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    Summary
    EudraCT Number:2016-001384-37
    Sponsor's Protocol Code Number:ECU-MG-303
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-12-14
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2016-001384-37
    A.3Full title of the trial
    An Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Eculizumab in Pediatric Patients with Refractory Generalized Myasthenia Gravis (gMG)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Eculizumab in Pediatric Patients with Refractory Generalized Myasthenia Gravis (gMG)
    A.4.1Sponsor's protocol code numberECU-MG-303
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/388/2020
    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 supportAlexion Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlexion Europe SAS
    B.5.2Functional name of contact pointEuropean Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address103-105 rue Anatole France
    B.5.3.2Town/ cityLevallois-Perret
    B.5.3.3Post code92300
    B.5.3.4CountryFrance
    B.5.4Telephone number+33147100615
    B.5.5Fax number+33147100611
    B.5.6E-mailclinicaltrials.eu@alexion.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 Soliris
    D.2.1.1.2Name of the Marketing Authorisation holderAlexion Europe SAS
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1304
    D.3 Description of the IMP
    D.3.1Product nameSoliris
    D.3.2Product code Soliris
    D.3.4Pharmaceutical form Concentrate for 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 INNECULIZUMAB
    D.3.9.1CAS number 219685-50-4
    D.3.9.4EV Substance CodeSUB25187
    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) 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 No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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
    Refractory Generalized Myasthenia Gravis
    E.1.1.1Medical condition in easily understood language
    Refractory Generalized 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 21.1
    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
    - Evaluate the efficacy of eculizumab in the treatment of pediatric refractory generalized myasthenia gravis (gMG)
    E.2.2Secondary objectives of the trial
    - Evaluate the safety and tolerability of eculizumab in the treatment of pediatric refractory gMG
    - Evaluate the efficacy of eculizumab in the treatment of pediatric refractory gMG
    - Evaluate MGFA Post-Interventional Status over time
    - Describe the total number and percentage of patients with clinical deteriorations, myasthenic crises, and rescue therapy use over time
    - Describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab treatment in pediatric refractory gMG patients
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    ● Male or female pediatric patients 6 to < 18 years of age at time of assent/consent
    ● Diagnosis of MG confirmed by positive serologic test for anti-AChR-Ab at Screening, and one of the following: history of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography or repetitive nerve stimulation, or history of positive anticholinesterase test, or patient demonstrated improvement in MG signs on oral AChIs, as assessed by the Investigator
    ● Presence of refractory gMG, defined as patients with gMG who have one or more of the following: failed treatment ≥ 1 year with at least 1 IST, require maintenance PE or IVIg to control symptoms, in the opinion of the Investigator MG poses a significant functional burden despite current MG treatment.
    ● All MG-specific treatment has been administered at a stable dosing regimen of adequate duration prior to Screening
    ● Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to IV at Screening
    ● In patients aged 12 to 18 years, QMG total score ≥ 12 at Screening; in patients aged 6 to 11 years, no minimum QMG is required for inclusion; however, patients must have documented limb weakness in at least one limb.
    ● Patients must be vaccinated against meningococcal infections within the 3 years prior to, or at the time of, initiating study drug. Patients who initiate study drug treatment less than 2 weeks after receiving a meningococcal vaccine must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination.
    ● Documented vaccination against H influenzae and S pneumoniae infections prior to dosing as per local and country specific immunization guidelines for the appropriate age group.
    E.4Principal exclusion criteria
    ● Any active or untreated thymoma. History of thymic carcinoma or thymic malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥5 years before Screening.
    ● History of thymectomy within 12 months prior to Screening.
    ● Weakness only affecting ocular or periocular muscles (MGFA Class I).
    ● Myasthenia Gravis crisis or impending crisis at or during Screening (MGFA Class V).
    ● Any unresolved acute, or chronic, systemic bacterial or other infection, which is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics.
    ● Unresolved meningococcal infection
    ● Patients who are under 15 kg and are receiving maintenance
    IVIg.
    ● For patients who are not receiving a stable maintenance dose of IVIg, use of IVIg (eg, as rescue therapy) within 4 weeks prior to first dose
    ● Use of PE within 4 weeks prior to first dose
    ● Use of rituximab within 6 months prior to first dose
    ● Hypersensitivity to murine proteins or to one of the excipients of eculizumab
    E.5 End points
    E.5.1Primary end point(s)
    Change from Baseline in the QMG total score over time regardless of rescue treatment
    E.5.1.1Timepoint(s) of evaluation of this end point
    26 Weeks
    E.5.2Secondary end point(s)
    - Change from Baseline in the MG-ADL total score over time regardless of rescue treatment
    - Proportion of patients with ≥ 3-point reduction in the MG-ADL total score over time with no rescue treatment
    - Proportion of patients with ≥ 3-point reduction in the MG-ADL total score over time regardless of rescue treatment
    - Proportion of patients with ≥ 5-point reduction in the QMG total score over time with no rescue treatment
    - Proportion of patients with ≥ 5-point reduction in the QMG total score over time regardless of rescue treatment
    - Change from Baseline in the MGC total score over time regardless of rescue treatment
    - Change from Baseline in EQ-5D-Y over time regardless of rescue treatment
    - Change from Baseline in Neuro-QoL Pediatric Fatigue over time regardless of rescue treatment
    - MGFA Post-Interventional Status over time
    - Total number and percentage of patients with clinical deteriorations, myasthenic crises, and rescue therapy use over time
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 26
    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 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 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.8.2.4Number of treatment arms in the trial1
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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
    Germany
    Japan
    Netherlands
    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
    Last Patient Last Visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days29
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 12
    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.1.1.5.1Number of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 12
    F.1.2Adults (18-64 years) No
    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 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
    Children to be enrolled. The parent or legal guardian will provide 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 4
    F.4.2.2In the whole clinical trial 12
    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)
    Patients will return to the care of his/her physician.
    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 Decision2020-12-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-13
    P. End of Trial
    P.End of Trial StatusOngoing
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