Paroxysmal Nocturnal Hemoglobinuria
Conditions
Brief summary
This is a multicenter, randomized, open-label phase II clinical trial. A total of 24 patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HRS-5965 for 12 weeks.
Interventions
DRUGHRS-5965 tablets
HRS-5965 tablets for 12 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; 4. Hemoglobin level \< 10 g/dL.
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 |
|---|---|
| Changes in hemoglobin. | 12 weeks |
Secondary
| Measure | Time frame |
|---|---|
| Changes in haptoglobin. | 12 weeks |
| Changes in bilirubin. | 12 weeks |
| Changes in reticulocyte counts. | 12weeks |
| Changes in C3 complement fragment deposition. | 12 week |
| Changes in LDH. | 12 weeks |
| Number of RBC units of transfused. | 12weeks |
| Incidence and severity of adverse events | 16 weeks |
| Incidence of thromboembolic events. | 16 weeks |
| Plasma concentration of HRS-5965. | 12 weeks |
| Percentage of patients who did not receive a blood transfusion. | 12 weeks |
Countries
China
Outcome results
None listed