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

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).
Brief Title
A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Completion Date
Completion Date Type
Estimated
Conditions
Cardiomyopathy, Hypertrophic
Eligibility Criteria
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.
Inclusion Criteria
Inclusion Criteria:

* Diagnosis of HCM
* Presence of LVOT obstruction
* Presence of symptoms

Inclusion/
Gender
All
Gender Based
false
Keywords
HCM
mavacamten
Obstructive Hypertrophic Cardiomyopathy
SCOUT
SCOUT-HCM
Pediatric HCM
Pediatric hypertrophic cardiomyopathy
Symptomatic HCM
oHCM
Healthy Volunteers
No
Last Update Submit Date
Maximum Age
17 Years
Minimum Age
12 Years
NCT Id
NCT06253221
Org Class
Industry
Org Full Name
Bristol-Myers Squibb
Org Study Id
CV027-010
Overall Status
Active, not recruiting
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adolescents (Age 12 Years to < 18 Years) With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Primary Outcomes
Outcome Measure
Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient
Outcome Time Frame
At Week 28
Secondary Ids
Secondary Id
2023-505650-17-00
Secondary Id
U1111-1290-1175
Secondary Outcomes
Outcome Time Frame
At Week 28
Outcome Measure
Change from baseline in resting LVOT gradient
Outcome Time Frame
At Week 28
Outcome Measure
Change from baseline in post-exercise peak LVOT gradient
Outcome Time Frame
At Week 28
Outcome Measure
Change from baseline in maximal wall thickness
Outcome Time Frame
At Week 28
Outcome Measure
Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e')
Outcome Time Frame
From baseline up to Week 28
Outcome Measure
Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)
Outcome Time Frame
From baseline up to Week 28
Outcome Measure
Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg
Outcome Time Frame
From baseline up to Week 28
Outcome Measure
Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28
Outcome Time Frame
From baseline up to Week 28
Outcome Measure
Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28
Outcome Time Frame
Up to Week 218
Outcome Measure
Number of participants with treatment-emergent adverse events (TEAEs)
Outcome Time Frame
Up to Week 218
Outcome Measure
Number of participants with treatment-emergent serious adverse events (TESAEs)
Outcome Time Frame
At Week 28
Outcome Measure
Change from baseline in electrocardiogram (ECG) (QT interval)
Outcome Time Frame
Up to Week 200
Outcome Measure
Number of participants with left ventricular ejection fraction (LVEF) ≤ 30%
Outcome Time Frame
Up to Week 200
Outcome Measure
Number of participants with LVEF < 50%
Outcome Time Frame
Up to Week 200
Outcome Measure
Trough observed plasma concentration (Ctrough)
Outcome Time Frame
Up to Week 200
Outcome Measure
Post-dose plasma concentration of mavacamten
Outcome Time Frame
Up to Week 200
Outcome Measure
Maximum observed concentration (Cmax)
Outcome Time Frame
Up to Week 200
Outcome Measure
Area under the concentration-time curve (AUC)
Outcome Time Frame
At Day 1 and Week 11
Outcome Measure
Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales
Outcome Time Frame
At Week 28
Outcome Measure
Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain
Start Date
Start Date Type
Actual
Status Verified Date
First Submit Date
First Submit QC Date
Std Ages
Child
Maximum Age Number (converted to Years and rounded down)
17
Minimum Age Number (converted to Years and rounded down)
12
Investigators
Investigator Type
Principal Investigator
Investigator Name
Daphne Hsu
Investigator Email
dhsu@montefiore.org
Investigator Phone
718-741-2538