A Multicenter, Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）

Haisco Pharmaceutical Group Co., Ltd.1 site in 1 country47 target enrollmentJanuary 3, 2025

ConditionsParoxysmal Nocturnal Hemoglobinuria

InterventionsHSK39297 tablets

DrugsHSK39297 tablets

Overview

Phase: Phase 2
Intervention: HSK39297 tablets
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Sponsor: Haisco Pharmaceutical Group Co., Ltd.
Enrollment: 47
Locations: 1
Primary Endpoint: Incidence and severity of adverse events
Status: Recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This is a multicenter, open-label study to evaluate the long-term safety, tolerability and efficacy of HSK39297. Adult patients with PNH who had previously received and completed HSK39297 study treatment will be included. Eligible subjects can maintain HSK39297 treatment until the end of the study.

Registry

clinicaltrials.gov

Start Date

January 3, 2025

End Date

June 2027

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Haisco Pharmaceutical Group Co., Ltd.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Patients With PNH who have previously received and completed HSK39297 treatment , and According to the researchers' judgment, the benefits of treatment outweigh the risks and may benefit from continued treatment with HSK39297;
•Understand the study procedures and methods, voluntarily participate in this trial.

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;
•History of serious comorbidities that have been determined to be unsuitable for participation in the study;
•Pregnant or Lactating women.

Arms & Interventions

HSK39297

Intervention: HSK39297 tablets

Outcomes

Primary Outcomes

Incidence and severity of adverse events

Time Frame: through study completion，about 2 years

Secondary Outcomes

Proportion of participants achieving hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions(through study completion，about 2 years)
Change From Baseline in Hemoglobin in the absence of red blood cell transfusions(through study completion，about 2 years)
Proportion of participants who remain free from transfusions(through study completion，about 2 years)
Rate of breakthrough hemolysis (BTH)(through study completion，about 2 years)
Proportion of participants with Major Adverse Vascular Events (MAVEs)(through study completion，about 2 years)