PNH
Conditions
Brief summary
The purpose of this study is to evaluate the efficacy and safety of HSK39297 tablets compared to eculizumab in Patients with PNH who Are Naive to Complement Inhibitor Therapy.
Interventions
DRUGHSK39297 tablets
200mg QD for 24 weeks
Eculizumab Injection for 24 weeks
Sponsors
Haisco Pharmaceutical Group Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
1. Age ≥ 18 and ≤ 75 years, Male and female patients; 2. Diagnosis of PNH based on flow cytometry with clone size \> 10% by granulocytes; 3. Have not received complement inhibitor treatment; 4. Blood LDH values \> 1.5 ×upper limit of the normal range (ULN) ; 5. Hemoglobin level \< 10 g/dL at screening.
Exclusion criteria
1. Hereditary or acquired complement deficiency; 2. Active primary or secondary immunodeficiency; 3. History of splenectomy, bone marrow/ hematopoietic stem cell or solid organ transplants; 4. History of recurrent invasive infections caused by encapsulated organisms( e.g. meningococcus or pneumococcus) or Mycobacterium tuberculosis; 5. Patients with laboratory evidence of bone marrow failure (reticulocytes \< 100x10\^9/L, or platelets \< 30x10\^9/L or neutrophils \< 0.5x10\^9/L) ; 6. Active systemic infection within 2 weeks prior to study drug administration; 7. History of serious comorbidities that have been determined to be unsuitable for participation in the study. 8. Pregnant or Lactating women.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of participants achieving hemoglobin levels ≥ 12 g/dL at least on three out of four measurements in the absence of red blood cell transfusions | Between Week 18 and Week 24 |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in FACIT-Fatigue score | Baseline, week 18 to 24 |
| Rate of breakthrough hemolysis (BTH) | 24 weeks |
| Proportion of participants with Major Adverse Vascular Events (MAVEs) | 24 weeks |
| Percent change from baseline in LDH | Baseline, week 18 to 24 |
| Proportion of participants who did not receive blood transfusion. | Between Week 2 and Week 24 |
| Change from baseline in hemoglobin | Baseline, week 18 to 24 |
| Change From Baseline in Reticulocyte Count | Baseline, week 18 to 24 |
| Proportion of participants with increase in hemoglobin levels from baseline of ≥20 g/L at least on three out of four measurements in the absence of red blood cell transfusions | Between Week 18 and Week 24 |
Countries
China
Outcome results
None listed