Heart failure & cardiomyopathiesRandomised controlled trial
Mavacamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy
- Randomised controlled trial
- phase 3
- international
- double-blind
- placebo-controlled
Authors' conclusion
Among patients with nonobstructive HCM, mavacamten did not result in a significantly greater improvement in peak oxygen uptake or decrease in symptoms than placebo.
Study context
- Population
- Adults with symptomatic nonobstructive hypertrophic cardiomyopathy (HCM)
- Participants
- 289 vs 291
- Comparison
- Mavacamten vs placebo
- Mavacamtenn = 289
- Placebon = 291reference
- Primary endpoint
- Change from baseline in peak oxygen uptake and in KCCQ-CSS (0–100, higher = better health status) (two primary end points)
- Follow-up
- Week 48
Primary result
As reported in the abstract
baseline to week 48
Mavacamten
0.52 ml per kilogram of body weight per minute (95% CI, 0.09 to 0.95)
Peak oxygen uptake · Kansas City Cardiomyopathy Questionnaire clinical summary score (KCCQ-CSS) 13.1 points (95% CI, 10.7 to 15.5)
Placebo
0.05 ml per kilogram per minute (95% CI, −0.38 to 0.47)
Peak oxygen uptake · KCCQ-CSS 10.4 points (95% CI, 8.0 to 12.8)
Mavacamten vs PlaceboBetween-group difference, ml per kilogram per minute (95% CI)P value
peak oxygen uptake
0.47(−0.03 to 0.98)
P = 0.07Mavacamten vs PlaceboBetween-group difference, points (95% CI)P value
KCCQ-CSS
2.7(−0.1 to 5.6)
P = 0.06Safety
Reductions in EF and interruptions in the trial regimen
- More common with mavacamten than with placebo
Original publication
Mavacamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy. N Engl J Med. Published 30 August 2025. doi:10.1056/nejmoa2505927
Summarised from the published abstract; every number is checked against it