2023-505650-17-00RecruitingPhase 3
A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adolescents (age 12 years to < 18 years) with Symptomatic Obstructive Hypertrophic Cardiomyopathy (SCOUT-HCM)
Bristol-Myers Squibb Services Unlimited Company11 sites in 5 countries11 target enrollmentStarted: April 25, 2024Last updated:
Drugs
Trial Snapshot
- Phase
- Phase 3
- Status
- Recruiting
- Sponsor
- Enrollment
- 11
- Locations
- 11
- Primary Endpoint
- Change from baseline in Valsalva LVOT gradient at Week 28
Study Overview
Brief Summary
To evaluate the effect of mavacamten compared to placebo on left ventricular outflow tract (LVOT) obstruction in adolescents with symptomatic obstructive HCM
Eligibility Criteria
- Ages
- 0 years to 17 years (0-17 Years)
- Accepts Healthy Volunteers
- Yes
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)
Outcomes
Primary Outcomes
Change from baseline in Valsalva LVOT gradient at Week 28
Change from baseline in Valsalva LVOT gradient at Week 28
Secondary Outcomes
- Change from baseline in the following at Week 28: 1) Resting LVOT gradient 2) Post-exercise peak LVOT gradient 3) Maximal wall thickness 4) Ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e’)
- Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)
- Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg
- Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28
- Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28 Incidence of treatment emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs)
- Change from baseline in QT interval on electrocardiogram at Week 28
- Incidence of left ventricular ejection fraction (LVEF) ≤ 30%
- Incidence of LVEF < 50%
- Summary of plasma concentrations by visit (Ctrough and post dose)
- Cmax and AUC using sparse sampling utilizing population PK approach
- 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 HCMSQ SoB domain at Week 28
Investigators
GSM-CT
Scientific
Bristol-Myers Squibb Services Unlimited Company
Study Sites (11)
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