Efficacy and Safety of HSK39297 in Anti-C5 Treated PNH Patients With Anemia

NCT07052838 | Completed Phase 3

• 1 other identifier: HSK39297-302
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 2

Brief Summary

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

Conditions

Paroxysmal Nocturnal Haemoglobinuria (PNH)

Keywords

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Proportion of participants achieving hemoglobin levels ≥ 120 g/L at least on three out of four measurements in the absence of red blood cell transfusions

  18 to 24 weeks

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

  18 to 24 weeks

• Percentage of patients who did not receive a blood transfusion.

  18 to 24 weeks

• Change from baseline in hemoglobin

  Baseline, week 18 to 24

• Change From Baseline in Reticulocyte Count

  Baseline, week 18 to 24

• Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue score

  Baseline, week 18 to 24

Study Arms (1)

HSK39297

EXPERIMENTAL

Drug: HSK39297 tablets

Interventions

HSK39297 tablets DRUG

200mg QD

HSK39297

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 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.

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 \< 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.

Sponsors & Collaborators

• Haisco Pharmaceutical Group Co., Ltd.: lead

Study Sites (2)

The First Affiliated Hospital of Nanjing Medical University

Nanjing, Jiangsu, China

Location

The First Affiliated Hospital, College of Medicine, Zhejiang University

Hangzhou, Zhejiang, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Design

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

Study Record Dates

• First Submitted: June 29, 2025
• First Posted: July 7, 2025
• Study Start: May 12, 2025
• Primary Completion: December 23, 2025
• Study Completion: January 5, 2026
• Last Updated: February 11, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)