Phase 3
N=112
A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy
HOCM, Hypertrophic Obstructive Cardiomyopathy
Bottom Line
View on ClinicalTrials.gov: NCT04349072 ↗Enrolled (actual)
112
Serious AEs
13.5%
Results posted
Mar 2023
Primary outcome: Primary: Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eligible at Week 16 — 10; 43 Participants — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Mavacamten (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Bristol-Myers Squibb
- Primary completion
- Feb 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eligible at Week 16 |
10; 43 | <0.0001 sig |
| SECONDARY Number of Participants With at Least One Class Improvement From Baseline in New York Heart Association (NYHA) Class at Week 16 |
35; 12 | <0.0001 sig |
| SECONDARY Change From Baseline to Week 16 in Kansas City Cardiomyopathy Questionnaire 23-item Version, Clinical Summary Score (KCCQ-23, CSS) |
10.4; 1.8 | <0.0001 sig |
| SECONDARY Change From Baseline to Week 16 in N-Terminal Pro-b-Type Natriuretic Peptide (NT-proBNP) |
0.35; 1.13 | <0.0001 sig |
| SECONDARY Change From Baseline to Week 16 in Cardiac Troponin |
0.50; 1.03 | <0.0001 sig |
| SECONDARY Change From Baseline to Week 16 in Post-Exercise Left Ventricular Outflow Tract (LVOT) Gradient |
-39.1; -1.8 | <0.0001 sig |
Summary
This is a randomized, double-blind, placebo-controlled, multi-center study in the United States (U.S.) that will evaluate the effect of mavacamten treatment on reducing the number of septal reduction therapy (SRT) procedures performed in subjects with symptomatic obstructive hypertrophic cardiomyopathy (oHCM [also known as HOCM]) who are eligible for SRT based on ACCF/AHA 2011 and/or ESC 2014 guidelines.
Eligibility Criteria
Key Inclusion Criteria
- At least 18 years old at screening and body weight > 45 kg at screening
- Diagnosed with oHCM consistent with current ACCF/AHA 2011 and meet their recommendations for invasive therapies
- Referred or under active consideration within the past 12 months for SRT procedure and willing to have SRT procedure
- Has documented left ventricular ejection fraction (LVEF) ≥ 60% at Screening
- Has documented oxygen saturation at rest ≥ 90% at Screening
Key Exclusion Criteria
- Persistent or permanent atrial fibrillation and subject not on anticoagulation for ≥ 4 weeks prior to screening and/or not adequately rate controlled ≤ 6 months prior to screening
- Previously treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA])
- For individuals on beta blockers, calcium channel blockers, or disopyramide, any dose adjustment of these medications < 14 days prior to screening or an anticipated change in regimen during the first 16 weeks of the study
- Any medical condition that precludes upright exercise stress testing
- Paroxysmal, intermittent atrial fibrillation with atrial fibrillation present at screening
- Prior treatment with cardiotoxic agents, such as doxorubicin or similar
- Has a history or evidence of any other clinically significant disorder, condition, or disease that would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion
Data sourced from ClinicalTrials.gov (NCT04349072). 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.