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    Summary
    EudraCT Number:2008-006953-41
    Sponsor's Protocol Code Number:C08-002B
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2009-03-04
    Trial results View 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
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSweden - MPA
    A.2EudraCT number2008-006953-41
    A.3Full title of the trial
    AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADOLESCENT PATIENTS WITH PLASMA THERAPY-RESISTANT ATYPICAL HEMOLYTIC-UREMIC SYNDROME (AHUS)
    A.4.1Sponsor's protocol code numberC08-002B
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorALEXION PHARMACEUTICALS, INC.
    B.1.3.4CountryUnited States
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/09/653
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNeculizumab
    D.3.9.2Current sponsor codeh5G1.1-mAb
    D.3.9.3Other descriptive nameAnti-C5 antibody
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) Yes
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Information not present in EudraCT
    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
    Adolescent patients (from 12 and up to 18 years of age) with plasma therapyresistant Atypical Hemolytic-Uremic Syndrome (aHUS).
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10018932
    E.1.2Term Haemolytic uraemic syndrome
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Assess the effect of eculizumab to reduce thrombotic microangiopathy (TMA) as
    indicated by thrombocytopenia as measured by platelet count change from baseline
    during the treatment period to the first 26 weeks in patients with PT-resistant aHUS.
    E.2.2Secondary objectives of the trial
    • Evaluate additional efficacy endpoints during the treatment period to the first 26
    weeks such as the effect of eculizumab on:
    − TMA Intervention Rate (# PT and # Dialysis Events/Patient/Day) during the first
    26 weeks of the Treatment Period compared with the TMA Intervention Rate
    prior to the first dose of Investigational Product.
    − TMA-Event Free status defined as the absence for at least 12 weeks of [1]
    decrease in platelet count of >25% from the Platelet Count Pre-PT Baseline Set-
    Point; [2] PT while the patient is receiving eculizumab, and [3] new dialysis
    − Key Hemolytic measures.
    − Quality of Life measures.
    − Renal function measures.
    − TMA Remission.
    • Characterize the overall safety and tolerability of eculizumab.
    • Describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in
    patients with aHUS.
    • Perform a series of exploratory efficacy analyses during the treatment period to the
    first 26 weeks.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patients between ages from 12 and up to 18 year weighing ≥ 40 kg who
    have been diagnosed with aHUS. Patients may be newly diagnosed, experiencing a
    relapse of the disease, or having a post-transplant recurrence of the disease.
    2. Patients must exhibit a decrease in platelet count despite at least 4 PT treatments in the 1
    week immediately prior to screening. At screening, platelet count must be < 150 x
    109/L and at least 25% lower than the average of 3 platelet counts obtained during the
    most recent TMA remission and at least 1 month apart during that remission prior to
    screening (designated the “average remission platelet count”).
    3. If historical counts are not available, platelet count at onset of the current aHUS episode
    must be ≤ 75 x 109/L, and platelet count at screening must be ≤ 100 x 109/L despite PT
    treatment administration of at least 4 PT treatments in the 1 week immediately prior to
    screening.
    4. Known complement regulatory protein genetic abnormality, i.e., a mutation in
    Complement Protein 3, factor H or associated factor, factor I, or membrane cofactor
    protein (MCP) or known Factor B gain-of-function mutation, or known Anti-CFH
    antibody (“aHUS lesions”).
    • Patients diagnosed with aHUS with any of these aHUS lesions are eligible and will be assigned to one of the following parallel categories during the treatment period of
    the trial:
    − (Category 1) Factor H or factor I functional deficiency, abnormal factor
    interaction (CFH/CFI FFP Group), or deletions of the CFHR1 and CFHR3
    genes;
    − (Category 2) Complement Protein 3, abnormal factor interaction (C3) or Factor
    B Gain of Function;
    − (Category 3) Anti-CFH Antibody (anti-CFH Group);
    − (Category 4) MCP deficiency (MCP Group);
    5. Patients diagnosed with aHUS without documented complement regulatory protein
    genetic abnormality or known anti-CFH antibody are eligible if other etiologies of
    hemolytic uremic syndrome (HUS) have been ruled out as confirmed in the Exclusion
    Criteria (i.e., including Shiga-toxin negative, non-infectious, non-drug-exposure-related
    [e.g., cyclosporine]), no known human immunodeficiency syndrome (HIV) positivity,
    and anti-phospholipid antibody negative). Patients meeting these conditions will be
    assigned to Category 5. In addition, these patients will undergo genetic testing to
    determine if a mutation can be identified. If a mutation is identified, the patient will be
    reassigned to the appropriate category.
