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

Phase 2

Recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Interventions: Drug: HSK39297 tablets

• Registration Number: NCT06745622
• Lead Sponsor: Haisco Pharmaceutical Group Co., Ltd.

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-12
• Study First Submitted: 2024-12-12
• Study First Submitted QC: 2024-12-17
• Last Update Submitted: 2024-12-17
• Study First Posted: 2024-12-20
• Last Update Posted: 2024-12-20
• Study Start: 2025-01-03
• Primary Completion: 2027-06
• Study Completion: 2027-06

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 47

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
HSK39297	HSK39297 tablets	-

Primary Outcome Measures

Name	Time	Method
Incidence and severity of adverse events	through study completion，about 2 years

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

The First Affiliated Hospital of Nanjing Medical University; 🇨🇳 Nanjing, Jiangsu, China