Clinical trial NCT06799546: A Phase III Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy
Official title: A Phase III, Multicenter, Randomized, Open Label, Active-Controlled Study to Evaluate the Efficacy and Safety of HSK39297 Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Naive to Complement Inhibitor Therapy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: PNH
Lead sponsor: Haisco Pharmaceutical Group Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2025-01-29
Enrollment: 66
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: 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
timeFrame: Between Week 18 and Week 24
Secondary outcomes:
measure: 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
timeFrame: Between Week 18 and Week 24
measure: Proportion of participants who did not receive blood transfusion.
timeFrame: Between Week 2 and Week 24
measure: Change from baseline in hemoglobin
timeFrame: Baseline, week 18 to 24
measure: Change From Baseline in Reticulocyte Count
timeFrame: Baseline, week 18 to 24
measure: Percent change from baseline in LDH
timeFrame: Baseline, week 18 to 24
measure: Change from baseline in FACIT-Fatigue score
timeFrame: Baseline, week 18 to 24
measure: Rate of breakthrough hemolysis (BTH)
timeFrame: 24 weeks
measure: Proportion of participants with Major Adverse Vascular Events (MAVEs)
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Nanjing
country: China
facility: The First Affiliated Hospital of Nanjing Medical University
state: Jiangsu
city: Hangzhou
country: China
facility: The First Affiliated Hospital, College of Medicine, Zhejiang University
state: Zhejiang
Study officials:
References: