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    Summary
    EudraCT Number:2025-000162-29
    Sponsor's Protocol Code Number:D7413C00001
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2025-08-27
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2025-000162-29
    A.3Full title of the trial
    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
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Single-Arm Study of Eculizumab in Pediatric and Adult Participants with Atypical Hemolytic Uremic Syndrome (aHUS) in China
    A.4.1Sponsor's protocol code numberD7413C00001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05876351
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAlexion Europe SAS
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAlexion Europe SAS
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlexion Pharmaceuticals Inc
    B.5.2Functional name of contact pointAshish Thomas Mathew
    B.5.3 Address:
    B.5.3.1Street AddressBlanchardstown Road North
    B.5.3.2Town/ cityDublin College Park
    B.5.3.3Post codeD15R925
    B.5.3.4CountryIreland
    B.5.6E-mailclinicaltrials.eu@alexion.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Soliris
    D.2.1.1.2Name of the Marketing Authorisation holderAlexion Europe SAS
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSoliris
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Atypical Hemolytic Uremic Syndrome (aHUS)
    E.1.1.1Medical condition in easily understood language
    A rare and life-threatening disease characterized
    by complement-mediated thrombotic microangiopathy (TMA)
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of eculizumab in
    the treatment of participants with aHUS
    E.2.2Secondary objectives of the trial
    To characterize the safety and tolerability
    of eculizumab in participants with aHUS
    E.2.3Trial contains a sub-study No
    E.3Principal 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
    E.4Principal 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.
    E.5 End points
    E.5.1Primary end point(s)
    Number of Participants Considered as Complete Thrombotic Microangiopathy (TMA) Responders
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline through Week 26
    E.5.2Secondary end point(s)
    1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    2. Pharmacokinetics (PK): Serum Eculizumab Concentration

    3. Change From Baseline in Serum Free and Total Complement 5 (C5) Concentration at Week 26

    4. Number of Participants With Positive Antidrug Antibodies (ADAs) and Neutralizing Antibodies (NAbs) to Eculizumab

    5. Time to Complete TMA Response

    6. Number of Participants Requiring Dialysis

    7. Number of Participants Classified as Improved, Stable (No Change), or Worsened Per Chronic Kidney Disease (CKD) Stage Classification

    8. Number of participants observed value and change from baseline in hematologic parameters (platelets, LDH, hemoglobin) at visits

    9. Number of participants Increase in hemoglobin of ≥ 20 g/L

    10. Number of participants Changes from baseline in vital signs and laboratory parameters at scheduled visits
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline through Week 26
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    China
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days27
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 10
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 7
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 15
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 25
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: China
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