Heart failure & cardiomyopathiesRandomised controlled trial
Mavacamten in Adolescents with Obstructive Hypertrophic Cardiomyopathy
- Randomised controlled trial
- phase 3
- double-blind
- placebo-controlled
Authors' conclusion
Among adolescent patients with obstructive hypertrophic cardiomyopathy, the reduction in left ventricular outflow tract obstruction was significantly greater with mavacamten than with placebo over a 28-week period.
Study context
- Population
- Symptomatic adolescents (12 to <18 years) with NYHA class II or III obstructive hypertrophic cardiomyopathy
- Participants
- 44 adolescents
- Comparison
- Mavacamten vs placebo
- Mavacamtenn = 23
- Placebon = 21reference
- Primary endpoint
- Change from baseline in Valsalva-provoked LV outflow tract pressure gradient
- Follow-up
- Week 28
Primary result
Change in Valsalva LV outflow tract gradient
Difference
−48.0 mm Hg
95% CI −67.7 to −28.3
P < 0.001
By group
Mavacamten
−48.5
least-squares mean change · mm Hg
Placebo
−0.5
least-squares mean change · mm Hg
Mavacamten vs Placebo
difference −48.0 mm Hg (95% CI −67.7 to −28.3); P < 0.001
As reported in the abstract
Further findings
Deaths
- No deaths during the trial
Safety
events
| Outcome | Mavacamten | Placebo |
|---|---|---|
| Serious adverse events | 2 | 2 |
- Serious adverse events: Mavacamten: 1 patient with two episodes of syncope, 1 with inappropriate ICD shock
- Serious adverse events: Placebo: 1 patient with chest pain, 1 with depression with suicidal ideation
Adverse events
- Incidence of adverse events similar in the two groups
LVEF reduction to <50%
- No patient had a reduction in LVEF to <50%
Original publication
Mavacamten in Adolescents with Obstructive Hypertrophic Cardiomyopathy. N Engl J Med. Published 29 March 2026. doi:10.1056/nejmoa2601103
Summarised from the published abstract; every number is checked against it