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    Summary
    EudraCT Number:2022-000460-21
    Sponsor's Protocol Code Number:ALXN1720-MG-301
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-07-25
    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 ConcernedFrance - ANSM
    A.2EudraCT number2022-000460-21
    A.3Full title of the trial
    A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel, Multicenter Study to Evaluate the Safety and Efficacy of ALXN1720 in Adults with Generalized Myasthenia Gravis.
    Une étude de phase 3, randomisée, en double aveugle, contrôlée par placebo, parallèle et multicentrique pour évaluer l'innocuité et l'efficacité de l'ALXN1720 chez les adultes atteints de myasthénie grave généralisée
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy of ALXN1720 in Adults with Generalized Myasthenia Gravis.
    Innocuité et efficacité de l'ALXN1720 chez les adultes atteints de myasthénie grave généralisée
    A.3.2Name or abbreviated title of the trial where available
    Safety and Efficacy of ALXN1720 in Adults with Generalized Myasthenia Gravis.
    Innocuité et efficacité de l'ALXN1720 chez les adultes atteints de myasthénie grave généralisée
    A.4.1Sponsor's protocol code numberALXN1720-MG-301
    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 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+331 47 10 06 15
    B.5.5Fax number+331 47 10 06 11
    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 No
    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.3.2Product code ALXN1720
    D.3.4Pharmaceutical form Concentrate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot available
    D.3.9.2Current sponsor codeALXN1720
    D.3.9.3Other descriptive nameHumanised VHH-type bispecific antibody that binds to Complement component 5 and serum albumin
    D.3.9.4EV Substance CodeSUB219394
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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 Yes
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboConcentrate for solution for injection
    D.8.4Route of administration of the placeboSubcutaneous 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
    Myasthénie grave généralisée
    E.1.1.1Medical condition in easily understood language
    Generalized Myasthenia Gravis
    Myasthénie grave généralisée
    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
    To assess the efficacy of ALXN1720 compared with placebo in the treatment of gMG based on change in MG-ADL total score
    Évaluer l’efficacité d’ALXN1720 par rapport au placebo dans le traitement de la MGg en se basant sur le changement du score total MG-ADL
    E.2.2Secondary objectives of the trial
    To assess the efficacy of ALXN1720 compared with placebo in the treatment of gMG based on change in QMG total score
    Évaluer l’efficacité d’ALXN1720 par rapport au placebo dans le traitement de la MGg en se basant sur le changement du score total QMG
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Must be ≥ 18 years of age at the time of signing the informed consent
    - Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV
    - Positive serological test for autoantibodies against AChR.
    - Doit être âgé d’au moins 18 ans au moment de la signature du consentement éclairé
    - Diagnostic documenté de MGg avec une faiblesse musculaire généralisée de II à IV en accord avec les critères cliniques définis par la Myasthenia Gravis Foundation of America (MGFA)
    - Test sérologique positif pour les auto-anticorps dirigés contre l’AChR
    E.4Principal exclusion criteria
    - History of thymectomy or any other thymic surgery within 12 months prior to Screening
    - Untreated thymic malignancy, carcinoma, or thymoma
    - History of Neisseria meningitidis infection
    - Pregnancy, breastfeeding, or intention to conceive during the course of the study.
    - Antécédents de thymectomie ou de toute autre opération du thymus dans les 12 mois précédant la sélection
    - Toute tumeur maligne thymique, carcinome ou thymome non traité
    - Antécédents d’infection à Neisseria meningitidis
    - Grossesse, allaitement ou intention de concevoir un enfant pendant l’étude.
    E.5 End points
    E.5.1Primary end point(s)
    Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 26
    variation du score total MG-ADL entre la visite de référence et la Semaine 26
    E.5.1.1Timepoint(s) of evaluation of this end point
    At Week 26
    Semaine 26
    E.5.2Secondary end point(s)
    - Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Week 26

    -Percentage of Responders Based on Reduction of the MG-ADL Total Score at Week 26

    -Percentage of Responders based on Reduction of the QMG Total Score at Week 26

    - Change From Baseline in Myasthenia Gravis Composite (MGC) Total Score at Week 26
    - variation du score total QMG entre la visite de référence et la Semaine 26

    - pourcentage de répondeurs basé sur la réduction du score total MG-ADL à la Semaine 26

    - pourcentage de répondeurs basé sur la réduction du score total QMG à la Semaine 26

    - variation du score total MGC entre la visite de référence et la Semaine 26
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 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 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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA65
    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
    Australia
    Brazil
    Canada
    China
    Israel
    Japan
    Korea, Republic of
    Taiwan
    United States
    Austria
    France
    Poland
    Netherlands
    Spain
    Switzerland
    Czechia
    Germany
    Italy
    Belgium
    Denmark
    Hungary
    Portugal
    Serbia
    Turkey
    United Kingdom
    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
    The end of the study is defined as the date of the last visit of the last participant in the study, or the last scheduled procedure shown in the SoAs for the last participant in the study.
    La fin de l'étude est définie comme la date de la dernière visite du dernier participant de l'étude, ou de la dernière procédure prévue comme indiqué dans les calendriers des activités pour le dernier participant de l'étude.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months8
    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: 180
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 90
    F.4.2.2In the whole clinical trial 200
    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 the end of the study, participants will no longer receive the study treatment and will continue to receive their standard treatments.
    A la fin de l'étude, les participants ne recevront plus le traitement à l'étude et continueront à recevoir leurs traitements standards.
    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 Decision2022-11-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-11-10
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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