What type of study is this?

This is a Pre-Registered Trial study.

December 8, 2025Blood

Long-term safety and efficacy of iptacopan in patients with paroxysmal nocturnal hemoglobinuria: 4- and 5-year follow-up of patients from Phase 2 studies who entered the roll-over extension program

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Open-label study shows sustained hemoglobin levels in PNH patients using iptacopan, indicating a strong safety profile.

To evaluate the long-term safety and efficacy of iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Ongoing, open-label, single-arm, multicenter Phase 3 study
Involved PNH patients from Phase 2 studies
Long-term follow-up data assessed after 4-5 years
Mean hemoglobin levels maintained at 12.1 g/dL and 12.9 g/dL at 4 and 5 years, respectively
84.6% reported ≥1 adverse event, primarily COVID-19 and pyrexia
Minimal breakthrough hemolysis and adverse vascular events reported during follow-up

Frieri et al. (2025) studied this question.

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