A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
Cytokinetics
Study ID
NCT06412666
Phase
PHASE2/PHASE3
Status
Active Not Recruiting

Conditions

  • Pediatric
  • Symptomatic Obstructive Hypertrophic Cardiomyopathy

Eligibility Criteria

Sex
ALL
Age
12 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • Aficamten — DRUG
    Oral Tablet
  • Placebo — DRUG
    Oral Tablet

Study Details

The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

Key Dates

First listed
May 14, 2024
Start date
May 29, 2024
Status verified
Aug 2026
Primary completion
Jan 31, 2030
Completion
Jan 31, 2030

Study Design

Enrollment
65 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • 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.

Primary Outcome Measure

Change from baseline in Valsalva left ventricular outflow tract gradient (LVOT-G) [ Time Frame: Baseline to week 12 (Period 1) ]

Locations (24)

FacilityCityStateZIPSite coordinators
Phoenix Children's HospitalPhoenixArizona85016-
Arkansas Children's HospitalLittle RockArkansas72202-
Children's Hospital Los AngelesLos AngelesCalifornia90027-
University of California, Los Angeles (UCLA)Los AngelesCalifornia90095-
Children's Hospital ColoradoAuroraColorado80045-
Children's National HospitalWashington D.C.District of Columbia20010-
Nicklaus Children's HospitalMiamiFlorida33155-
Ann & Robert H. Lurie Children's HospitalChicagoIllinois60611-
University of MichiganAnn ArborMichigan48109-
Children's Hospital of MichiganDetroitMichigan48201-
Mayo ClinicRochesterMinnesota55905-
Children's Mercy HospitalKansas CityMissouri64108-
University of Nebraska Medical CenterOmahaNebraska68198-
Morristown Medical CenterMorristownNew Jersey07960-
NYP/Columbia University Medical CenterNew YorkNew York10027-
Children's Hospital at MontefioreThe BronxNew York10467-
Duke Clinical Research InstituteDurhamNorth Carolina27701-
Oregon Health & Science UniversityPortlandOregon97239-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
LeBonheur Children's HospitalMemphisTennessee38103-
Vanderbilt University Medical CenterNashvilleTennessee37235-
Dell Children's HospitalAustinTexas78723-
UT SouthwesternDallasTexas75390-
Children's WisconsinMilwaukeeWisconsin53226-

Find similar trials in Phoenix, AZ

By research site
Phoenix Children's Hospital· Phoenix, AZArkansas Children's Hospital· Little Rock, ARChildren's Hospital Los Angeles· Los Angeles, CAUniversity of California, Los Angeles (UCLA)· Los Angeles, CAChildren's Hospital Colorado· Aurora, COChildren's National Hospital· Washington D.C., DC

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