING
Liverpool
Evelina Children's Hospital
NOT_YET_RECRUITING
London
Great Ormond Street Hospital for Children
RECRUITING
London
Contact Information
Primary
Cytokinetics MD
medicalaffairs@cytokinetics.com
6506242929
Time Frame
Start Date: 2024-05-29
Estimated Completion Date: 2030-01
Participants
Target number of participants: 40
Treatments
Experimental: 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.
Placebo_comparator: 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.
Sponsors
Leads: Cytokinetics

This content was sourced from clinicaltrials.gov