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

Haisco Pharmaceutical Group

Status and phase: Enrolling; Phase 3

Conditions: Paroxysmal Nocturnal Haemoglobinuria (PNH)

Treatments: Drug: HSK39297 tablets

Study type: Interventional

Funder types: Industry

Identifiers

NCT07052838

HSK39297-302

Details and patient eligibility

About

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.

Enrollment: 32 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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32 participants in 1 patient group

HSK39297

Experimental group

Treatment:

Drug: HSK39297 tablets