A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）

NCT ID: NCT06561841

Last Updated: 2025-06-29

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 47 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-07-17
• Study Completion Date: 2025-04-02

Brief Summary

This is a multicenter, randomized, open-label phase 2 study. Adult Patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HSK39297 for 24 weeks.

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment group A

Group Type: EXPERIMENTAL

HSK39297

Intervention Type: DRUG

HSK39297 tablets for 24 weeks

Treatment group B

Group Type: EXPERIMENTAL

HSK39297

Intervention Type: DRUG

HSK39297 tablets for 24 weeks

Treatment group C

Group Type: EXPERIMENTAL

HSK39297

Intervention Type: DRUG

HSK39297 tablets for 24 weeks

Interventions

HSK39297

HSK39297 tablets for 24 weeks

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Male and female participants ≥ 18 years of age;

2. Diagnosis of PNH based on flow cytometry with clone size > 10% by granulocytes;

3. Have not received complement inhibitor treatment;

4. Blood lactate dehydrogenase(LDH) values > 1.5 ×upper limit of the normal range (ULN) ;

5. Hemoglobin level < 100 g/L during the screening period.

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Haisco Pharmaceutical Group Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

The First Affiliated Hospital of Nanjing Medical University

Nanjing, Jiangsu, China

Countries

China

Other Identifiers

HSK39297-201

Identifier Type: -

Identifier Source: org_study_id