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

• Status: Active, not recruiting
• Trial end date: 2024-10-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Chengdu Suncadia Medicine Co., Ltd.

Criteria

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.