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The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   43693   clinical trials with a EudraCT protocol, of which   7245   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    EudraCT Number:2008-006954-17
    Sponsor's Protocol Code Number:C08-003A
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2009-03-18
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedAustria - BASG
    A.2EudraCT number2008-006954-17
    A.3Full title of the trial
    A.4.1Sponsor's protocol code numberC08-003A
    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.1.3.4CountryUnited 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.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. name SOLIRIS
    D. 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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. 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
    Adult patients with plasma therapy-sensitive 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 on 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, or [3] new dialysis.
    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.
    − Reduction of thrombotic microangiopathy (TMA) as indicated by thrombocytopenia as measured by platelet count change from baseline duringthe first 26 weeks of the treatment period.
    − 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 ≥ 18 years of age who have been diagnosed with aHUS.
    2. Patients must be receiving PT for aHUS and must be observed to (i) receive ≥1 PT
    treatment every two weeks and no more than 3 PT treatments/week (at an unchanged
    frequency) for at least 8 weeks before the first dose of Investigational Product.
    3. Platelet Count Pre-PT Baseline Set-Point (collected in the hours before the
    Qualifying PT Episode) is within 75% of the average of the pre-PT platelet counts
    collected at Screening and during the Observation Period.
    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 HIV positivity, and antiphospholipid 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) at screening or at the onset of the current aHUS
    episode was ≥ 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.
    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 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. Able to give written informed consent.
    10. Able and willing to comply with study procedures.
    E.4Principal exclusion criteria
    1. Thrombotic Thrombocytopenic Purpura (TTP).
    2. History of malignancy within 5 years of screening.
    3. Typical HUS (known Shiga toxin +).
    4. Known human immunodeficiency virus (HIV) infection.
    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. 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.
    10. 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.
    11. Pregnancy or lactation.
    12. Unresolved meningococcal disease.
    13. Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or syndrome.
    14. 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.
    15. Patients who have received previous treatment with eculizumab.
    16. Patients receiving intravenous immunoglobulin (IVIg) within 8 weeks or Rituximab
    therapy within 12 weeks of the screening visit.
    17. 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 and throughout the Observation Period or [3] patient is experiencing an acute aHUS relapse immediately after transplant.
    18. 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.
    19. 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.
    20. Hypersensitivity to eculizumab, to murine proteins, or to one of the excipients.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint for this protocol is to assess the effect of
    eculizumab on 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. Dialysis events occurring within the 14 days after the first dose of
    Investigational Product will not be considered as a new Treatment Period dialysis
    event. In addition, dialysis events that commence within the 14 days before the
    first dose of Investigational Product and continue up to 14 days after the first
    dose of Investigational Product will not be considered a new Treatment Period
    dialysis event.
    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. 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. 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. 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA54
    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
    Last Patient Last Visit.
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 15
    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 discontinue investigational product during the study or who do not enter the extension 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 Decision2009-06-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-06-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2013-10-09
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