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    Summary
    EudraCT Number:2013-002191-41
    Sponsor's Protocol Code Number:ECU-MG-302
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-04-16
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2013-002191-41
    A.3Full title of the trial
    A PHASE III, OPEN-LABEL, EXTENSION TRIAL OF ECU-MG-301 TO EVALUATE THE SAFETY AND EFFICACY OF ECULIZUMAB IN SUBJECTS WITH REFRACTORY GENERALIZED MYASTHENIA GRAVIS (gMG)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    EXTENSION STUDY OF ECU-MG-301 TO EVALUATE ECULIZUMAB IN REFRACTORY GENERALIZED MYASTHENIA GRAVIS (GMG) SUBJECTS
    A.4.1Sponsor's protocol code numberECU-MG-302
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02301624
    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 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 supportALEXION PHARMACEUTICALS INC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationALEXION EUROPE SAS
    B.5.2Functional name of contact pointEuropean Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address1-15, avenue Edouard Belin
    B.5.3.2Town/ cityRueil-Malmaison
    B.5.3.3Post code92500
    B.5.3.4CountryFrance
    B.5.4Telephone number+33147100606
    B.5.5Fax number+33147100611
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1304
    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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNECULIZUMAB
    D.3.9.1CAS number 219685-50-4
    D.3.9.2Current sponsor codeh5G1.1-mAb
    D.3.9.4EV Substance CodeSUB25187
    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) 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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboConcentrate for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Generalized Myasthenia Gravis
    E.1.1.1Medical condition in easily understood language
    Myasthenia Gravis
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLT
    E.1.2Classification code 10071942
    E.1.2Term Myasthenia gravis and related conditions
    E.1.2System Organ Class 100000004859
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the long-term safety of eculizumab in subjects with refractory gMG.
    E.2.2Secondary objectives of the trial
    - To evaluate the long-term efficacy of eculizumab in subjects with refractory gMG as measured by the improvement or maintenance of the MG-specific Activities of Daily Living profile (MG-ADL).
    - To evaluate the long-term efficacy of eculizumab by additional efficacy measures including: Quantitative Myasthenia Gravis (QMG) score, Myasthenia Gravis Composite (MGC) score, and Improvement or maintenance in primary symptoms that are most clinically meaningful to the subjects.
    - To characterize the effect of eculizumab on quality of life measures.
    - To describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in subjects with gMG.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject has completed the ECU-MG-301 trial.
    2. Subject has given written informed consent.
    3. Subject is willing and able to comply with the protocol requirements for the duration of the trial.
    4. Female subjects of child-bearing potential must have a negative pregnancy test (serum human chorionic gonadotropin [HCG]). All subjects must practice an effective, reliable and medically approved contraceptive regimen during the trial and for up to 5 months following discontinuation of treatment.
    E.4Principal exclusion criteria
    1. Subjects who withdrew from the ECU-MG-301 trial as a result of an AE related to trial drug.
    2. Female subjects who are pregnant, breastfeeding or intend to conceive during the course of the trial.
    3. Unresolved meningococcal infection
    4. Hypersensitivity to murine proteins or to one of the excipients of eculizumab
    5.Any medical condition or circumstances that, in the opinion of the Investigator, might interfere with the subject’s participation in the trial, pose any added risk for the subject, or confound the assessment of the subjects.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in the MG-ADL total score.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From week 1 to 182
    E.5.2Secondary end point(s)
    1. Change from baseline in QMG total score.
    2. Proportion of subjects with at least a 3-point reduction in the MG-ADL total score from baseline and with no rescue therapy.
    3. Proportion of subjects with at least a 5-point reduction in the QMG total score from baseline and with no rescue therapy.
    4. Change from baseline in the MGC scale total score.
    5. Change from baseline in MG-QOL-15.
    E.5.2.1Timepoint(s) of evaluation of this end point
    From week 1 to 182
    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 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 No
    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
    Induction blinded phase followed by an open label maintenance phase
    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.8.2.4Number of treatment arms in the trial1
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA49
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Australia
    Austria
    Belgium
    Brazil
    Canada
    Czech Republic
    Denmark
    Finland
    France
    Germany
    Greece
    Hungary
    Ireland
    Israel
    Italy
    Japan
    Netherlands
    Norway
    Russian Federation
    Spain
    Sweden
    Switzerland
    Turkey
    United Kingdom
    United States
    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 years4
    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 years4
    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.1.2.1Number of subjects for this age range: 83
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 9
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 92
    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 return to the care of his/her physician.
    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 Decision2015-06-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-03-17
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-01-15
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