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

Phase 3

Active, not recruiting

• Conditions: Cardiomyopathy, Hypertrophic
• Interventions: Drug: Placebo; Drug: Mavacamten

• Registration Number: NCT06253221
• Lead Sponsor: Bristol-Myers Squibb

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-02-02
• Study First Submitted QC: 2024-02-02
• Study First Posted: 2024-02-12
• Study Start: 2024-04-17
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-28
• Last Update Posted: 2025-05-29
• Primary Completion: 2028-02-15
• Study Completion: 2031-03-28

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 40

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from week 28 to end of treatment at week 200.
Mavacamten	Mavacamten	Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from day 1 to end of treatment at week 200.

Primary Outcome Measures

Name	Time	Method
Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient	At Week 28

Secondary Outcome Measures

Name	Time	Method
Change from baseline in resting LVOT gradient	At Week 28
Change from baseline in post-exercise peak LVOT gradient	At Week 28
Change from baseline in maximal wall thickness	At Week 28
Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e')	At Week 28
Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)	From baseline up to Week 28
Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg	From baseline up to Week 28
Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28	From baseline up to Week 28
Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28	From baseline up to Week 28
Number of participants with treatment-emergent adverse events (TEAEs)	Up to Week 218
Number of participants with treatment-emergent serious adverse events (TESAEs)	Up to Week 218
Change from baseline in electrocardiogram (ECG) (QT interval)	At Week 28
Number of participants with left ventricular ejection fraction (LVEF) ≤ 30%	Up to Week 200
Number of participants with LVEF < 50%	Up to Week 200
Trough observed plasma concentration (Ctrough)	Up to Week 200
Post-dose plasma concentration of mavacamten	Up to Week 200
Maximum observed concentration (Cmax)	Up to Week 200
Area under the concentration-time curve (AUC)	Up to Week 200
Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales	At Day 1 and Week 11
Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain	At Week 28

Trial Locations

Locations (47)

Local Institution - 0017; 🇺🇸 Birmingham, Alabama, United States

Local Institution - 0050; 🇺🇸 Phoenix, Arizona, United States

Local Institution - 0032; 🇺🇸 Los Angeles, California, United States

Local Institution - 0008; 🇺🇸 Los Angeles, California, United States

Local Institution - 0033; 🇺🇸 Palo Alto, California, United States

Local Institution - 0044; 🇺🇸 San Diego, California, United States

Local Institution - 0038; 🇺🇸 Aurora, Colorado, United States

Local Institution - 0031; 🇺🇸 Saint Petersburg, Florida, United States

Local Institution - 0053; 🇺🇸 Atlanta, Georgia, United States

Local Institution - 0013; 🇺🇸 Chicago, Illinois, United States

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