Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China

NCT05876351 | Completed Phase 3 Results

• 3 other identifiers: D7413C00001ECU-aHUS-3022025-000162-29
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 6

Brief Summary

This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with atypical hemolytic uremic syndrome (aHUS) in China

Conditions

Atypical Hemolytic Uremic

Keywords

atypical hemolytic uremic; aHUS

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response

  The criteria for complete TMA response were: 1. Normalization of platelet count (defined as platelet count ≥ 150000/microliter (ul). 2. Normalization of lactate dehydrogenase (LDH, defined as LDH ≤ upper limit of normal \[ULN\]). 3. ≥ 25% improvement in serum creatinine from baseline.

  Up to Week 26

Secondary Outcomes (12)

• Number of Participants With an Adverse Event (AE)

  Up to Week 34

• Mean Serum Concentration of Eculizumab

  Pre-dose and post-dose at Days 1, 8, 29, 85, and 141; Pre-dose at Day 183

• Change From Baseline in Serum Free Complement 5 (C5)

  Baseline (Day 1 pre-dose) to Days 1, 8, 29, 85 and 141 (pre-dose and post-dose) and pre-dose at Day 183

• Change From Baseline in Serum Total C5

  Baseline (Day 1 pre-dose) to Days 1, 8, 29, 85 and 141 (pre-dose and post-dose) and pre-dose at Day 183

• Number of Participants With an Anti-drug Antibody (ADA) Response

  Up to Week 26

Study Arms (1)

Eculizumab

EXPERIMENTAL

Participants will receive Eculizumab in a single dose vial.

Drug: Eculizumab

Interventions

Eculizumab DRUG

Weight-based doses of Eculizumab will be administered intravenously as an induction dose followed by maintenance dose at Day 8, 15, or 29 depending on weight; then every 2 or 3 weeks, depending upon weight.

Eculizumab

Eligibility Criteria

• Age: 0 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Any age weighing ≥ 5 kg
• Complement treatment naïve with evidence of TMA.
• History of aHUS prior to kidney transplant,or persistent evidence of TMA at least 4 days after modifying the immunosuppressive regimen.
• Among participants with onset of TMA postpartum, persistent evidence of TMA for \> 3 days after the day of childbirth
• All participants must be vaccinated against N meningitidis if not already vaccinated within the time period of active coverage specified by the vaccine manufacturer.
• Participants \< 18 years of age must have been vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae according to local vaccination schedule guidelines.
• In participants receiving treatment with medications known to cause TMA, persistent evidence of TMA at least 4 days after modifying the excluded medication

You may not qualify if:

• Known familial or acquired ADAMTS13deficiency (activity \< 5%).
• ST-HUS as demonstrated by local guidelines.
• Positive direct Coombs test which is indicative of a clinically significant immune-mediated hemolysis not due to aHUS.
• HIV infection, and /or unresolved meningococcal disease
• Ongoing sepsis, and / or presence or suspicion of active and untreated systemic infection
• Organ transplantation history, and/or Bone marrow transplant/hematopoietic stem cell transplant within 6 months prior to the start of Screening.
• Among participants with a kidney transplant, acute kidney dysfunction within 4 weeks of transplant consistent with the diagnosis of acute antibody-mediated rejection.
• Among participants without a kidney transplant, history of kidney disease other than aHUS
• Identified drug exposure-related HUS, and / or HUS related to vitamin B12 deficiency and / or known genetic defects of cobalamin C metabolism.
• History of malignancy within 5 years of Screening.
• Known systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid antibody positivity or syndrome.
• Chronic dialysis.
• Prior use of complement inhibitors.
• Use of tranexamic acid within 7 days prior to the start of Screening.
• Other immunosuppressive therapies.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead
• AstraZeneca: collaborator

Study Sites (6)

Research Site

Beijing, 100034, China

Location

Research Site

Beijing, 100045, China

Location

Research Site

Changsha, 410007, China

Location

Research Site

Qingdao, 110016, China

Location

Research Site

Taiyuan, 030012, China

Location

Research Site

Wuhan, 430030, China

Location

Related Links

• Related Info

MeSH Terms

Interventions

eculizumab

Results Point of Contact

• Title: Alexion Pharmaceuticals Inc.
• Organization: European Clinical Trial Information

clinicaltrials@alexion.com

+1.855.752.2356

Publication Agreements

• PI is Sponsor Employee: No
• 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

Model Details: open-label study

Study Record Dates

• First Submitted: May 17, 2023
• First Posted: May 25, 2023
• Study Start: July 14, 2023
• Primary Completion: May 7, 2025
• Study Completion: May 7, 2025
• Last Updated: December 8, 2025
• Results First Posted: December 8, 2025

Record last verified: 2025-12

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.

Locations

CN (6)