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NCT Number: NCT06051357

Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria

This is a multicenter, randomized, open-label phase II clinical trial. A total of 24 patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HRS-5965 for 12 weeks.

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, Beijing Municipality, 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

  • Diagnosis of PNH confirmed by flow cytometry with clone size ≥ 10%.
  • Have not received complement inhibitor therapy ;
  • LDH > 1.5×ULN;
  • Hemoglobin level < 10 g/dL.

Exclusion criteria

  • Known or suspected hereditary or acquired complement deficiency;
  • Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L);
  • Presence or suspicion of a systemic active bacterial, viral, or fungal infection (based on judgment of the investigator) within 2 weeks prior to the first dose of HRS-5965;
  • History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)
  • Positive of HIV, HBsAg or HCVAb.

Treatment and study plan

HRS-5965 tablets

Drug

HRS-5965 tablets for 12 weeks

Primary outcomes

  1. Changes in hemoglobin.

    Time frame: 12 weeks

Secondary outcomes

  1. Changes in LDH.

    Time frame: 12 weeks

  2. Changes in haptoglobin.

    Time frame: 12 weeks

  3. Changes in bilirubin.

    Time frame: 12 weeks

  4. Changes in reticulocyte counts.

    Time frame: 12weeks

  5. Changes in C3 complement fragment deposition.

    Time frame: 12 week

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

    Time frame: 12 weeks

  7. Number of RBC units of transfused.

    Time frame: 12weeks

  8. Incidence and severity of adverse events

    Time frame: 16 weeks

  9. Incidence of thromboembolic events.

    Time frame: 16 weeks

  10. Plasma concentration of HRS-5965.

    Time frame: 12 weeks

Sponsors and collaborators

Lead sponsor

Chengdu Suncadia Medicine Co., Ltd.

Industry

Registry information

Official study title

An Multicenter, Randomized, Open-label Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Important dates

Study start
2023
Primary completion
2024
Study completion
2024
First posted
Sep 22, 2023
Registry last updated
Oct 15, 2024

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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