Back to resultsProof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Primary Purpose
Paroxysmal Nocturnal Hemoglobinuria
Status
Not yet recruiting
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
HRS-5965 tablets
Sponsored by
About this trial
This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria
Eligibility Criteria
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.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Treatment group A
Treatment group B
Arm Description
Outcomes
Primary Outcome Measures
Changes in hemoglobin.
Secondary Outcome Measures
Changes in LDH.
Changes in haptoglobin.
Changes in bilirubin.
Changes in reticulocyte counts.
Changes in C3 complement fragment deposition.
Percentage of patients who did not receive a blood transfusion.
Number of RBC units of transfused.
Incidence and severity of adverse events
Incidence of thromboembolic events.
Plasma concentration of HRS-5965.
Full Information
NCT ID
NCT06051357
First Posted
September 5, 2023
Last Updated
September 20, 2023
Sponsor
Chengdu Suncadia Medicine Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT06051357
Brief Title
Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Official 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)
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
September 2023 (Anticipated)
Primary Completion Date
October 2024 (Anticipated)
Study Completion Date
October 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Chengdu Suncadia Medicine Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
24 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Treatment group A
Arm Type
Experimental
Arm Title
Treatment group B
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
HRS-5965 tablets
Intervention Description
HRS-5965 tablets for 12 weeks
Primary Outcome Measure Information:
Title
Changes in hemoglobin.
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Changes in LDH.
Time Frame
12 weeks
Title
Changes in haptoglobin.
Time Frame
12 weeks
Title
Changes in bilirubin.
Time Frame
12 weeks
Title
Changes in reticulocyte counts.
Time Frame
12weeks
Title
Changes in C3 complement fragment deposition.
Time Frame
12 week
Title
Percentage of patients who did not receive a blood transfusion.
Time Frame
12 weeks
Title
Number of RBC units of transfused.
Time Frame
12weeks
Title
Incidence and severity of adverse events
Time Frame
16 weeks
Title
Incidence of thromboembolic events.
Time Frame
16 weeks
Title
Plasma concentration of HRS-5965.
Time Frame
12 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Luyao Dong
Phone
0518-82342973
Email
luyao.dong@hengrui.com
First Name & Middle Initial & Last Name or Official Title & Degree
Bin Hu
Phone
+86 18210289829
Email
bin.hu5@hengrui.com
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
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