Cardiomyopathy, Hypertrophic
Conditions
Keywords
HCM, mavacamten, Obstructive Hypertrophic Cardiomyopathy, SCOUT, SCOUT-HCM, Pediatric HCM, Pediatric hypertrophic cardiomyopathy, Symptomatic HCM, oHCM
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).
Interventions
DRUGMavacamten
Specified dose on specified days
DRUGPlacebo
Specified dose on specified days
Sponsors
Bristol-Myers Squibb
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to 17 Years
Healthy volunteers
No
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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient | At Week 28 |
Secondary
| Measure | Time frame |
|---|---|
| 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 resting LVOT gradient | 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 |
| Change from baseline in electrocardiogram (ECG) (QT interval) | At Week 28 |
Countries
Australia, Canada, France, Germany, Ireland, Italy, Spain, United Kingdom, United States
Outcome results
None listed