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

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 PK of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

Key Dates

Start date

May 29, 2024

Status verified

May 2025

Primary completion

Jan 31, 2030

Completion

Jan 31, 2030

Study Design

Enrollment

55 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 ]

Central Contacts

• Cytokinetics MD
  6506242929
  [email protected]

Locations (26)

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