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    Summary
    EudraCT Number:2009-014669-13
    Sponsor's Protocol Code Number:C08-001
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2009-10-23
    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 ConcernedUK - MHRA
    A.2EudraCT number2009-014669-13
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled, Cross-Over, Multi-Center Study of Eculizumab in Patients with Generalized Myasthenia Gravis (gMG) Who Have Moderate to Severe Muscle Weakness Despite Treatment with Immunosuppressants.
    A.3.2Name or abbreviated title of the trial where available
    C08-001
    A.4.1Sponsor's protocol code numberC08-001
    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 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 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 Yes
    D.2.1.1.1Trade name SOLIRIS
    D.2.1.1.2Name of the Marketing Authorisation holderALEXION EUROPE
    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 No
    D.2.5.1Orphan drug designation number
    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 Information not present in EudraCT
    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.3.9.3Other descriptive nameAnti-C5 antibody
    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) Information not present in EudraCT
    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 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) Yes
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    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
    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 (gMG)
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 12.0
    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
    • Treatment-emergent adverse events (TEAEs).
    • Percentage of patients with a 3-point reduction from baseline in the Quantitative
    Myasthenia Gravis (QMG) total score for disease severity.
    E.2.2Secondary objectives of the trial
    Change from baseline in the QMG total score for disease severity;
    • Change from baseline in the two most affected QMG individual test items;
    • Change from baseline in the Myasthenia Gravis Foundation of American (MGFA) Post-
    Intervention Status;
    • Change from baseline in MG activities of daily living profile (MG-ADL);
    • Change from baseline in respiratory function tests including spirometry to characterize the degree of involvement of respiratory muscles;
    • Change from baseline in the Quality of life (QoL) instrument SF-36.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patient ≥18 – ≤80 years old.
    2. Generalized MG with prominent clinical symptoms.
    3. Diagnosis of MG must be made by the tests described in the protocol (section 8.1).
    4. MGFA Clinical Classification Class II, III or IVa.
    QMG for Disease Severity total score ≥12.
    6. Minimum score of two (2) in four (4) or more test items in the QMG.
    7. Able to give informed consent.
    8. Able and willing to comply with study procedures.
    9. Sexually active women of childbearing potential must be practicing an effective, reliable and medically acceptable contraceptive regimen during the entire duration of the study and for 5 months after taking the last dose of study drug
    10. Patients shall have failed treatment or have not achieved significant clinical benefit with at least two immunomodulators
    11. If patients enter the study taking azathioprine (AZA) they must have been on AZA for ≥12 months and have been on a stable dose for ≥2 months prior to screening.
    12. If patients enter the study taking other immunosuppressive treatments, they must have been on treatment for ≥6 months and have been on a stable dose for ≥2
    months prior to screening.
    13. Patients who enter the study taking oral corticosteroids must have been on a stable dose for ≥4 weeks (28 days) prior to screening.
    14. Patients who enter the study taking a cholinesterase inhibitor must have been on a stable dose for ≥2 weeks prior to screening.
    E.4Principal exclusion criteria
    1. History of thymoma or other neoplasms of the thymus.
    2. History of thymectomy within 12 months prior to screening.
    3. MG status which in the opinion of the Investigator is unstable or with fixed weakness (i.e. “burned-out”) such that the patient is unlikely to respond to therapy based on the patient’s disease severity, pace of progression and prior MG treatment history.
    4. Pregnancy or lactation.
    5. Active infections at screening.
    6. Current chronic use of plasmapheresis/plasma exchange defined as requiring plasma
    exchange on a regular basis for the management of muscle weakness two or more times
    in one year, or any plasma exchange within 3 months prior to screening.
    7. IVIG treatment within 8 weeks prior to screening.
    8. Use of etanercept (TNF inhibitor, Enbrel®) within 2 months prior to screening.
    9. Use of rituximab (RITUXAN®) within 6 months prior to screening.
    10. Severe weakness predominantly affecting oropharyngeal or respiratory muscles or both
    (MGFA Class IVb).
    11. Crisis or impending crisis as evidenced by forced vital capacity (FVC) <10mL/Kg or
    <35% of predicted value, or bulbar weakness severe enough to compromise airway
    protection at screening.
    12. Need or use of intubation, with or without mechanical ventilation, except when employed during routine postoperative management at screening (MGFA Clinical Classification V).
    13. Weakness only affecting ocular or peri-ocular muscles (MGFA Class I).
    14. History of splenectomy.
    15. Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures within 30 days prior to screening.
    16. History of meningococcal disease.
    17. Known or suspected complement deficiency.
    18. Patients who are not vaccinated against N. meningitidis at least 14 days prior to Visit 2.
    19. Any medical or psychological condition that, in the opinion of the investigator, could increase the patient’s risk by participating in the study or confound the outcome of the study.
    E.5 End points
    E.5.1Primary end point(s)
    1)Treatment-emergent adverse events
    2) Percentage of patients with a 3-point reduction from baseline in the Quantitative Myasthenia Gravis (QMG) total score for disease severity
    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 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 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 Yes
    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 concerned3
    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 Yes
    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
    Last visit of the last patient.
    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 days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    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 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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 24
    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)
    At this time there is no arrangements made for the continued provision of the intervention for participants on this trial when it ends.
    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 Decision2009-11-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-12-29
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2011-03-16
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