Paroxysmal Nocturnal Hemoglobinuria
Conditions
Brief summary
A study of the efficacy and safety of HRS-5965 capsules compared to eculizumab for 24 weeks in patients with PNH.
Interventions
DRUGHRS-5965 capsule
HRS-5965 capsule for 24 weeks
Eculizumab Injection for 24 weeks
Sponsors
Chengdu Suncadia Medicine Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| Proportion of subjects who absence of administration of red blood cell transfusions | 24 weeks |
| Mean change from baseline in hemoglobin | 24 weeks |
| 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 |
| Mean change from baseline in reticulocyte counts | 24 weeks |
| Mean change from baseline in FACIT-Fatigue scores | 24 weeks |
| Mean percent change from baseline in LDH levels | 24 weeks |
Countries
China
Outcome results
None listed