Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
Soliris
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
2 other identifiers
interventional
25
1 country
7
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jul 2023
7 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 24, 2023
CompletedFirst Posted
Study publicly available on registry
June 2, 2023
CompletedStudy Start
First participant enrolled
July 5, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 14, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
April 14, 2025
CompletedResults Posted
Study results publicly available
June 1, 2026
CompletedJune 1, 2026
May 1, 2026
1.8 years
May 24, 2023
March 26, 2026
May 6, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage Change From Baseline in LDH at Week 12
Blood samples were collected for measurement of serum LDH at specified timepoints. Statistical analysis was performed using a mixed model for repeated measures (MMRM), including the percentage change from baseline of LDH at the scheduled visits from Week 1 to Week 12 as the dependent variable, with the fixed categorical effect of visit, fixed continuous effect of LDH baseline value as covariates, and participant as random effect. Assessed at baseline, 1, 2, 3, 4, 6, 8, 10, and 12 weeks, Week 12 reported. A decrease indicated a reduction in disease activity.
Baseline, Week 12
Secondary Outcomes (10)
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Baseline through Week 64
Serum Concentration of Eculizumab
Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449
Change From Baseline in Serum Free Complement 5 (C5) Concentration
Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449
Change From Baseline in Serum Total C5 Concentration
Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449
Number of Participants With Treatment-emergent Antidrug Antibodies (ADAs) to Eculizumab
Baseline through Week 64
- +5 more secondary outcomes
Study Arms (1)
Eculizumab
EXPERIMENTALEculizumab will be administered by IV infusion.
Interventions
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.
Eligibility Criteria
You may qualify if:
- Adult C5 inhibitor naïve PNH patients (age\>=18), which is confirmed by flow cytometry evaluation
- Must be vaccinated against N meningitidis
You may not qualify if:
- Meningitidis infection or unresolved meningococcal disease
- Significant bone marrow failure
- Other significant systemic diseases that might have impact on efficacy and safety assessment
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Alexion Pharmaceuticals, Inc.lead
- AstraZenecacollaborator
Study Sites (7)
Research Site
Beijing, CN-100730, China
Research Site
Hangzhou, 310003, China
Research Site
Nantong, 226001, China
Research Site
Shanghai, 200040, China
Research Site
Tianjin, 300020, China
Research Site
Tianjin, 300050, China
Research Site
Wuhan, 430022, China
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Alexion Pharmaceuticals, Inc.
- Organization
- Alexion Pharmaceuticals, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 24, 2023
First Posted
June 2, 2023
Study Start
July 5, 2023
Primary Completion
April 14, 2025
Study Completion
April 14, 2025
Last Updated
June 1, 2026
Results First Posted
June 1, 2026
Record last verified: 2026-05
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP
- 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.
- 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.
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.