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

Haisco Pharmaceutical Group

Status and phase: Enrolling; Phase 3

Conditions: PNH

Treatments: Drug: Eculizumab Injection; Drug: HSK39297 tablets

Study type: Interventional

Funder types: Industry

Identifiers

NCT06799546

HSK39297-301

Details and patient eligibility

About

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.

Enrollment: 66 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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66 participants in 2 patient groups

HSK39297 tablets

Experimental group

Treatment:

Drug: HSK39297 tablets

Eculizumab Injection

Active Comparator group

Treatment:

Drug: Eculizumab Injection