Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria

Phase 2

Recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Interventions: Drug: HRS-5965 tablets; Drug: HRS-5965 Capsules

• Registration Number: NCT06238544
• Lead Sponsor: Chengdu Suncadia Medicine Co., Ltd.

Brief Summary

This is a multicenter, single-arm, open-label study. Patients with Paroxysmal Nocturnal Hemoglobinuria who had previously received and completed the HRS-5965 study well included. All eligible subjects received HRS-5965 tablets or capsules until the end of treatment in this study.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-01-22
• Study First Submitted QC: 2024-01-31
• Study First Posted: 2024-02-02
• Study Start: 2024-02-06
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-22
• Last Update Posted: 2025-05-28
• Primary Completion: 2027-12
• Study Completion: 2027-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 120

Inclusion Criteria

1. Patients who have previously received and completed HRS-5965 study treatment, and are judged by the investigator to have treatment benefit and may benefit from continued treatment of HRS-5965 and patients in the control group of who received eculizumab treatment.

Exclusion Criteria

1. Known or suspected hereditary or acquired complement deficiency;
2. History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
HRS-5965 Group	HRS-5965 tablets	-
HRS-5965 Group	HRS-5965 Capsules	-

Primary Outcome Measures

Name	Time	Method
Incidence and severity of adverse events.	Every six months,for about 3 years

Secondary Outcome Measures

Name	Time	Method
Proportion of participants who remain free from transfusions was assessed every half a year during treatment..	Every six months,for about 3 years
Proportion of participants with Major Adverse Vascular Events MAVEs.	Every six months,for about 3 years
Plasma concentration of HRS-5965.	Start of Treatment to end of study，for about 3 years
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions was assessed every half a year during treatment..	Every six months ,for about 3 years
Rate of breakthrough hemolysis was assessed every half a year during treatment	Every six months,for about 3 years

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