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    Summary
    EudraCT Number:2013-005099-17
    Sponsor's Protocol Code Number:GTI1306
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-06-17
    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 ConcernedCzechia - SUKL
    A.2EudraCT number2013-005099-17
    A.3Full title of the trial
    A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Immune Globulin (Human), 10% Caprylate/ chromatography Purified (lGIV -C) as a Corticosteroid Sparing Agent in Corticosteroid Dependent Patients with Generalized Myasthenia Gravis
    Multicentrické, randomizované, dvojitě zaslepené, placebem kontrolované klinické hodnocení posuzující účinnost a bezpečnost (lidského) imunoglobulinu, s 10% kaprylátem, chromatograficky purifikovaného (IGIV-C), jako přípravku snižujícího nutnost užívání kortikosteroidu u pacientů se symptomatickou generalizovanou myastenií gravis závisejících na užívání kortikosteroidu
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study investigating Immune Globuline (Human), 10% Caprylate/Chromatography Purified (IGIV-C) for the treatment of patients with Myasthenia Gravis, dependent on Corticosteroids. 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 13 (Week 36). During maintenance doses the investigator will try to slowly reduce the patient's corticosteroid dose.
    A.4.1Sponsor's protocol code numberGTI1306
    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 2945
    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 of this study is to evaluate the efficacy of IV infusions of IGIV-C as compared to Placebo in reducing the maintenance dosage of corticosteroids in corticosteroid (CS)-dependent subjects with MG when given as an initial loading dose (2 g/kg) followed by 12 maintenance doses (1 g/kg) every 3 weeks through Week 36 by assessing the percent of subjects achieving a 50% or greater reduction in CS dose(prednisone or equivalent) at Week 39 (Visit 14) from Baseline/Week 0 (Visit 1).
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to evaluate the efficacy of IGIV-C as compared to Placebo from baseline through Week 39 in the following:
    * Percent reduction in daily CS (prednisone or equivalent) dose from Baseline to Week 39
    (Visit 14)
    * Time to first episode of MG worsening, as defined in Section 3.3.3 “Definition and
    Management of MG Worsening”, from Baseline/Week 0 through Week 39 (Visit 1
    through Visit 14)
    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 meeting the clinical criteria for diagnosis of MG defined by the MGFA classification of Class II, III, IV, or V historically (Appendix 2).
    4. At Screening, subjects may have symptoms controlled by CS (for example, only ocular [Class I] symptoms may be evident or there may be no symptoms) or be MGFA Class II-IVa inclusive (Class IVb and Class V excluded). Note: Subjects who only have a history of ocular MG may not enroll.
    5. On systemic CS for a minimum period of at least three months and on a stable CS dose of ≥15 mg/day and ≤60 mg/day (prednisone equivalent) for the month prior to Screening. Individuals on alternate day CS dosing will be judged to be on a daily dose equivalent to half their alternate day dose (i.e., 40 mg/every other day = 20 mg/day).
    6. The investigator feels that tapering the CS dose is currently appropriate (to be commenced as prescribed during this protocol)
    7. At least one previous attempt to taper CS in order to minimize CS dose
    8. Subjects must be willing and able to provide written informed consent (if applicable, a legally authorized representative may provide informed consent on behalf of the subject).
    9. Subjects must be willing to comply with all aspects of the clinical trial protocol.
    E.4Principal exclusion criteria
    1. Any dose change in concomitant immunosuppressant therapy, other
    than CS, in the prior six months
    2. Any change in CS dose or acetylcholinesterase inhibitor (e.g.,
    pyridostigmine) dose in the one month prior to Screening
    3. A three-point change in QMG score, increased or decreased, between
    the Screening/Week -3 (Visit 0) and Baseline (Week 0 [Visit 1])
    4. Any episode of MC in the one month prior to Screening or (at any time
    in the past) MC
    or hospitalization for MG exacerbation associated with a previous CS
    taper attempt
    5. Evidence of malignancy or bulky thymoma potentially requiring surgical intervention during the course of the trial
    6. Thymectomy within the preceding three months prior to Screening
    7. Rituximab, belimumab, eculizumab or any monoclonal antibody used for
    immunomodulation within the past 12 months prior to Screening
    8. History of non-response to IVIg when used in maintenance therapy of the subject’s MG, as judged by the investigator
    9. Have received immune globulin treatment given by IV, subcutaneous, or intramuscular route within the last 3 months prior to Screening
    10. Received plasma exchange (PLEX) performed within the last 3 months prior to Screening
    11. Inadequate venous access
    12. History of anaphylactic reactions or severe reactions to any blood-derived product
    13. History of intolerance to any component of the IPs
    14. Documented diagnosis of thrombotic complications to polyclonal IVIg therapy in the past
    15. History of recent (within the last year) myocardial infarction or stroke
    16. Uncontrolled congestive heart failure; embolism; or historically documented (within the last year) electrocardiogram (ECG) changes indicative of myocardial ischemia or atrial fibrillation
    17. Current known hyperviscosity or hypercoagulable state
    18. Currently receiving anti-coagulation therapy (vitamin K antagonists, nonvitamin K antagonist oral anticoagulants [e.g., dabigatran etexilate targeting Factor IIa, rivaroxaban, edoxaban, and apixaban targeting Factor Xa], parenteral anticoagulants [e.g., fondaparinux]). Note that oral anti-platelet agents are allowed (e.g., aspirin, clopidogrel, ticlodipine)
    19. History of chronic alcoholism or illicit drug abuse (addiction) in the 12 months preceding the Screening/Week -3 (Visit 0)
    20. Active psychiatric illness that interferes with compliance or communication with health care personnel
    21. Females of child-bearing potential who are pregnant, have a positive serum pregnancy test (human chorionic gonadotropin [HCG]-based assay), breastfeeding, or 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.)
    22. 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.
    23. Known Immunoglobulin A (IgA) deficiency and anti-IgA serum antibodies
    24. 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)
    25. 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.
    26. Hemoglobin levels <9 g/dL
    27. 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 Principal investigator would compromise safety of the subject or
    the ability of the subject to complete the trial. No subject whose only MG
    treatment is CS alone may enroll, because in the blinded placebo arm
    this would mean that all MG treatment would be discontinued during CS
    taper, posing a substantial risk for the subject.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint will be the percent of subjects in each arm achieving a 50% or greater reduction in CS dose (prednisone or equivalent) at Week 39 (Visit 14) from Baseline/Week 0 (Visit 1).
    E.5.1.1Timepoint(s) of evaluation of this end point
    at Week 39 (Visit 14) from Baseline/Week 0 (Visit 1).
    E.5.2Secondary end point(s)
    1. Percent reduction in daily CS (prednisone or equivalent) dose from Baseline/Week 0 to Week 39 (Visit 14)
    2. Time to first episode of MG worsening, as defined in Section 3.3.3 “Definition and Management of MG Worsening”, from Week 0 through Week 39 (Visit 1 through Visit 14)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Throughout the study at each visit until the Week 39 including.
    2. Throughout the study at each visit until the Week 39 including.
    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 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 EEA24
    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
    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 months8
    E.8.9.1In the Member State concerned days27
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days27
    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: 54
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 43
    F.4.2.2In the whole clinical trial 60
    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 Decision2015-09-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-07-08
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-02-14
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