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

Phase 2

Recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Interventions: Drug: HSK39297

• Registration Number: NCT06561841
• Lead Sponsor: Haisco Pharmaceutical Group Co., Ltd.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2024-07-17
• Status Verified: 2024-08
• Study First Submitted: 2024-08-16
• Study First Submitted QC: 2024-08-16
• Last Update Submitted: 2024-08-16
• Study First Posted: 2024-08-20
• Last Update Posted: 2024-08-20
• Primary Completion: 2025-02-13
• Study Completion: 2025-03-13

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 45

Inclusion Criteria

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

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. Patients with laboratory evidence of bone marrow failure (reticulocytes < 100x10^9/L, or platelets < 30x10^9/L or neutrophils < 0.5x10^9/L) ;
6. Active systemic infection within 2 weeks prior to study drug administration;
7. History of serious comorbidities that have been determined to be unsuitable for participation in the study.
8. Pregnant or Lactating women.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Treatment group C	HSK39297	-
Treatment group A	HSK39297	-
Treatment group B	HSK39297	-

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Change from baseline in FACIT-Fatigue score	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 free hemoglobin	Baseline, 24 weeks
Change in the average number of RBC transfused per week	From week 4 to week 24
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 C3 fragment deposition on PNH RBC	Baseline, 24 weeks
Proportion of participants without requiring red blood cells (RBC) transfusions	From week 4 to week 24
Change from baseline in Indirect bilirubin	Baseline, 24 weeks
Change from baseline in PNH RBC clone size	Baseline, 24 weeks
Incidence and severity of adverse events	28 weeks

Trial Locations

Locations (1)

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