A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）

NCT06561841 | Completed Phase 2

• 1 other identifier: HSK39297-201
• Study Type: interventional
• Target: 47
• Locations: 1 country
• Sites: 1

Brief Summary

This is a multicenter, randomized, open-label phase 2 study. Adult Patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HSK39297 for 24 weeks.

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Outcome Measures

Primary Outcomes (1)

• Proportion of participants with increase in hemoglobin levels from baseline of ≥20 g/L in the absence of red blood cell transfusions

  Baseline, 24 weeks

Secondary Outcomes (12)

• Proportion of participants with at least 60% reduction in LDH compared to baseline or LDH below the upper limit of normal

  Baseline, 24 weeks

• Change from baseline in hemoglobin

  Baseline, 24 weeks

• Change from baseline in reticulocyte count

  Baseline, 24 weeks

• Change from baseline in LDH

  Baseline, 24 weeks

• Change from baseline in Indirect bilirubin

  Baseline, 24 weeks

Study Arms (3)

Treatment group A

EXPERIMENTAL

Drug: HSK39297

Treatment group B

EXPERIMENTAL

Drug: HSK39297

Treatment group C

EXPERIMENTAL

Drug: HSK39297

Interventions

HSK39297 DRUG

HSK39297 tablets for 24 weeks

Treatment group A; Treatment group B; Treatment group C

Eligibility Criteria

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

You may qualify if:

• Male and female participants ≥ 18 years of age;
• Diagnosis of PNH based on flow cytometry with clone size \> 10% by granulocytes;
• Have not received complement inhibitor treatment;
• Blood lactate dehydrogenase(LDH) values \> 1.5 ×upper limit of the normal range (ULN) ;
• Hemoglobin level \< 100 g/L during the screening period.

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;
• Patients with laboratory evidence of bone marrow failure (reticulocytes \< 100x10\^9/L, or platelets \< 30x10\^9/L or neutrophils \< 0.5x10\^9/L) ;
• Active systemic infection within 2 weeks prior to study drug administration;
• 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, 210029, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

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

Study Design

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

Study Record Dates

• First Submitted: August 16, 2024
• First Posted: August 20, 2024
• Study Start: July 17, 2024
• Primary Completion: April 2, 2025
• Study Completion: April 2, 2025
• Last Updated: June 29, 2025

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)