NCT07052838CompletedPhase 3
A Multicenter, Single-Arm, Open-Label Phase III Clinical Study Evaluating the Efficacy and Safety of HSK39297 Tablets in Paroxysmal Nocturnal Hemoglobinuria Patients With Anemia Despite Stable Anti-C5 Antibody Therapy
Haisco Pharmaceutical Group Co., Ltd.2 sites in 1 country36 target enrollmentStarted: May 12, 2025Last updated:
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Enrollment
- 36
- Locations
- 2
- Primary Endpoint
- 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
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 75 Years (Adult, Older Adult)
- Sex
- All
- Accepts 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.
Arms & Interventions
HSK39297
Experimental
Intervention: HSK39297 tablets (Drug)
Outcomes
Primary Outcomes
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
Time Frame: 18 to 24 weeks
Secondary Outcomes
- 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)
- Percent change from baseline in lactate dehydrogenase (LDH)(Baseline, week 18 to 24)
- Rate of breakthrough hemolysis (BTH)(24 weeks)
- Proportion of participants with Major Adverse Vascular Events (MAVEs)(24 weeks)
Investigators
Study Sites (2)
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