Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: HSK39297 tablets.
- Who it may be relevant to
- Registry conditions: Paroxysmal Nocturnal Hemoglobinuria. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Multicenter, Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
Overview
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
- Drug HSK39297 tablets
HSK39297 tablets until the end of treatment
Primary outcome measures
- Incidence and severity of adverse events [Time frame: through study completion，about 2 years]
Secondary outcome measures (5)
- Proportion of participants achieving hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions [Time frame: through study completion，about 2 years]
- Change From Baseline in Hemoglobin in the absence of red blood cell transfusions [Time frame: through study completion，about 2 years]
- Proportion of participants who remain free from transfusions [Time frame: through study completion，about 2 years]
- Rate of breakthrough hemolysis (BTH) [Time frame: through study completion，about 2 years]
- Proportion of participants with Major Adverse Vascular Events (MAVEs) [Time frame: through study completion，about 2 years]
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- The First Affiliated Hospital of Nanjing Medical University — Nanjing
Identifiers
NCT: NCT06745622 · HSK39297-203