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A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy
About
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).
Primary Purpose
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
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.
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Study Stats
Protocol No.
23-5208
Category
Heart/Cardiovascular Diseases
Pediatric and Prenatal Disorders
Principal Investigator
Contact
Location
- UCLA Westwood