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Completed

NCT Number: NCT07052838

Efficacy and Safety of HSK39297 in Anti-C5 Treated PNH Patients With Anemia

The purpose of this study is to evaluate the efficacy of HSK39297 tablets in paroxysmal nocturnal hemoglobinuria (PNH) patients with anemia after stable treatment of anti-C5 antibody.

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Key information

Conditions

Paroxysmal Nocturnal Haemoglobinuria (PNH) Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

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

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥ 18 and ≤ 75 years, Male and female patients.
  • Diagnosis of PNH based on flow cytometry showing >10% granulocyte clone size during the screening period.
  • Stable use of Anti-C5 antibody at least 6 months prior to enrollment.
  • Hemoglobin level < 10 g/dL at screening.

Exclusion criteria

  • 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 < 100x109/L, or platelets < 30x109/L or neutrophils < 0.5x109/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.

Treatment and study plan

HSK39297 tablets

Drug

200mg QD

Primary outcomes

  1. Proportion of participants achieving hemoglobin levels ≥ 120 g/L at least on three out of four measurements in the absence of red blood cell transfusions

    Time frame: 18 to 24 weeks

Secondary outcomes

  1. Proportion of participants with increase in hemoglobin levels from baseline of ≥20 g/L at least on three out of four measurements in the absence of red blood cell transfusions

    Time frame: 18 to 24 weeks

  2. Percentage of patients who did not receive a blood transfusion.

    Time frame: 18 to 24 weeks

  3. Change from baseline in hemoglobin

    Time frame: Baseline, week 18 to 24

  4. Change From Baseline in Reticulocyte Count

    Time frame: Baseline, week 18 to 24

  5. Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue score

    Time frame: Baseline, week 18 to 24

    The FACIT-F is a 13-item questionnaire with a 0-52 range. A higher total score indicates less fatigue and better function.

  6. Percent change from baseline in lactate dehydrogenase (LDH)

    Time frame: Baseline, week 18 to 24

  7. Rate of breakthrough hemolysis (BTH)

    Time frame: 24 weeks

  8. Proportion of participants with Major Adverse Vascular Events (MAVEs)

    Time frame: 24 weeks

Sponsors and collaborators

Lead sponsor

Haisco Pharmaceutical Group Co., Ltd.

Industry

Registry information

Official study title

A Multicenter, Single-Arm, Open-Label Phase III Clinical Study Evaluating the Efficacy and Safety of HSK39297 Tablets in Paroxysmal Nocturnal Hemoglobinuria Patients With Anemia Despite Stable Anti-C5 Antibody Therapy

Important dates

Study start
2025
Primary completion
2025
Study completion
2026
First posted
Jul 7, 2025
Registry last updated
Feb 11, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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