Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria

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

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06745622

Enrollment

Registered

2024-12-20

Start date

2025-01-03

Completion date

2027-06-30

Last updated

2024-12-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paroxysmal Nocturnal Hemoglobinuria

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.

Interventions

DRUGHSK39297 tablets

HSK39297 tablets until the end of treatment

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

1. 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; 2. Understand the study procedures and methods, voluntarily participate in this trial.

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. History of serious comorbidities that have been determined to be unsuitable for participation in the study; 6. Pregnant or Lactating women.

Design outcomes

Primary

Measure	Time frame
Incidence and severity of adverse events	through study completion，about 2 years

Secondary

Measure	Time frame
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

Countries

China

Contacts

Primary ContactLi

lifangq@haisco.com+8602867258840

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026