Heart failure & cardiomyopathiesRandomised controlled trial
Safety and Efficacy of Metabolic Modulation With Ninerafaxstat in Patients With Nonobstructive Hypertrophic Cardiomyopathy
- Randomised controlled trial
- placebo-controlled
- multicentre (12 centres)
Authors' conclusion
In symptomatic nHCM, novel drug therapy targeting myocardial energetics was safe and well tolerated and associated with better exercise performance and health status among those most symptomatically limited.
Study context
- Population
- Nonobstructive hypertrophic cardiomyopathy (LV outflow tract gradient <30 mm Hg, EF ≥50%, peak oxygen consumption <80% predicted)
- Participants
- 67 participants
- Comparison
- Ninerafaxstat vs placebo
- Ninerafaxstatn = 34
- Placebon = 33reference
- Primary endpoint
- Safety and tolerability
- Follow-up
- 12 weeks
Primary result
As reported in the abstract
Serious adverse events
Secondary outcomes
| Outcome | LS mean difference (95% CI) | P value | |
|---|---|---|---|
| VE/Vco2 slope (ventilatory efficiency) · baseline to 12 weeks | −2.1 (−3.6 to −0.6) | P = 0.006 | |
| −4−202Difference (linear scale) |
| Outcome | LS mean (95% CI) | P value |
|---|---|---|
| KCCQ Clinical Summary Score | 3.2 (−2.9 to 9.2) | P = 0.30 |
| KCCQ Clinical Summary Score, baseline score ≤80 (subgroup, n = 35) · post hoc | 9.4 (0.3–18.5) | P = 0.04 |
Peak VO2 · baseline to 12 weeks
- No significant difference in peak VO2
- P = 0.90
Safety
Serious adverse events
Original publication
Safety and Efficacy of Metabolic Modulation With Ninerafaxstat in Patients With Nonobstructive Hypertrophic Cardiomyopathy. J Am Coll Cardiol. Published 8 April 2024. doi:10.1016/j.jacc.2024.03.387
Summarised from the published abstract; every number is checked against it