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

Haisco Pharmaceutical Group

Status and phase: Completed; Phase 2

Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)

Treatments: Drug: HSK39297

Study type: Interventional

Funder types: Industry

Identifiers

NCT06561841

HSK39297-201

Details and patient eligibility

About

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.

Enrollment: 47 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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47 participants in 3 patient groups

Treatment group A

Experimental group

Treatment:

Drug: HSK39297

Treatment group B

Experimental group

Treatment:

Drug: HSK39297

Treatment group C

Experimental group

Treatment:

Drug: HSK39297