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    The EU Clinical Trials Register currently displays   43851   clinical trials with a EudraCT protocol, of which   7283   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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    Summary
    EudraCT Number:2014-003997-18
    Sponsor's Protocol Code Number:GTI1408
    National Competent Authority:Estonia - SAM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-05-26
    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 ConcernedEstonia - SAM
    A.2EudraCT number2014-003997-18
    A.3Full title of the trial
    A Multi-center, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Immune Globulin (Human), 10% Caprylate/ Chromatography Purified (IGIV -C) in Symptomatic Subjects with Generalized Myasthenia Gravis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study investigating Immune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C) for the treatment of patients with Myasthenia Gravis. The patients will receive 2g/kg of IP as a loading dose. The loading dosage is followed by maintenance doses of 1 g/kg administered every third week until Visit 8 (Week 21).
    A.4.1Sponsor's protocol code numberGTI1408
    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 SponsorGrifols Therapeutics 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 supportGrifols Therapeutics Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGrifols Therapeutics Inc.
    B.5.2Functional name of contact pointRhonda Griffin-Director,ClinDev
    B.5.3 Address:
    B.5.3.1Street Address79 T.W. Alexander Drive, Research Triangle Park,
    B.5.3.2Town/ city4101 Research Commons,
    B.5.3.3Post codeNC 27709
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 919 316 6693
    B.5.5Fax number+1 919 287 2089
    B.5.6E-mailrhonda.griffin@grifols.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 GAMUNEX 10%
    D.2.1.1.2Name of the Marketing Authorisation holderGrifols Deutschland GmbH
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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.3.4Pharmaceutical form 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 INNImmune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C)
    D.3.9.3Other descriptive nameHUMAN NORMAL IMMUNOGLOBULIN (IV)
    D.3.9.4EV Substance CodeSUB12041MIG
    D.3.10 Strength
    D.3.10.1Concentration unit g/ml gram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.10
    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 Yes
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution 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
    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 PT
    E.1.2Classification code 10028417
    E.1.2Term Myasthenia gravis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate the efficacy of IGIV-C in subjects with generalized myasthenia gravis (MG) on standard of care treatment at study entry in terms of improvement in MG symptoms as measured by the mean change in Quantitative Myasthenia Gravis (QMG) score from Baseline (Week 0) to Week 24 as compared to placebo.
    E.2.2Secondary objectives of the trial
    1) Percentage of subjects who experience a clinical improvement assessed by QMG score from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 3-point decrease in QMG score
    2) Percentage of subjects who experience a clinical improvement assessed by the MG Composite from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 3-point decrease in the MG Composite
    3) Percentage of subjects who experience a clinical improvement assessed by MG –Activities of Daily Living (MG-ADL) from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 2-point decrease in MG-ADL
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female, ages 18 to 85 years
    2. Anti-AChR antibody positive
    3. Confirmed diagnosis of generalized MG. Historically, subjects may have previously had the Myasthenia Gravis Foundation of America (MGFA) Class II, III, IV, or V.
    4. MGFA classification of Class II, III, or IVa inclusive at Screening.
    5. QMG score ≥10 at Screening. Note: Subjects who only have a history of ocular MG may not enroll.
    6. Receiving standard of care MG treatment at a stable dose consisting of any one of the following for the time intervals delineated below (time intervals apply to medications and maintenance of stable dose level):
    -Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening and no immunosuppressants
    - Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks priorto Screening and/or only one of the following:
    * Prednisone (up to 60 mg/day or equivalent) for at least two months prior to Screening, or
    * Azathioprine for at least 6 months prior to Screening, or
    * Mycophenolate mofetil for at least 6 months prior to Screening, or
    *Methotrexate for at least 6 months prior to Screening, or
    * Cyclosporine or tacrolimus for at least 3 months prior to Screening
    - Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening and/or prednisone (up to 60 mg/day or equivalent) for at least one month prior to Screening and only one of the following:
