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

Phase 3

Recruiting

• Conditions: PNH
• Interventions: Drug: HSK39297 tablets; Drug: Eculizumab Injection

• Registration Number: NCT06799546
• Lead Sponsor: Haisco Pharmaceutical Group Co., Ltd.

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-01
• Study First Submitted: 2025-01-23
• Study First Submitted QC: 2025-01-23
• Last Update Submitted: 2025-01-23
• Study First Posted: 2025-01-29
• Last Update Posted: 2025-01-29
• Study Start: 2025-02-20
• Primary Completion: 2025-12-31
• Study Completion: 2025-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 66

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
HSK39297 tablets	HSK39297 tablets	-
Eculizumab Injection	Eculizumab Injection	-

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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
Proportion of participants who did not receive blood transfusion.	Between Week 2 and Week 24
Proportion of participants with Major Adverse Vascular Events (MAVEs)	24 weeks
Change from baseline in hemoglobin	Baseline, week 18 to 24
Rate of breakthrough hemolysis (BTH)	24 weeks
Change From Baseline in Reticulocyte Count	Baseline, week 18 to 24
Percent change from baseline in LDH	Baseline, week 18 to 24
Change from baseline in FACIT-Fatigue score	Baseline, week 18 to 24

Trial Locations

Locations (2)

The First Affiliated Hospital of Nanjing Medical University; 🇨🇳 Nanjing, Jiangsu, China

The First Affiliated Hospital, College of Medicine, Zhejiang University; 🇨🇳 Hangzhou, Zhejiang, China