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

October 10, 2024 updated by: Chengdu Suncadia Medicine Co., Ltd.

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

26

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Beijing
      • Beijing, Beijing, China, 100730
        • Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
    • Tianjin
      • Tianjin, Tianjin, China, 300020
        • The Blood Disease Hospital of the Chinese Academy of Medical Sciences

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

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

Exclusion Criteria:

  1. Known or suspected hereditary or acquired complement deficiency;
  2. Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L);
  3. 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;
  4. History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)
  5. Positive of HIV, HBsAg or HCVAb.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Treatment group A
Drug: HRS-5965 tablets
HRS-5965 tablets for 12 weeks
Experimental: Treatment group B
Drug: HRS-5965 tablets
HRS-5965 tablets for 12 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Changes in hemoglobin.
Time Frame: 12 weeks
12 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
Incidence and severity of adverse events
Time Frame: 16 weeks
16 weeks
Changes in LDH.
Time Frame: 12 weeks
12 weeks
Changes in haptoglobin.
Time Frame: 12 weeks
12 weeks
Changes in bilirubin.
Time Frame: 12 weeks
12 weeks
Changes in reticulocyte counts.
Time Frame: 12weeks
12weeks
Changes in C3 complement fragment deposition.
Time Frame: 12 week
12 week
Percentage of patients who did not receive a blood transfusion.
Time Frame: 12 weeks
12 weeks
Number of RBC units of transfused.
Time Frame: 12weeks
12weeks
Incidence of thromboembolic events.
Time Frame: 16 weeks
16 weeks
Plasma concentration of HRS-5965.
Time Frame: 12 weeks
12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chengdu Suncadia Medicine Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 15, 2023

Primary Completion (Actual)

March 25, 2024

Study Completion (Actual)

March 25, 2024

Study Registration Dates

First Submitted

September 5, 2023

First Submitted That Met QC Criteria

September 20, 2023

First Posted (Actual)

September 22, 2023

Study Record Updates

Last Update Posted (Actual)

October 15, 2024

Last Update Submitted That Met QC Criteria

October 10, 2024

Last Verified

October 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HRS-5965-202

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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