Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria

An Multicenter, Randomized, Open-label Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is a multicenter, randomized, open-label phase II clinical trial. A total of 24 patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HRS-5965 for 12 weeks.

Inclusion Criteria:

1. Diagnosis of PNH confirmed by flow cytometry with clone size ≥ 10%.
2. Have not received complement inhibitor therapy ;
3. LDH > 1.5×ULN;
4. Hemoglobin level < 10 g/dL.

Exclusion Criteria:

1. Known or suspected hereditary or acquired complement deficiency;
2. Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L);
3. Presence or suspicion of a systemic active bacterial, viral, or fungal infection (based on judgment of the investigator) within 2 weeks prior to the first dose of HRS-5965;
4. History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)
5. Positive of HIV, HBsAg or HCVAb.