    6. Lactate dehydrogenase (LDH) level ≥ upper limit of normal (ULN) unless the patient
    has been receiving plasma exchange (PE) and LDH at the onset of the current aHUS
    episode was at least the ULN. If LDH is normal at screening, other markers indicative
    of ongoing hemolysis should be evaluated, such as haptoglobin, schistocytes, and
    discussed with the Sponsor;
    7. Creatinine level ≥ ULN for age (patients requiring acute dialysis for acute renal failure
    also eligible).
    8. Female patients of childbearing potential must be practicing an effective, reliable and
    medically approved contraceptive regimen during the entire duration of the study,
    including the follow-up period. At the time of the last follow-up visit, patients will be
    counseled by the principal investigator (PI) or designated study staff that they must
    continue to use adequate contraception methods for up to 5 months following
    discontinuation of eculizumab treatment.
    9. Patient’s parents/legal guardian must be willing and able to give written informed
    consent and patients must be willing to give written informed assent (if applicable as
    determined by the IRB/IEC).
    10. Able and willing to comply with study procedures.
    E.4Principal exclusion criteria
    1. Thrombotic Thrombocytopenic Purpura (TTP), defined as ADAMTS13 activity <5% from an historical observation (prior to initiation of Plasma Therapy) or as tested at the screening visit by the central laboratory.
    2. History of malignancy within 5 years of screening.
    3. Typical HUS (known Shiga toxin +).
    4. Known HIVinfection.
    5. Identified drug exposure-related HUS.
    6. Infection-related HUS
    7. HUS related to bone marrow transplant (BMT)
    8. HUS related to vitamin B12 deficiency.
    9. Renal function status requiring chronic dialysis (defined as dialysis on a regular basis as
    renal replacement therapy).
    10. Patients with a confirmed diagnosis of sepsis defined as positive blood cultures within 7
    days of the screening visit and not treated with antibiotics to which the organism is
    sensitive.
    11. Presence or suspicion of active and untreated systemic bacterial infection that, in the
    opinion of the Investigator confounds an accurate diagnosis of aHUS or impedes the
    ability to manage the aHUS disease.
    12. Pregnancy or lactation.
    13. Unresolved meningococcal disease.
    14. Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or
    syndrome.
    15. Any medical or psychological condition that, in the opinion of the investigator, could
    increase the patient’s risk by participating in the study or confound the outcome of the
    study.
    16. Patients who have received previous treatment with eculizumab.
    17. Patients receiving intravenous immunoglobulin (IVIg) within 8 weeks or Rituximab
    therapy within 12 weeks of the screening visit.
    18. Patients receiving other immunosuppressive therapies such as steroids, calcineurin
    inhibitors (mTOR) or tacrolimus are excluded unless: [1] part of an established posttransplant
    anti-rejection regime and dose of such medications have been unchanged for at least 4 weeks prior to the screening period, or [2] patient has confirmed anti-CFH
    antibody requiring immunosuppressive therapy and dose of such medications have been
    unchanged for at least 4 weeks prior to the screening period or [3] patient is
    experiencing an acute aHUS relapse immediately after transplant.
    19. Patients receiving Erythrocyte stimulating agents (ESAs) unless already on a stable dose
    for at least 4 weeks prior to the screening period, or a washout period of at least 2 weeks
    from the last dose of ESA therapy.
    20. Participation in any other investigational drug trial or exposure to other investigational
    agent, device, or procedures beginning 4 weeks prior to screening and throughout the
    entire trial.
    21. Hypersensitivity to eculizumab, to murine proteins or to one of the excipients.
    22. Patients between the ages from 12 and up to 18 years weighing < 40 kg.
    E.5 End points
    E.5.1Primary end point(s)
    Platelet count change from baseline (Platelet Count Pre-PT Baseline Set-point
    value) to the end of the first 26 weeks of treatment with the Investigational Product.
    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 Yes
    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 Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    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 Yes
    E.8.1.7.1Other trial design description
    The protocol has pre-defined objectives and pre-specified end points to assess eculizumab’s efficacy
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    In protocol each patient serves as his/her own control as described by the endpoints in the protocol
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA31
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    E.8.7Trial has a data monitoring committee Yes
    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
    The last visit of the last patient
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Non Adult Subjects
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 7
    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)
    Patients will be allowed to continue to receive treatment with the investigational product until product is registered for the treatment of aHUS and available to these patients (in accordance with country specific regulation).
    Patients who prematurely discontinue investigational product during the study will
    require follow-up visits for 8 weeks after the last dose of eculizumab.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2009-04-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-03-24
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2011-10-17
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