    * Azathioprine for at least 6 months prior to Screening, or
    * Mycophenolate mofetil for at least 6 months prior to Screening, or
    *Methotrexate for at least 6 months prior to Screening, or
    * Cyclosporine or tacrolimus for at least 3 months prior to Screening
    7. Subjects must be willing and able to provide written informed consent
    8. Subjects must be willing to comply with all aspects of the clinical trial protocol.
    E.4Principal exclusion criteria
    1. Have received cyclophosphamide or any other immunosuppressive agent apart from the ones allowed per inclusion criteria within the past 6 months
    2. Any change in MG treatment regimen between Screening (Week -3, Visit 0) and Baseline (Week 0, Visit 1)
    3. Greater than two (>2) point change in QMG score, increased or decreased, between Screening (Week -3, Visit 0) and Baseline (Week 0, Visit 1)
    4. Any episode of myasthenic crisis in the one month prior to Screening
    5. Evidence of malignancy within the past 5 years (nonmelanoma skin cancer, carcinoma in situ of cervix is allowed) or thymoma potentially requiring surgical intervention during the course of the trial (intent to perform thymectomy)
    6. Thymectomy within the preceding six months
    7. Rituximab, belimumab, eculizumab or any monoclonal antibody used for immunomodulation within the past 12 months
    8. Have received immune globulin (Ig) treatment given by IV, subcutaneous, or intramuscular route within the last 3 months
    9. Current known hyperviscosity or hypercoagulable state
    10. Currently receiving anti-coagulation therapy (vitamin K antagonists, nonvitamin K antagonist oral anticoagulants [e.g., dabigatran etexilate, rivaroxaban, edoxaban, and apixaban], parenteral anticoagulants [e.g., fondaparinux]). Note that oral anti-platelet agents are allowed (e.g., aspirin, clopidogrel, ticlodipine)
    11. Plasma exchange (PLEX) performed within the last 3 months
    12. History of non-response to IVIg when used in maintenance therapy of the subject’s MG, as judged by the Investigator
    13. Any comorbid condition that in the opinion of the Investigator would put the subject at undue safety risk or compromise the ability of the subject to participate in the trial or the scientific integrity of the study
    14. Inadequate venous access to support repeated intravenous infusions
    15. History of anaphylactic reactions or severe reactions to any blood-derived product
    16. History of intolerance to any component of the IP
    17. Documented diagnosis of thrombotic complications to polyclonal IVIg therapy in the past
    18. History of recent (within the last year) myocardial infarction or stroke
    19. Uncontrolled congestive heart failure; embolism; or historically documented (within the last year) electrocardiogram (ECG) changes indicative of myocardial ischemia or atrial fibrillation
    20. History of chronic alcoholism or illicit drug abuse (addiction) in the 12 months preceding the Screening/Week -3 (Visit 0)
    21. Active psychiatric illness that interferes with compliance or communication with health care personnel
    22. Females of child-bearing potential who are pregnant or have a positive serum pregnancy test (beta-human chorionic gonadotropin [β-HCG]-based assay)
    23. Females who are breastfeeding
    24. Females of child-bearing potential who are unwilling to practice a highly effective method of contraception (oral, injectable or implanted hormonal methods of contraception, placement of an intrauterine device or intrauterine system, condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinence*) throughout the study.
    * True abstinence: When this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [e.g., calendar, ovulation, symptothermal, post-ovulation methods], declaration of abstinence for the duration of a trial, and withdrawal are not acceptable methods of contraception.)
    25. Currently receiving, or having received within 1 month prior to the Screening/Week -3 (Visit 0), any investigational medicinal product or device. In the case of an investigational medicinal product trial, at least five half-lives (if known) must have elapsed prior to Screening.
    26. Known Immunoglobulin A (IgA) deficiency and anti-IgA serum antibodies
    27. Renal impairment (i.e., serum creatinine exceeds more than 1.5 times the upper limit of normal [ULN] for the expected normal range for the testing laboratory)
    28. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) levels exceeding more than 2.5 times the ULN for the expected normal range for the testing laboratory.
    29. Hemoglobin levels <9 g/dL
    30. Any medical condition which makes the clinical trial participation unadvisable or which is likely to interfere with the evaluation of the study treatment and/or the satisfactory conduct of the clinical trial according to the Investigator’s judgment. Any factor that in the opinion of the Investigator would compromise the ability of the subject to complete the trial.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is improvement in MG symptoms as measured by the mean change in QMG score from Baseline (Week 0) to Week 24 as compared to placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throuhout the study from Baseline (Week 0) to Week 24.
    E.5.2Secondary end point(s)
    1) Percentage of subjects who experience a clinical improvement assessed by QMG score from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 3-point decrease in QMG score
    2) Percentage of subjects who experience a clinical improvement assessed by the MG Composite from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 3-point decrease in the MG Composite
    3) Percentage of subjects who experience a clinical improvement assessed by MG-ADL from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 2-point decrease in MG-ADL
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) Throughout the study, from Baseline (Week 0) to Week 24
    2) Throughout the study, from Baseline (Week 0) to Week 24
    3) Throughout the study, from Baseline (Week 0) to Week 24
    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) 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 Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Information not present in EudraCT
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    Belgium
    Canada
    Czech Republic
    Estonia
    France
    Germany
    Hungary
    Lithuania
    Poland
    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 years1
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days12
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days12
    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: 56
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects must be willing and able to provide written informed consent.
    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 44
    F.4.2.2In the whole clinical trial 62
    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)
    After the participation in the trial, the patients return to the expected
    normal treatment of that condition
    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 Decision2015-06-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-05-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-01-26
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