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

NCT06799546 | Completed Phase 3

• 1 other identifier: HSK39297-301
• Study Type: interventional
• Target: 73
• Locations: 1 country
• Sites: 2

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.

Conditions

PNH

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (8)

• 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

• Change from baseline in hemoglobin

  Baseline, week 18 to 24

• Change From Baseline in Reticulocyte Count

  Baseline, week 18 to 24

• Percent change from baseline in LDH

  Baseline, week 18 to 24

Study Arms (2)

HSK39297 tablets

EXPERIMENTAL

Drug: HSK39297 tablets

Eculizumab Injection

ACTIVE COMPARATOR

Drug: Eculizumab Injection

Interventions

HSK39297 tablets DRUG

200mg QD for 24 weeks

HSK39297 tablets

Eculizumab Injection DRUG

Eculizumab Injection for 24 weeks

Eculizumab Injection

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 and ≤ 75 years, Male and female patients;
• Diagnosis of PNH based on flow cytometry with clone size \> 10% by granulocytes;
• Have not received complement inhibitor treatment;
• Blood LDH values \> 1.5 ×upper limit of the normal range (ULN) ;
• Hemoglobin level \< 10 g/dL at screening.

You may not qualify if:

• 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 \< 100x10\^9/L, or platelets \< 30x10\^9/L or neutrophils \< 0.5x10\^9/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.

Sponsors & Collaborators

• Haisco Pharmaceutical Group Co., Ltd.: lead

Study Sites (2)

The First Affiliated Hospital of Nanjing Medical University

Nanjing, Jiangsu, 210029, China

Location

The First Affiliated Hospital, College of Medicine, Zhejiang University

Hangzhou, Zhejiang, 310003, China

Location

MeSH Terms

Interventions

eculizumab

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2025
• First Posted: January 29, 2025
• Study Start: February 13, 2025
• Primary Completion: November 5, 2025
• Study Completion: November 5, 2025
• Last Updated: December 22, 2025

Record last verified: 2025-12

Locations

CN (2)