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

A Multicenter, Randomized, Open-label Phase 2 Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）

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.

Study Overview

• Status: Completed
• Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Intervention / Treatment: Drug: HSK39297

• Study Type: Interventional
• Enrollment (Actual): 47
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Jiangsu
    • Nanjing, Jiangsu, China, 210029
      • The First Affiliated Hospital of Nanjing Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment group A	Drug: HSK39297; HSK39297 tablets for 24 weeks
Experimental: Treatment group B	Drug: HSK39297; HSK39297 tablets for 24 weeks
Experimental: Treatment group C	Drug: HSK39297; HSK39297 tablets for 24 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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

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

Collaborators and Investigators

• Sponsor: Haisco Pharmaceutical Group Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): July 17, 2024
• Primary Completion (Actual): April 2, 2025
• Study Completion (Actual): April 2, 2025

Study Registration Dates

• First Submitted: August 16, 2024
• First Submitted That Met QC Criteria: August 16, 2024
• First Posted (Actual): August 20, 2024

Study Record Updates

• Last Update Posted (Actual): June 29, 2025
• Last Update Submitted That Met QC Criteria: June 25, 2025
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urination Disorders; Urological Manifestations; Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Proteinuria; Hemoglobinuria; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: HSK39297-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No