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    Summary
    EudraCT Number:2014-001169-28
    Sponsor's Protocol Code Number:13705201
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-07-31
    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
    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 ConcernedFrance - ANSM
    A.2EudraCT number2014-001169-28
    A.3Full title of the trial
    Traitement précoce de patients pédiatriques présentant un syndrome hémolytique et urémique typique par l’anticorps monoclonal anti-C5 eculizumab : essai thérapeutique prospectif randomisé contrôlé contre placebo de phase III
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Traitement précoce de patients pédiatriques présentant un syndrome hémolytique et urémique typique par l’anticorps monoclonal anti-C5 eculizumab : essai thérapeutique prospectif randomisé contrôlé contre placebo de phase III
    A.4.1Sponsor's protocol code number13705201
    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 SponsorCentre Hospitalier de Toulouse
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportMinistry of Health (DGOS)
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCentre hospitalier de Toulouse
    B.5.2Functional name of contact pointNadège Algans
    B.5.3 Address:
    B.5.3.1Street Address2, rue Viguerie
    B.5.3.2Town/ cityToulouse
    B.5.3.3Post code31059
    B.5.3.4CountryFrance
    B.5.4Telephone number+33561777204
    B.5.5Fax number+33561778411
    B.5.6E-mailalgans.n@chu-toulouse.fr
    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 AuthorisationFrance
    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 nameeculizumab
    D.3.4Pharmaceutical form Solution for injection
    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) Yes
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 glucose 5% Viaflo
    D.2.1.1.2Name of the Marketing Authorisation holderBaxter SAS
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 nameGlucose solution 5%
    D.3.4Pharmaceutical form Solution for injection
    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 Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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
    Hemolytic and uremic syndrome
    E.1.1.1Medical condition in easily understood language
    Hemolytic and uremic syndrome
    E.1.1.2Therapeutic area Diseases [C] - Symptoms and general pathology [C23]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10042814
    E.1.2Term Syndrome hemolytic uremic
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluer l’impact d’un traitement précoce par ECZ sur l’évolution de l’IRA chez des enfants atteints de SHU-STEC dans le cadre d’un essai thérapeutique prospectif contrôlé contre placebo.
    E.2.2Secondary objectives of the trial
    - Evaluer la tolérance et l’innocuité d’un traitement par ECZ chez des enfants atteints de SHU-STEC.
    - Evaluer la durée de l’IRA chez des enfants atteints de SHU-STEC traités par ECZ ou placebo.
    - Evaluer les séquelles rénales à court et moyen terme chez des enfants atteints de SHU-STEC traités par ECZ ou placebo.
    - Evaluer la durée des anomalies hématologiques chez des enfants atteints de SHU-STEC traités par ECZ ou placebo.
    - Evaluer les paramètres biologiques d’activation de la VAC chez des enfants atteints de SHU-STEC traités par ECZ ou placebo.
    - Evaluer l’inhibition du CAM chez des enfants atteints de SHU-STEC traités par ECZ ou placebo.
    - Evaluer l’incidence des manifestations extra rénales (neurologiques, cardiaques et digestives) chez des enfants atteints de SHU-STEC traités par ECZ ou placebo.
    - Evaluer la mortalité à court terme chez des enfants atteints de SHU-STEC traités par ECZ ou placebo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Pediatric patient (1 month-18 years old)
    . Affected by STEC-HUS defined by:
    - Thrombocytopenia (<150 000/mm3)
    - Mechanic hemolytic anemia (Hemoglobin < 10g/dL, haptoglobin <LLN, LDH >ULN and/or bilirubine >ULN, presence of schizocytes)
    - ARF defined by an estimated Schwartz 2009 creatinin clairance <75ml/min/1,73m²
    - With prodromal diarrhea and/or presence of an enterohemorragic strain of Escherichia Coli and/or identification of the Stx 1 or 2 genes in the stool sample or rectal swab
    E.4Principal exclusion criteria
    . Neonatal HUS
    . Malignancy
    . Known HIV infection
    . Pregnancy or lactation
    . Identified drug exposure-related HUS
    . Infection-related HUS
    . Known systemic lupus erythematosus or antiphospholid antibody positivity or syndrome
    . Patient already enrolled in a drug trial
    . Patient with ongoing meningococcal infection
    . Patient affected by aHUS or family history of aHUS
    . STEC-HUS patient with severe multiorgan involvement at diagnostic:
    • Neurological involvement (seizures, coma, focal deficit) with signs of microangiopathy on cerebral Magnetic Resonance Imaging.
    • Cardiac involvement (cardiac failure, ischemic myocarditis, conduction or rhythm troubles)
    • Digestive involvement (severe pancreatitis defined by lipasemia>500UI/L, severe hepatitis defined by transaminase >x10ULN and/or prothrombin time<60%, hemorrhagic colitis, bowel perforation, rectal prolapsus)
    E.5 End points
    E.5.1Primary end point(s)
    Primary evaluation criteria will be the duration in days of extrarenal epuration
    E.5.1.1Timepoint(s) of evaluation of this end point
    during 9 weeks
    E.5.2Secondary end point(s)
    Safety of ECZ injections. Adverse reactions of ECZ
    Duration of ARF will be evaluated as follow: urine output measurement, GFR estimated with the Schwartz 2009 assay (based on creatinin plasma levels), ionogram, proteinuria.
    Renal sequels will be evaluated at 1, 6 and 12 months after last injection of ECZ: blood pressure, GFR, ionogram, proteinuria and microalbuminuria.
    Hematological abnormalities will be evaluated as follow:
    • Duration of the thrombocytopenia
    • Duration of the hemolytic anemia
    Blood parameters of CAP will be evaluated with plasmatic dosages of the complement components C3 and CD46.
    Inhibition of the TCC will be evaluated trough the CH50 assay and plasmatic free ECZ levels.
    Incidence of extrarenal manifestations:
    • Neurological involvement (seizures, coma, focal deficit)
    • Cardiac involvement (cardiac failure, ischemic myocarditis, conduction or rhythm troubles)
    • Digestive involvement (pancreatitis, hepatitis, hemorrhagic colitis, bowel perforation, rectal prolapsus)
    Mortality rates
    E.5.2.1Timepoint(s) of evaluation of this end point
    during 9 weeks
    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 No
    E.6.7Pharmacodynamic No
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind Yes
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3.1Comparator description
    solution de dilution du médicament expérimental
    E.8.2.4Number of treatment arms in the trial2
    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 concerned17
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months1
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 100
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 100
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 100
    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 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.4.1In the member state100
    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
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-07-17
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
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