Phase 2
N=59
A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)
Non-obstructive Hypertrophic Cardiomyopathy
Bottom Line
View on ClinicalTrials.gov: NCT03442764 ↗Enrolled (actual)
59
Serious AEs
12.4%
Results posted
Aug 2022
Primary outcome: Primary: Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE) — 88.9; 90.5; 68.4 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- mavacamten (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- MyoKardia, Inc.
- Primary completion
- Jan 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE) |
88.9; 90.5; 68.4 | — |
| PRIMARY Percentage of Participants Who Experienced at Least One Serious Treatment-emergent Adverse Event (STEAE) |
11.1; 9.5; 21.1 | — |
Summary
This is a multicenter, exploratory, randomized, double-blind study of the administration of mavacamten in 60 participants with symptomatic nHCM randomized to receive a 16-week course of mavacamten doses titrated to achieve 1 of 2 target drug concentrations.
Eligibility Criteria
Key Inclusion Criteria
- Diagnosed with nHCM (hypertrophied and non-dilated left ventricle in absence of systemic or other known cause), with LV wall thickness ≥ 15mm at Screening or ≥ 13mm with a positive family history of HCM.
- Age 18 and greater, Body weight > 45kg
- Documented LVEF ≥ 55% at the Screening as determined by echo central lab
- LVOT gradient 30 mmHg unless subsequently treated by septal reduction
- Has QTc Fridericia (QTcF) >480 ms or any other ECG abnormality considered by the investigator to pose a risk to participant safety (eg, second-degree atrioventricular block type II)
- Has persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate-controlled within 1 year of Screening
- History of clinically significant malignant disease within 10 years such as non-metastatic cutaneous squamous cell or basal cell carcinoma
- History or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator or MyoKardia physician, would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion.
Data sourced from ClinicalTrials.gov (NCT03442764). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.