Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Phase 2
Completed
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Drug: HRS-5965 tablets
- Registration Number
- NCT06051357
- Lead Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 26
Inclusion Criteria
- Diagnosis of PNH confirmed by flow cytometry with clone size ≥ 10%.
- Have not received complement inhibitor therapy ;
- LDH > 1.5×ULN;
- Hemoglobin level < 10 g/dL.
Exclusion Criteria
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Treatment group A HRS-5965 tablets - Treatment group B HRS-5965 tablets -
- Primary Outcome Measures
Name Time Method Changes in hemoglobin. 12 weeks
- Secondary Outcome Measures
Name Time Method Plasma concentration of HRS-5965. 12 weeks Changes in C3 complement fragment deposition. 12 week Incidence and severity of adverse events 16 weeks Incidence of thromboembolic events. 16 weeks Changes in LDH. 12 weeks Changes in haptoglobin. 12 weeks Changes in reticulocyte counts. 12weeks Number of RBC units of transfused. 12weeks Changes in bilirubin. 12 weeks Percentage of patients who did not receive a blood transfusion. 12 weeks
Trial Locations
- Locations (2)
The Blood Disease Hospital of the Chinese Academy of Medical Sciences
🇨🇳Tianjin, Tianjin, China
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
🇨🇳Beijing, Beijing, China