What type of study is this?

This is a Human Clinical Trial study (also classified as: Phase 2, Pre-Registered Trial).

What question did this study set out to answer?

To evaluate the long-term efficacy and safety of KP104 in complement inhibitor-naïve patients with PNH.

December 8, 2025BloodOpen Access

View Full Paper

Ask AI

Bookmark

Authors

Phase 2 trial shows sustained hemoglobin improvements and safety in PNH patients, implying KP104's potential as a novel treatment.

To evaluate the long-term efficacy and safety of KP104 in complement inhibitor-naïve patients with PNH.
Conducted a Phase 2 trial with 18 PNH patients across three dose-escalation cohorts.
Administered KP104 for two years, followed by an 8-week safety follow-up.
Evaluated hemoglobin levels and safety outcomes throughout and after treatment.
100% of patients maintained a hemoglobin increase of ≥2 g/dL from baseline after 2 years.
82.4% of patients achieved hemoglobin normalization (≥12 g/dL) by treatment's end.
No patients required red blood cell transfusions during the study, highlighting treatment efficacy.

Zhang et al. (2025) studied this question.

View Full Paper

Ask AI

Bookmark