A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy

Bristol-Myers Squibb (BMS)

Status and phase: Enrolling; Phase 3

Conditions: Cardiomyopathy, Hypertrophic

Treatments: Drug: Placebo; Drug: Mavacamten

Study type: Interventional

Funder types: Industry

Identifiers

NCT06253221

2023-505650-17-00 (Other Identifier)

CV027-010

U1111-1290-1175 (Other Identifier)

Details and patient eligibility

About

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).

Enrollment: 40 estimated patients

Sex: All

Ages: 12 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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)

Other protocol-defined Inclusion/Exclusion criteria apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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40 participants in 2 patient groups

Mavacamten

Experimental group

Description:

Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from day 1 to end of treatment at week 200.

Treatment:

Drug: Mavacamten

Placebo

Experimental group

Description:

Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from week 28 to end of treatment at week 200.

Treatment:

Drug: Placebo