Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Treatment group A
|
Drug: HRS-5965 tablets
HRS-5965 tablets for 12 weeks
|
| Experimental: Treatment group B
|
Drug: HRS-5965 tablets
HRS-5965 tablets for 12 weeks
|
Outcome Measures
Primary Outcome Measures
- Changes in hemoglobin. [12 weeks]
Secondary Outcome Measures
- Changes in LDH. [12 weeks]
- Changes in haptoglobin. [12 weeks]
- Changes in bilirubin. [12 weeks]
- Changes in reticulocyte counts. [12weeks]
- Changes in C3 complement fragment deposition. [12 week]
- Percentage of patients who did not receive a blood transfusion. [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]
Eligibility Criteria
Criteria
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.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Chengdu Suncadia Medicine Co., Ltd.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- HRS-5965-202