Heart failure & cardiomyopathiesRandomised controlled trial
Myeloperoxidase inhibition with mitiperstat in heart failure with preserved or mildly reduced ejection fraction: a randomized phase 2b trial
- Randomised controlled trial
- multicenter
- double-blind
- placebo-controlled
- three-arm
- parallel-group
- phase 2b
Authors' conclusion
These results indicate that mitiperstat was safe and well tolerated but did not improve symptoms or exercise function in chronic heart failure with preserved or mildly reduced ejection fraction.
Study context
- Population
- Patients with HF and EF >40%45% women
- Participants
- 711 patients
- Comparison
- Mitiperstat vs placebo
- Primary endpoint
- Co-primary: KCCQ Total Symptom Score (Kansas City Cardiomyopathy Questionnaire Total Symptom Score (KCCQ-TSS)) and 6-minute walk distance (6MWD)
- Follow-up
- 16 weeks
Primary result
As reported in the abstract
Mitiperstat (pooled doses) vs PlaceboPlacebo-corrected difference in mean change from baseline, points (95% CI)P value
KCCQ-TSS
−1.4(−3.9 to 1.2)
P = 0.29Mitiperstat (pooled doses) vs PlaceboPlacebo-corrected difference in mean change from baseline, m (95% CI)P value
6MWD
3.8(−3.1 to 10.8)
P = 0.28Secondary outcomes
Natriuretic peptides, inflammatory markers (up to 48 weeks), echocardiographic parameters (up to 24 weeks)
- No improvement in any secondary endpoint
Safety
| Outcome | Mitiperstat 2.5 mg | Mitiperstat 5 mg | Placebo |
|---|---|---|---|
| Maculopapular rash | Not reported in the abstract | Not reported in the abstract | 0.4% |
- Maculopapular rash: Mitiperstat 3.6%
Adverse and serious adverse events, including infections
- Similar among groups (except maculopapular rash)
Original publication
Myeloperoxidase inhibition with mitiperstat in heart failure with preserved or mildly reduced ejection fraction: a randomized phase 2b trial. Nat Med. Published 9 September 2026. doi:10.1038/s41591-026-04615-z
Summarised from the published abstract; every number is checked against it