Paroxysmal Nocturnal Hemoglobinuria (PNH)
Conditions
Brief summary
This is a multicenter, randomized, open-label phase 2 study. Adult Patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HSK39297 for 24 weeks.
Interventions
DRUGHSK39297
HSK39297 tablets 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 No maximum
Healthy volunteers
No
Inclusion criteria
1. Male and female participants ≥ 18 years of age; 2. Diagnosis of PNH based on flow cytometry with clone size \> 10% by granulocytes; 3. Have not received complement inhibitor treatment; 4. Blood lactate dehydrogenase(LDH) values \> 1.5 ×upper limit of the normal range (ULN) ; 5. Hemoglobin level \< 100 g/L during the screening period.
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 with increase in hemoglobin levels from baseline of ≥20 g/L in the absence of red blood cell transfusions | Baseline, 24 weeks |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in hemoglobin | Baseline, 24 weeks |
| Change from baseline in reticulocyte count | Baseline, 24 weeks |
| Change from baseline in LDH | Baseline, 24 weeks |
| Change from baseline in Indirect bilirubin | Baseline, 24 weeks |
| Change from baseline in free hemoglobin | Baseline, 24 weeks |
| Proportion of participants with at least 60% reduction in LDH compared to baseline or LDH below the upper limit of normal | Baseline, 24 weeks |
| Change in the average number of RBC transfused per week | From week 4 to week 24 |
| Change from baseline in PNH RBC clone size | Baseline, 24 weeks |
| Change from baseline in C3 fragment deposition on PNH RBC | Baseline, 24 weeks |
| Change from baseline in FACIT-Fatigue score | Baseline, 24 weeks |
| Incidence and severity of adverse events | 28 weeks |
| Proportion of participants without requiring red blood cells (RBC) transfusions | From week 4 to week 24 |
Countries
China
Outcome results
None listed