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

Prospective, Single-Arm, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment-Naïve Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China

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

Study Overview

• Status: Completed
• Conditions: Atypical Hemolytic Uremic
• Intervention / Treatment: Drug: Eculizumab

Detailed Description

This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with aHUS in China. The study will be conducted in participants of any age who weigh ≥ 5 kg and who previously have not been treated with complement inhibitors. The study consists of an up to 7-day Screening Period and a 26-week Treatment Period. An 8-week Safety Follow-up Phone Call will be required only for participants who discontinue eculizumab treatment during the study or for participants who will not receive continued access to eculizumab after completing study treatment. Approximately 25 eligible participants in China will be enrolled.

• Study Type: Interventional
• Enrollment (Actual): 25
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100034
    • Research Site
  • Beijing, China, 100045
    • Research Site
  • Changsha, China, 410007
    • Research Site
  • Qingdao, China, 110016
    • Research Site
  • Taiyuan, China, 030012
    • Research Site
  • Wuhan, China, 430030
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

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

Exclusion Criteria:

1. Known familial or acquired ADAMTS13deficiency (activity < 5%).
2. ST-HUS as demonstrated by local guidelines.
3. Positive direct Coombs test which is indicative of a clinically significant immune-mediated hemolysis not due to aHUS.
4. HIV infection, and /or unresolved meningococcal disease
5. Ongoing sepsis, and / or presence or suspicion of active and untreated systemic infection
6. Organ transplantation history, and/or Bone marrow transplant/hematopoietic stem cell transplant within 6 months prior to the start of Screening.
7. Among participants with a kidney transplant, acute kidney dysfunction within 4 weeks of transplant consistent with the diagnosis of acute antibody-mediated rejection.
8. Among participants without a kidney transplant, history of kidney disease other than aHUS
9. Identified drug exposure-related HUS, and / or HUS related to vitamin B12 deficiency and / or known genetic defects of cobalamin C metabolism.
10. History of malignancy within 5 years of Screening.
11. Known systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid antibody positivity or syndrome.
12. Chronic dialysis.
13. Prior use of complement inhibitors.
14. Use of tranexamic acid within 7 days prior to the start of Screening.
15. Other immunosuppressive therapies.
16. Receiving chronic intravenous immunoglobulin (IVIg) within 8 weeks prior to the start of Screening.
17. Received vasopressors or inotropes within 7 days prior to Screening.
18. Previously or currently treated with a complement inhibitor.
19. Has participated in another interventional treatment study or used any experimental therapy.
20. Hypersensitivity to any excipient in eculizumab.
21. Pregnant or breastfeeding.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Eculizumab; Participants will receive Eculizumab in a single dose vial.	Drug: Eculizumab; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response	The criteria for complete TMA response were: Normalization of platelet count (defined as platelet count ≥ 150000/microliter (ul).; Normalization of lactate dehydrogenase (LDH, defined as LDH ≤ upper limit of normal [ULN]).; ≥ 25% improvement in serum creatinine from baseline.	Up to Week 26

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With an Adverse Event (AE)	An AE was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which did not necessarily have a causal relationship with this treatment. A serious AE (SAE) was defined as any untoward medical occurrence that, at any dose: resulted in death,; was life-threatening,; required inpatient hospitalization or prolongation of existing hospitalization,; resulted in persistent disability/incapacity,; was a congenital anomaly/birth defect, or; was an important medical event. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Adverse Events' Section.	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	An ADA response was defined as a positive ADA sample at any time during the study.	Up to Week 26
Time to Complete TMA Response	Time to complete TMA response was defined as the time from first infusion to the first time point at which all criteria for complete TMA response was met. The criteria for complete TMA response were: Normalization of platelet count (defined as platelet count ≥ 150000/ul.; Normalization of LDH, defined as LDH ≤ ULN).; ≥ 25% improvement in serum creatinine from baseline. Participants who did not have a response were censored at the date of last visit or study discontinuation at the time when the analysis was performed.	Up to Week 26
Proportion of Participants On or Off Dialysis at Each Timepoint	Participants were considered as 'off' dialysis at a specific time point if they were dialysis free for more than 5 days prior to that time point. Participants were considered as 'on' dialysis at a specific time point if they were dialysis free to 5 days or less up prior to that time point.	Baseline and Days 22, 43, 71, 99, 113, 127, 155 and 183
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit	Expressed in milliliters per minute per 1.73 square meters of body surface area.	Baseline, Days 22, 43, 71, 99, 113, 127, 155 and 183
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline	CKD stage was classified based on the National Kidney Foundation Chronic Kidney Disease Stage where Stage 5 represents the most severe disease and Stage 1 represents the least severe disease. "Improved" excluded participants with Stage 1 at baseline as there was no room for improvement. "Worsened" excludes participants with Stage 5 at baseline as there was no room to worsen.	Baseline to Days 22, 43, 71, 99, 113, 127, 155 and 183
Change From Baseline in Platelets	Platelet values obtained from the day of a blood transfusion of platelets through 3 days after the transfusion are excluded from all analysis.	Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183
Change From Baseline in LDH		Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183
Change From Baseline in Hemoglobin	Hemoglobin values obtained from the day of a blood transfusion of either whole blood or packed red blood cells through 7 days after the transfusion are excluded from all analysis.	Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals, Inc.
• Collaborators: AstraZeneca

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): July 14, 2023
• Primary Completion (Actual): May 7, 2025
• Study Completion (Actual): May 7, 2025

Study Registration Dates

• First Submitted: May 17, 2023
• First Submitted That Met QC Criteria: May 17, 2023
• First Posted (Actual): May 25, 2023

Study Record Updates

• Last Update Posted (Actual): December 8, 2025
• Last Update Submitted That Met QC Criteria: December 5, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: aHUS; atypical hemolytic uremic
• Additional Relevant MeSH Terms: Urogenital Diseases; Cytopenia; Pathologic Processes; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Hematologic Diseases; Anemia, Hemolytic; Anemia; Blood Platelet Disorders; Thrombotic Microangiopathies; Thrombocytopenia; Uremia; Hemolysis; Hemolytic-Uremic Syndrome; Atypical Hemolytic Uremic Syndrome; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Complement Inactivating Agents; eculizumab
• Other Study ID Numbers: D7413C00001; 2025-000162-29 (EudraCT Number); ECU-aHUS-302 (Other Identifier: Alexion)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD 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 Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No