What type of study is this?

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

December 8, 2025BloodOpen Access

Efficacy and safety of HSK39297 monotherapy in patients with paroxysmal nocturnal hemoglobinuria (PNH)

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Multicenter study shows HSK39297 improves hemoglobin and reduces hemolysis in PNH patients, indicating favorable pharmacodynamics and safety.

To evaluate the efficacy and safety of HSK39297 monotherapy in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Open-label, multicenter study
47 adults with complement inhibitor-naïve PNH randomized to receive HSK39297 at various doses
Evaluated efficacy based on hemoglobin increase and safety across 24 weeks
89.4% met the primary endpoint of hemoglobin increase of ≥20 g/L
Mean hemoglobin increased by 50.7 g/L
100% reduction or normalization of LDH levels
95.7% of patients avoided transfusions
Adverse events occurred in 72.3%, mostly grade ≤2, with headache being the most common.

Gong et al. (2025) studied this question.

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December 8, 2025BloodOpen Access

View Full Paper

Ask AI

Bookmark

Authors

Multicenter study shows HSK39297 improves hemoglobin and reduces hemolysis in PNH patients, indicating favorable pharmacodynamics and safety.

To evaluate the efficacy and safety of HSK39297 monotherapy in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Open-label, multicenter study
47 adults with complement inhibitor-naïve PNH randomized to receive HSK39297 at various doses
Evaluated efficacy based on hemoglobin increase and safety across 24 weeks
89.4% met the primary endpoint of hemoglobin increase of ≥20 g/L
Mean hemoglobin increased by 50.7 g/L
100% reduction or normalization of LDH levels
95.7% of patients avoided transfusions
Adverse events occurred in 72.3%, mostly grade ≤2, with headache being the most common.

Gong et al. (2025) studied this question.

View Full Paper

Ask AI

Bookmark