Efficacy and Safety of HSK392975 in Anti-C5 Treated PNH Patients With Anemia

Not Applicable

Recruiting

• Conditions: Paroxysmal Nocturnal Haemoglobinuria (PNH)
• Interventions: Drug: HSK39297 tablets

• Registration Number: NCT07052838
• Lead Sponsor: Haisco Pharmaceutical Group Co., Ltd.

Brief Summary

The purpose of this study is to evaluate the efficacy of HSK39297 tablets in paroxysmal nocturnal hemoglobinuria patients with anemia after stable treatment of anti-C5 antibody.

Detailed Description

Not available

Study Timeline

• Study Start: 2025-05-12
• Status Verified: 2025-06
• Study First Submitted: 2025-06-29
• Study First Submitted QC: 2025-06-29
• Last Update Submitted: 2025-06-29
• Study First Posted: 2025-07-07
• Last Update Posted: 2025-07-07
• Primary Completion: 2025-12-25
• Study Completion: 2026-01-08

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 32

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
HSK39297	HSK39297 tablets	-

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	18 to 24 weeks

Secondary Outcome Measures

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

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