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
A Multicenter, Randomized, Open-label, Active-comparator Controlled Phase III Study to Evaluate the Efficacy and Safety of HRS-5965 Capsule in Patients With Paroxysmal Nocturnal Hemoglobinuria
1 other identifier
interventional
76
1 country
2
Brief Summary
A study of the efficacy and safety of HRS-5965 capsules compared to eculizumab for 24 weeks in patients with PNH.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Oct 2024
Shorter than P25 for phase_3
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 10, 2024
CompletedFirst Posted
Study publicly available on registry
September 19, 2024
CompletedStudy Start
First participant enrolled
October 25, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 31, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
July 31, 2025
CompletedSeptember 11, 2025
September 1, 2025
9 months
September 10, 2024
September 10, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
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 Outcomes (6)
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
- +1 more secondary outcomes
Study Arms (2)
HRS-5965 capsule
EXPERIMENTALEculizumab Injection
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- Diagnosis of PNH confirmed by flow cytometry with clone size \> 10%.
- Have not received complement inhibitor therapy;
- LDH \> 1.5\*ULN at screening.
- Hemoglobin level \< 10 g/dL at screening.
You may not qualify if:
- Known or suspected hereditary or acquired complement deficiency;
- Patients with laboratory evidence of bone marrow failure (reticulocytes \<100x109/L; platelets \<30x109/L; neutrophils \<0.5x109/L);
- 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;
- History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)
- Positive of HIV, HBsAg or HCVAb.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, 100730, China
The Blood Disease Hospital of the Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020, China
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 10, 2024
First Posted
September 19, 2024
Study Start
October 25, 2024
Primary Completion
July 31, 2025
Study Completion
July 31, 2025
Last Updated
September 11, 2025
Record last verified: 2025-09