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Atypical Hemolytic UremicPhase 3SolirisNCT05876351

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

A Phase 3 study testing Eculizumab in people with Atypical Hemolytic Uremic, run by Alexion Pharmaceuticals, Inc. at 6 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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 Read more about this study

Who is it for?
Ages 0 to 99 years
Conditions the study is for
Atypical Hemolytic Uremic
What is being tested?
Eculizumab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
25 participants
Where
6 sitesChina
Study dates
14 Jul 2023to 7 May 2025
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 0 to 99 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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.
Show all 21Show fewer
  • 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.
  • Receiving chronic intravenous immunoglobulin (IVIg) within 8 weeks prior to the start of Screening.
  • Received vasopressors or inotropes within 7 days prior to Screening.
  • Previously or currently treated with a complement inhibitor.
  • Has participated in another interventional treatment study or used any experimental therapy.
  • Hypersensitivity to any excipient in eculizumab.
  • Pregnant or breastfeeding.

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

China6 sites

  • Research Site

    Beijing, 100034

  • Research Site

    Beijing, 100045

  • Research Site

    Changsha, 410007

  • Research Site

    Qingdao, 110016

  • Research Site

    Taiyuan, 030012

  • Research Site

    Wuhan, 430030

Other trials in China

About this study

Official title: 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

Read the 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.

Being tested
Eculizumab
Keywords
atypical hemolytic uremic, aHUS

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EculizumabDrug

    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.

Study groups in NCT05876351
Study groupRoleWhat they receive
EculizumabExperimentalParticipants will receive Eculizumab in a single dose vial.
Receives: Drug: Eculizumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Up to Week 26

Secondary outcomes (12)

  1. 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.

    Measured: Up to Week 34

  2. Mean Serum Concentration of Eculizumab

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

  3. Change From Baseline in Serum Free Complement 5 (C5)

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

  4. Change From Baseline in Serum Total C5

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

  5. 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.

    Measured: Up to Week 26

  6. 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:

    1. Normalization of platelet count (defined as platelet count ≥ 150000/ul.
    2. Normalization of LDH, defined as LDH ≤ ULN).
    3. ≥ 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.

    Measured: Up to Week 26

  7. 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.

    Measured: Baseline and Days 22, 43, 71, 99, 113, 127, 155 and 183

  8. 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.

    Measured: Baseline, Days 22, 43, 71, 99, 113, 127, 155 and 183

  9. 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.

    Measured: Baseline to Days 22, 43, 71, 99, 113, 127, 155 and 183

  10. 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.

    Measured: Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183

  11. Change From Baseline in LDH

    Measured: Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183

  12. 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.

    Measured: Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 May 2023
  2. Study start14 Jul 2023
  3. Primary completion7 May 2025
  4. Study completion7 May 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
open-label study
Masking
None (open label)
Enrolment
25 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05876351
Sponsor's protocol ID
D7413C00001
Other IDs
ECU-aHUS-302, 2025-000162-29, 2025
Acronym
Soliris
Status last verified
Dec 2025
First submitted
17 May 2023
Last updated
8 Dec 2025
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: 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.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Eculizumab in people with Atypical Hemolytic Uremic, run by Alexion Pharmaceuticals, Inc. at 6 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 8 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 8 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response (time frame: Up to Week 26). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05876351.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Dec 2025.

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