A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adolescents (age 12 years to < 18 years) with Symptomatic Obstructive Hypertrophic Cardiomyopathy
Recipient organisationNIHR Great Ormond Street Hospital Clinical Research Facility
NIHR supportRecorded as supported by this research centre
PeriodJan 2025 — Apr 2028
In plain English
AI plain-English summary
A drug that relaxes the heart muscle is being tested in teenagers with a rare, inherited condition that blocks blood flow out of the heart. This matters because obstructive hypertrophic cardiomyopathy (HCM) thickens the heart wall and stiffens the left ventricle, making it harder for the heart to pump blood. In adolescents, this causes breathlessness, chest pain, and limits exercise—often preventing them from playing sports or keeping up with peers. Current treatments, such as beta-blockers, are not specifically approved for children, and surgery carries significant risks. The drug mavacamten, already approved for adults, works by reducing the excessive contraction of the heart muscle, which eases the obstruction. If this trial succeeds, it could become the first targeted drug therapy for adolescents with symptomatic obstructive HCM. For roughly 40 participants globally, the study measures whether mavacamten improves exercise capacity and daily symptoms compared to a placebo over 28 weeks, followed by an open-label phase where all receive the drug. A positive result would give clinicians a non-surgical option to manage a condition that otherwise forces young people to limit their physical activity and can lead to heart failure or sudden cardiac arrest.
View original technical description
This is a Phase 3, randomized, placebo-controlled, double-blind, multi-center, international, parallel-group study to evaluate the efficacy safety, and pharmacokinetics (PK) of mavacamten in adolescent patients with symptomatic obstructive HCM.The purpose of this study is to test the hypothesis that treatment with oral mavacamten compared to placebo improves health status (symptoms and physical limitations) and increases exercise capacity in symptomatic adolescents with obstructive HCM. The effect of treatment on health status and exercise capacity will be extrapolated using change from baseline in VLVOT.Double-blind treatment with oral mavacamten or placebo will occur for the first 28 weeks. The primary endpoint for all participants will be assessed at Week 28. Participants randomized to placebo will receive mavacamten starting at Week 28; all participants will receive mavacamten until the end of treatment visit at Week 56. A separate rollover/extension study may be offered to eligible participants who finish their study intervention per protocol.About 40 study participants will be taking part in this study globally and participation will last up to 79 weeks. This is counted from the date that the participant signs the Informed Consent Form, until their last study visit.Study intervention is defined as mavacamten or placebo.The study has 4 phases:• Screening (up to 5 weeks)• Double-blind Placebo-controlled (28 weeks)• Double-blind Active treatment (28 weeks)• Follow-up (participants not continuing to the rollover/extension: 8 or 18 weeks for CYP2C19 poor metabolizers)Patients will visit the study hospital for the following:• Day 1• Week 4• Week 5• Week 8• Week 9• Week 11• Week 12• Week 16• Week 20• Week 23• Week 24• Week 28• Week 32• Week 33• Week 36• Week 37• Week 39• Week 40• Week 44• Week 48• Week 51• Week 52• Week 56/EOT• Week 64/EOS 1• Week 74/EOS 2
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