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

NCT06238544 | Active Not Recruiting Phase 2

• 1 other identifier: HRS-5965-203
• Study Type: interventional
• Target: 132
• Locations: 1 country
• Sites: 2

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of adverse events.

  Every six months,for about 3 years

Secondary Outcomes (5)

• 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

• Proportion of participants who remain free from 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

• 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

Study Arms (1)

HRS-5965 Group

EXPERIMENTAL

Drug: HRS-5965 tablets; Drug: HRS-5965 Capsules

Interventions

HRS-5965 tablets DRUG

HRS-5965 tablets until the end of study treatment

HRS-5965 Group

HRS-5965 Capsules DRUG

HRS-5965 capsules.

HRS-5965 Group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. 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.

You may not qualify if:

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

Sponsors & Collaborators

• Chengdu Suncadia Medicine Co., Ltd.: lead

Study Sites (2)

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

Beijing, Beijing Municipality, 100730, China

Location

The Blood Disease Hospital of the Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 22, 2024
• First Posted: February 2, 2024
• Study Start: February 6, 2024
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: December 24, 2025

Record last verified: 2025-12

Locations

CN (2)