Heart failure & cardiomyopathies

Myeloperoxidase inhibition with mitiperstat in heart failure with preserved or mildly reduced ejection fraction: a randomized phase 2b trial

Nat MedPublished Randomised controlled trial

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
Methodological detailsmulticenter, double-blind, placebo-controlled, three-arm, parallel-group, phase 2b
  • Multicenter
  • Double-blind
  • Placebo-controlled
  • Three-arm
  • Parallel-group
  • Phase 2b

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 = 0.29
Mitiperstat (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 = 0.28

Secondary outcomes

Natriuretic peptides, inflammatory markers (up to 48 weeks), echocardiographic parameters (up to 24 weeks)

No improvement in any secondary endpoint

Safety

Maculopapular rash — Placebo
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

Composed by AI from the published abstract and checked against it. How Briefs are made →

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