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    Summary
    EudraCT Number:2005-002740-26
    Sponsor's Protocol Code Number:MG-02
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2005-12-01
    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 ConcernedUK - MHRA
    A.2EudraCT number2005-002740-26
    A.3Full title of the trial
    A Phase II Double Blind, Cross-Over Study to Compare the Safety and Efficacy of 10, 20 and 40 mg Monarsen (EN101) administered to Patients with Myasthenia Gravis
    A.4.1Sponsor's protocol code numberMG-02
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorEster Neuroscience Ltd
    B.1.3.4CountryIsrael
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 No
    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/3/04/203
    D.3 Description of the IMP
    D.3.1Product nameMONARSEN
    D.3.2Product code EN101
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeEN101
    D.3.9.3Other descriptive nameMONARSEN
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeEN101
    D.3.9.3Other descriptive nameMONARSEN
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeEN101
    D.3.9.3Other descriptive nameMONARSEN
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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) Information not present in EudraCT
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.8Extractive medicinal product Information not present in EudraCT
    D.3.11.9Recombinant medicinal product Information not present in EudraCT
    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
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 8.1
    E.1.2Level LLT
    E.1.2Classification code 10028417
    E.1.2Term Myasthenia gravis
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To assess the efficacy of three doses of Monarsen given once daily for one week. Efficacy will be assessed by evaluating changes in the QMG score.
    - Safety will be assessed by evaluating adverse events and laboratory tests during a 3 week treatment period (dosing on alternative weeks with Mestinon, over 5 weeks) and a 4 week follow up period.
    E.2.2Secondary objectives of the trial
    - To assess the quality of life using the ADL-MG
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To be considered eligible to participate in this study, a patient must meet the inclusion criteria listed below:

    (1) Clinical diagnosis of Myasthenia Gravis according to the MGFA (MG Foundation of America) classification being defined as a minimum of class II in whom it is safe to interrupt Mestinon treatment.

    (2) Patients (male or female) aged 18 years or above.

    (3) Patients must be seropositive for AChR antibodies (greater than 0.5 nM) or seropositive for anti MUSK antibodies. (Levels of anti MUSK greater then 0.05 fMol/L)

    (4) Laboratory Test of TSH and T4, within the normal range.

    (5) Previously treated with Mestinon (at least 3 tab. a day of 60mg each) until 12-18 hours prior to study initiation, with or without concomitant prednisolone or immunosuppressive treatment, which has been stable for at least 2 months.

    (6) Have hepatic and renal function, as well as coagulation parameters as documented by the following laboratory parameters, within local laboratory normal limits:
    – AST
    – Bilirubin
    – Creatinine
    – Platelets
    – Prothrombin time
    – Activated partial thromboplastin time (αPTT)

    (7) Women of childbearing potential are excluded
    Post-menopausal women (ie women who have been post-menopausal for at least 1 year) and women who have had a hysterectomy can be included.
    Men in relationship with women of childbearing potential, must agree to use effective contraceptive methods during the course of the study and for a 3 month follow-up period.

    (8) Have ability to understand the requirements of the study, have provided written informed consent, and agree to abide by the study restrictions and to return for the required assessments.

    (9) QMG score (while on Mestinon) > 3 points and QMG score while on washout > 3 points greater than score while on Mestinon.
    E.4Principal exclusion criteria
    To be eligible for entry into the study, the patient must not meet any of the exclusion criteria listed below:

    (1) Patients have one of the following symptoms: acute respiratory failure/insufficiency, major difficulty in swallowing, functional disability responsible for the discontinuation of physical activity, within 1 month prior to screening.

    (2) Patients have exclusive mild ocular MG.

    (3) Body weight >100 kg

    (4) Patients in whom disease is so severe that it would be unethical to delay conventional therapy.

    (5) Have participated in any experimental protocol within the preceding one month.

    (6) Have received an IVIG infusion or plasmapheresis treatment, within 8 weeks prior to study initiation.

    (7) Abnormal CBC count (Hgb less than 12 g/dl, WBC less than 4,000/ml, or platelet count less than 130,000/ml).

    (8) Have developed other autoimmune disease within one month prior to study initiation.

    (9) Have uncontrolled hypertension, severe hepatic or renal disease, or other severe general or psychiatric disease.

    (10) Be pregnant or lactating.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy
    The study is not designed to achieve a certain power to detect effectiveness of the study drug. The efficacy analysis will therefore involve evaluating the degree of success with which the study drug ameliorates symptoms that appear when Monarsen treatment is given.

    Safety
    Changes from the screening period (prior to removal from Mestinon) of vital signs, hematology, clinical chemistry, urinalysis, ECG parameters and physical exam to the end of the study will be examined. Treatment emergent and/or clinically significant changes from normal to abnormal values in key laboratory parameters will be identified.
    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 Yes
    E.6.10Pharmacogenetic Information not present in EudraCT
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Information not present in EudraCT
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans Information not present in EudraCT
    E.7.1.2Bioequivalence study Information not present in EudraCT
    E.7.1.3Other Information not present in EudraCT
    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 No
    E.8.1.6Cross over Yes
    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 concerned2
    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 Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    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 months0
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 30
    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)
    Expected normal treatment
    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 Decision2006-02-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2005-11-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2007-07-03
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