Paroxysmal Nocturnal Haemoglobinuria (PNH)
Conditions
Keywords
Paroxysmal Nocturnal Hemoglobinuria
Brief summary
The purpose of this study is to evaluate the efficacy of HSK39297 tablets in paroxysmal nocturnal hemoglobinuria (PNH) patients with anemia after stable treatment of anti-C5 antibody.
Interventions
DRUGHSK39297 tablets
200mg QD
Sponsors
Haisco Pharmaceutical Group Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Age ≥ 18 and ≤ 75 years, Male and female patients. * Diagnosis of PNH based on flow cytometry showing \>10% granulocyte clone size during the screening period. * Stable use of Anti-C5 antibody at least 6 months prior to enrollment. * Hemoglobin level \< 10 g/dL at screening.
Exclusion criteria
* Hereditary or acquired complement deficiency. * Active primary or secondary immunodeficiency. * History of splenectomy, bone marrow/hematopoietic stem cell or solid organ transplants. * History of recurrent invasive infections caused by encapsulated organisms( e.g. meningococcus or pneumococcus) or Mycobacterium tuberculosis. * Patients with laboratory evidence of bone marrow failure (reticulocytes \< 100x109/L, or platelets \< 30x109/L or neutrophils \< 0.5x109/L). * Active systemic infection within 2 weeks prior to study drug administration. * History of serious comorbidities that have been determined to be unsuitable for participation in the study. * Pregnant or Lactating women.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of participants achieving hemoglobin levels ≥ 120 g/L at least on three out of four measurements in the absence of red blood cell transfusions | 18 to 24 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Rate of breakthrough hemolysis (BTH) | 24 weeks | — |
| 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 | 18 to 24 weeks | — |
| Percentage of patients who did not receive a blood transfusion. | 18 to 24 weeks | — |
| Change from baseline in hemoglobin | Baseline, week 18 to 24 | — |
| Change From Baseline in Reticulocyte Count | Baseline, week 18 to 24 | — |
| Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue score | Baseline, week 18 to 24 | The FACIT-F is a 13-item questionnaire with a 0-52 range. A higher total score indicates less fatigue and better function. |
| Proportion of participants with Major Adverse Vascular Events (MAVEs) | 24 weeks | — |
| Percent change from baseline in lactate dehydrogenase (LDH) | Baseline, week 18 to 24 | — |
Countries
China
Outcome results
None listed