Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China (Soliris)
Primary Purpose
Paroxysmal Nocturnal Hemoglobinuria
Status
Recruiting
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
Eculizumab
Sponsored by
About this trial
This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring paroxysmal nocturnal hemoglobinuria, PNH
Eligibility Criteria
Inclusion Criteria: Adult C5 inhibitor naïve PNH patients (age>=18), which is confirmed by flow cytometry evaluation. Must be vaccinated against N meningitidis. Exclusion Criteria: Meningitidis infection or unresolved meningococcal disease Significant bone marrow failure Other significant systemic diseases that might have impact on efficacy and safety assessment
Sites / Locations
- Research SiteRecruiting
- Research SiteRecruiting
- Research SiteRecruiting
- Research SiteRecruiting
- Research SiteRecruiting
- Research SiteRecruiting
- Research SiteRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Eculizumab
Arm Description
Eculizumab will be administered by IV infusion.
Outcomes
Primary Outcome Measures
Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12
To assess efficacy of eculizumab in participants with PNH
Secondary Outcome Measures
Number(%) of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
To assess the safety and tolerability of eculizumab in participants with PNH
Pharmacokinetics (PK): Serum Eculizumab Concentration
To characterize the pharmacokinetics of eculizumab in participants with PNH
Change from baseline in Serum Free and total Complement 5 (C5) Concentration
To characterize the pharmacodynamics of eculizumab in participants with PNH
Number(%) of participants with Antidrug Antibodies (ADAs) to Eculizumab
To characterize the immunogenicity of eculizumab in participants with PNH
Change from baseline in functional assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 64
To assess the efficacy of eculizumab in participants with PNH
Number(%) of participants with Breakthrough Hemolysis
To assess the efficacy of eculizumab in participants with PNH
Number(%) of participants achieving LDH Normalization
To assess the efficacy of eculizumab in participants with PNH
Number(%) of participants needing Blood Transfusion
To assess the efficacy of eculizumab in participants with PNH
Number(%) of participants changes from baseline in vital signs and laboratory parameters at all scheduled visits.
To characterize the safety profile of eculizumab in participants with PNH
Full Information
NCT ID
NCT05886244
First Posted
May 24, 2023
Last Updated
October 4, 2023
Sponsor
Alexion
Collaborators
AstraZeneca
1. Study Identification
Unique Protocol Identification Number
NCT05886244
Brief Title
Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
Acronym
Soliris
Official Title
Open-Label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 5, 2023 (Actual)
Primary Completion Date
April 30, 2025 (Anticipated)
Study Completion Date
April 30, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion
Collaborators
AstraZeneca
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with paraxysmal noturnal hemoglobinuria (PNH) in China.
Detailed Description
This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with PNH in China who previously have not been treated with complement inhibitors. Approximately 25 eligible participants in China will be enrolled.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria
Keywords
paroxysmal nocturnal hemoglobinuria, PNH
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
open label
Masking
None (Open Label)
Allocation
N/A
Enrollment
25 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Eculizumab
Arm Type
Experimental
Arm Description
Eculizumab will be administered by IV infusion.
Intervention Type
Drug
Intervention Name(s)
Eculizumab
Intervention Description
Participants will receive 600 milligrams (mg) once a week on Day 1, 8, 15, and 22 followed by 900 mg every 2 weeks from Day 29 to Day 435.
Primary Outcome Measure Information:
Title
Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12
Description
To assess efficacy of eculizumab in participants with PNH
Time Frame
Baseline, Week 12
Secondary Outcome Measure Information:
Title
Number(%) of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Description
To assess the safety and tolerability of eculizumab in participants with PNH
Time Frame
Baseline through Week 64
Title
Pharmacokinetics (PK): Serum Eculizumab Concentration
Description
To characterize the pharmacokinetics of eculizumab in participants with PNH
Time Frame
Baseline through Week 64 (predose and postdose)
Title
Change from baseline in Serum Free and total Complement 5 (C5) Concentration
Description
To characterize the pharmacodynamics of eculizumab in participants with PNH
Time Frame
Baseline through week 64 (predose and postdose)
Title
Number(%) of participants with Antidrug Antibodies (ADAs) to Eculizumab
Description
To characterize the immunogenicity of eculizumab in participants with PNH
Time Frame
Baseline through Week 64
Title
Change from baseline in functional assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 64
Description
To assess the efficacy of eculizumab in participants with PNH
Time Frame
Baseline through Week 64
Title
Number(%) of participants with Breakthrough Hemolysis
Description
To assess the efficacy of eculizumab in participants with PNH
Time Frame
Baseline through Week 64
Title
Number(%) of participants achieving LDH Normalization
Description
To assess the efficacy of eculizumab in participants with PNH
Time Frame
Baseline through Week 64
Title
Number(%) of participants needing Blood Transfusion
Description
To assess the efficacy of eculizumab in participants with PNH
Time Frame
Baseline through Week 64
Title
Number(%) of participants changes from baseline in vital signs and laboratory parameters at all scheduled visits.
Description
To characterize the safety profile of eculizumab in participants with PNH
Time Frame
Baseline through Week 64
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Adult C5 inhibitor naïve PNH patients (age>=18), which is confirmed by flow cytometry evaluation.
Must be vaccinated against N meningitidis.
Exclusion Criteria:
Meningitidis infection or unresolved meningococcal disease
Significant bone marrow failure
Other significant systemic diseases that might have impact on efficacy and safety assessment
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
AstraZeneca Clinical Study Information Center
Phone
1-877-240-9479
Email
information.center@astrazeneca.com
Facility Information:
Facility Name
Research Site
City
Beijing
ZIP/Postal Code
CN-100730
Country
China
Individual Site Status
Recruiting
Facility Name
Research Site
City
Hangzhou
ZIP/Postal Code
310003
Country
China
Individual Site Status
Recruiting
Facility Name
Research Site
City
Nantong
ZIP/Postal Code
226001
Country
China
Individual Site Status
Recruiting
Facility Name
Research Site
City
Shanghai
ZIP/Postal Code
200040
Country
China
Individual Site Status
Recruiting
Facility Name
Research Site
City
Tianjian
ZIP/Postal Code
300020
Country
China
Individual Site Status
Recruiting
Facility Name
Research Site
City
Tianjin
ZIP/Postal Code
300052
Country
China
Individual Site Status
Recruiting
Facility Name
Research Site
City
Wuhan
ZIP/Postal Code
430022
Country
China
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal.
All request will be evaluated as per the AZ disclosure commitment:
https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
IPD Sharing Time Frame
AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA Pharma Data Sharing Principles. For details of our timelines, please refer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
IPD Sharing Access Criteria
When a request has been approved AstraZeneca will provide access to the deidentified individual patient-level data in an approved sponsored tool . Signed Data Sharing Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information. Additionally, all users will need to accept the terms and conditions of the SAS MSE to gain access. For additional details, please review the Disclosure Statements at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
IPD Sharing URL
https://astrazenecagroup-dt.pharmacm.com/DT/Home
Learn more about this trial
Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
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