A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy

Phase 3

Active, not recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Interventions: Drug: Eculizumab Injection; Drug: HRS-5965 capsule

• Registration Number: NCT06593938
• Lead Sponsor: Chengdu Suncadia Medicine Co., Ltd.

Brief Summary

A study of the efficacy and safety of HRS-5965 capsules compared to eculizumab for 24 weeks in patients with PNH.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-09-10
• Study First Submitted QC: 2024-09-10
• Study First Posted: 2024-09-19
• Study Start: 2024-10-25
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-24
• Last Update Posted: 2025-02-26
• Primary Completion: 2025-12-30
• Study Completion: 2025-12-30

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 76

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 at screening.
4. Hemoglobin level < 10 g/dL at screening.

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Eculizumab Injection	Eculizumab Injection	-
HRS-5965 capsule	HRS-5965 capsule	-

Primary Outcome Measures

Name	Time	Method
Proportion of subjects with the hemoglobin levels are ≥ 12 g/dL at least on three out of four measurements in the absence of red blood cell transfusions	24 weeks

Secondary Outcome Measures

Name	Time	Method
Proportion of subjects with the change from baseline in hemoglobin is ≥ 2 g/dL at least on three out of four measurements in the absence of red blood cell transfusions	24 weeks
Proportion of subjects who absence of administration of red blood cell transfusions	24 weeks
Mean change from baseline in hemoglobin	24 weeks
Mean percent change from baseline in LDH levels	24 weeks
Mean change from baseline in reticulocyte counts	24 weeks
Mean change from baseline in FACIT-Fatigue scores	24 weeks

Trial Locations

Locations (2)

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences; 🇨🇳 Beijing, Beijing, China

The Blood Disease Hospital of the Chinese Academy of Medical Sciences; 🇨🇳 Tianjin, Tianjin, China