A Study of Mavacamten in Adolescent Participants with Symptomatic Obstructive Hypertrophic Cardiomyopathy

Phase 1

Conditions: Symptomatic Obstructive Hypertrophic Cardiomyopathy
MedDRA version: 20.0Level: LLTClassification code: 10020876Term: Hypertrophic obstructive cardiomyopathy Class: 10010331
Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

Registration Number: CTIS2023-505650-17-00

Lead Sponsor: Bristol-Myers Squibb Services Unlimited Company

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 40

Inclusion Criteria

Adolescents aged 12 to <18 at the time of agreeing to participate, Diagnosis of HCM, Presence of LVOT obstruction, Presence of symptoms

Exclusion Criteria

Phenocopy diseases resulting in myocardial hypertrophy not related to sarcomere dysfunction, Evidence of LVEF <50% in prior 6 months, Planned escalation in HCM therapy or upcoming intervention (eg, major cardiac surgery, HCM medication dose increase)

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the effect of mavacamten compared to placebo on left ventricular outflow tract (LVOT) obstruction in adolescents with symptomatic obstructive HCM;Secondary Objective: To evaluate the effect of mavacamten compared to placebo on other clinically meaningful aspects of disease (eg, symptom status, other echocardiographic parameters) in adolescents with symptomatic obstructive HCM, To evaluate the safety of mavacamten in adolescents with symptomatic obstructive HCM, To assess the pharmacokinetics (PK) of mavacamten in adolescents with symptomatic obstructive HCM, To evaluate taste and swallowability of mavacamten capsules in adolescents with symptomatic obstructive HCM after first and multiple doses, To evaluate the effect of mavacamten on patient-reported symptoms;Primary end point(s): Change from baseline in Valsalva LVOT gradient at Week 28

Secondary Outcome Measures