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

NCT06745622 | Recruiting Phase 2

• 1 other identifier: HSK39297-203
• Study Type: interventional
• Target: 47
• Locations: 1 country
• Sites: 1

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of adverse events

  through study completion，about 2 years

Secondary Outcomes (5)

• 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

Study Arms (1)

HSK39297

EXPERIMENTAL

Drug: HSK39297 tablets

Interventions

HSK39297 tablets DRUG

HSK39297 tablets until the end of treatment

HSK39297

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Haisco Pharmaceutical Group Co., Ltd.: lead

Study Sites (1)

The First Affiliated Hospital of Nanjing Medical University

Nanjing, Jiangsu, China

RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Central Study Contacts

Li

CONTACT

+8602867258840; lifangq@haisco.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2024
• First Posted: December 20, 2024
• Study Start: January 3, 2025
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027
• Last Updated: December 20, 2024

Record last verified: 2024-12

Locations

CN (1)