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    Summary
    EudraCT Number:2013-005098-28
    Sponsor's Protocol Code Number:GTI1305
    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:2015-09-14
    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 number2013-005098-28
    A.3Full title of the trial
    A multicenter, prospective, open-label, non-controlled clinical trial to assess the efficacy and safety of Immune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C) in patients with Myasthenia Gravis exacerbations
    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 exacerbations. All patients will receive a single dose course of IGIV-C treatment followed by 28-days of post-infusion assessments.
    A.4.1Sponsor's protocol code numberGTI1305
    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 LLC
    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 LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGrifols Therapeutics LLC
    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+1919316 6693
    B.5.5Fax number+1919287 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
    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 number10g/100ml
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Myasthenia Gravis Exacerbations
    E.1.1.1Medical condition in easily understood language
    Myasthenia Gravis Exacerbations
    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 No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To evaluate the efficacy of an an intravenous infusion of IGIV-C (total dose of 2g/kg administered over 2 consecutive days at a dose of 1 g/kg per day) in subjects with MG exacerbations by assessing the change in score of MG symptoms as measured by the QMG scale from baseline to Day 14
    - To evaluate the safety and tolerability of an IV infusion of IGIV-C (total dose of 2 g/kg administered over 2 consecutive days at a dose of 1 g/kg per day) in subjects with MG exacerbations.
    E.2.2Secondary objectives of the trial
    - To evaluate the efficacy of an IV infusion of IGIV-C (total dose of 2 g/kg administered over 2 days at a dose of 1 g/kg per day) in subjects with MG exacerbations by:
    • Percentage of subjects who experience a clinical improvement assessed by QMG from Baseline (Day 0) to Day 14 where clinical improvement is defined as at least 3 point decrease in QMG
    • Percentages of subjects who experience a clinical improvement assessed by MG - Activities of Daily Living (MG-ADL) from Baseline (Day 0) to Day 14 where clinical improvement is defined as at least 2-point decrease in MG-ADL
    • Percentages of subjects who experience a clinical improvement assessed by the MG Composite from Baseline (Day 0) to DAy 14 where clinical improvement is defined as at least 3 point decrease in the MG Composite
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A subject must meet all the following inclusion criteria to be eligible for participation in this
    study:
    1. Male or female aged ≥18 years.
    2. 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).
    3. Subjects who meet the clinical criteria for diagnosis of MG with an exacerbation defined as worsening of MG symptoms as defined by an MGFA classification IVb or V.
    4. Subjects on long-term (8 weeks) corticosteroid treatment for MG.
    5. Female subjects of child-bearing potential must have a negative test for pregnancy (human chorionic gonadotropin (HCG)-based assay).
    6. Subjects must be willing to comply with all aspects of the clinical trial protocol, including blood sampling and long-term storage of extra samples, for the entire duration of the study.
    E.4Principal exclusion criteria
    A subject meeting any of the following exclusion criteria is NOT eligible for participation in the study:
    1. Subjects who have received immune globulin treatment given by IV, subcutaneous or intramuscular route within the last 30 days.
    2. Subjects with documentation of a lack of clinical response to IVIG therapy for MG.
    3. Subjects documented positive for antibodies directed against MuSK.
    4. Subjects with CS treatment initiated within the last 8 weeks or modified within the last 2 weeks.
    5. Subjects with PLEX within the last 30 days.
    6. Subjects with MG exacerbation attributable to change in medication or evident infection as defined by, but not limited to, the presence of at least one of the following diagnostic features: 1) axillary temperature ≥38°C, 2) positive blood culture of infective microorganism, 3) white blood cell count >12×109/L and differential white blood cell count of >10% band neutrophils (>1.2×109/L), and 4) pulmonary infiltrate with consolidation on chest X-ray. Alternatively, other signs and symptoms may be considered for the diagnosis of evident infection according to the Investigator’s judgement.
    7. Subjects with inadequate venous access.
    8. Subjects with a history of anaphylactic reactions or severe reactions to any blood-derived product.
    9. Subjects with a history of intolerance to any component of the investigational products (IPs).
    10. Subjects with a documented diagnosis of thrombotic complications to polyclonal IVIG therapy in the past.
    11. Subjects with a history of recent (within the last year) myocardial infarction, stroke or uncontrolled hypertension.
    12. Subjects who suffer from uncontrolled congestive heart failure, embolism or documented electrocardiogram (ECG) changes indicative of myocardial ischemia or atrial fibrillation.
    13. Subjects with current known hyperviscosity or hypercoagulable state.
    14. Subjects currently receiving anti-coagulation therapy.
    15. Subjects with a history of chronic alcoholism or illicit drug abuse (addiction) in the 12 months preceding the Baseline Visit.
    16. Subjects with active psychiatric illness that interferes with compliance or communication with health care personnel.
    17. Females who are pregnant, breastfeeding, or of child-bearing potential, and 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.)
    18. Subjects with 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.
    19. Subjects currently receiving, or having received within 3 months prior to the Baseline Visit, any investigational medicinal product or device.
    20. Subjects who are unlikely to adhere to the protocol requirements or are likely to be uncooperative or unable to provide a storage serum/plasma sample prior to the first IP infusion.
    21. Subjects with a known Immunoglobulin A (IgA) deficiency and anti-IgA serum antibodies.
    22. Subjects with 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).
    23. Subjects with 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.
    24. Subjects with haemoglobin levels <9 g/dL.
    E.5 End points
    E.5.1Primary end point(s)
    - The primary variable to assess efficacy in this study is the change in QMG score from Baseline (Day 0) to Day 14.
    E.5.1.1Timepoint(s) of evaluation of this end point
    28 ± 2 days
    E.5.2Secondary end point(s)
    Percentage of subjects who experience a clinical improvement assessed by QMG from Baseline (Day 0) to Day 14 where clinical improvement is defined as at least 3 point decrease in QMG
    • Percentage of subjects who experience a clinical improvement assessed by MG-ADL from Baseline (Day 0) to Day 14 where clinical improvement is defined as at least 2 point decrease in MG-ADL
    • Percentage of subjects who experience a clinical improvement assessed by the MG Composite from Baseline (Day 0) to Day 14 where clinical improvement is defined as at least 3 point decrease in the MG Composite
    E.5.2.1Timepoint(s) of evaluation of this end point
    28 ± 2 days
    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 No
    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 No
    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 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 No
    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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    Belgium
    Canada
    Czech Republic
    Estonia
    France
    Hungary
    Latvia
    Poland
    Romania
    Russian Federation
    South Africa
    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
    LSLV
    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 months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    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: 50
    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 Yes
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    The informed consent will have a section for Legal representative signature to be used in these situations.
    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 35
    F.4.2.2In the whole clinical trial 50
    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 Decision2015-10-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-09-21
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-06-12
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