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    Summary
    EudraCT Number:2022-000460-21
    Sponsor's Protocol Code Number:ALXN1720-MG-301
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-11-18
    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 ConcernedAustria - BASG
    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.
    Eine randomisierte, doppelblinde, placebokontrollierte, parallele, multizentrische Phase-3-Studie zur Bewertung der Sicherheit und Wirksamkeit von ALXN1720 bei Erwachsenen mit generalisierter Myasthenia Gravis.
    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.
    Sicherheit und Wirksamkeit von ALXN1720 bei Erwachsenen mit generalisierter Myasthenia gravis
    A.3.2Name or abbreviated title of the trial where available
    Safety and Efficacy of ALXN1720 in Adults with Generalized Myasthenia Gravis.
    Sicherheit und Wirksamkeit von ALXN1720 bei Erwachsenen mit generalisierter Myasthenia gravis
    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 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 INNgefurulimab
    D.3.9.2Current sponsor codeALXN1720
    D.3.9.3Other descriptive nameHumanised VHH-type bispecific antibody against 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 placeboSolution 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
    Generalisierte Myasthenia gravis
    E.1.1.1Medical condition in easily understood language
    Generalized Myasthenia Gravis
    Generalisierte 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
    To assess the efficacy of ALXN1720 compared with placebo in the treatment of gMG based on change in MG-ADL total score
    Bewertung der Wirksamkeit von ALXN1720 im Vergleich zu Placebo bei der Behandlung von gMG basierend auf der Änderung des MG-ADL Gesamtscores
    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
    Bewertung der Wirksamkeit von ALXN1720 im Vergleich zu Placebo bei der Behandlung von gMG basierend auf der Änderung des QMG Gesamtscores
    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.
    - Muss zum Zeitpunkt der Unterzeichnung der Einverständniserklärung ≥ 18 Jahre alt sein
    - Diagnose von MG mit generalisierter Muskelschwäche, die die von der Myasthenia Gravis Foundation of America (MGFA) Klasse II, III oder IV definierten klinischen Kriterien erfüllt
    - Positiver serologischer Test auf Autoantikörper gegen 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.
    - Anamnese einer Thymektomie oder einer anderen Thymusoperation innerhalb der letzten 12 Monate vor dem Screening
    - Unbehandelte bösartige Thymus Erkrankung, Karzinom oder Thymom
    - Anamnese einer Infektion mit Neisseria meningitidis
    - Schwangerschaft, Stillzeit oder beabsichtige Empfängnis während der klinischen Prüfung.
    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
    Veränderung des Myasthenia gravis-Aktivitäten des täglichen Lebens (MG-ADL) Gesamtscores in Woche 26 gegenüber dem Ausgangswert
    E.5.1.1Timepoint(s) of evaluation of this end point
    At Week 26
    zu Woche 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
    - Veränderung des quantitativen Myasthenia gravis (QMG)-Gesamtscores gegenüber dem Ausgangswert in Woche 26
    - Prozentsatz der Responder basierend auf der Reduktion des MG-ADL Gesamtscores in Woche 26
    - Prozentsatz der Responder basierend auf der Reduktion der QMG Gesamtpunktzahl in Woche 26
    - Änderung des Myasthenia gravis Composite (MGC)-Gesamtscores gegenüber dem Ausgangswert in Woche 26
    E.5.2.1Timepoint(s) of evaluation of this end point
    At week 26
    zu Woche 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA50
    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
    Switzerland
    Taiwan
    Brazil
    Canada
    China
    Israel
    Japan
    Korea, Republic of
    Serbia
    United Kingdom
    United States
    Austria
    Denmark
    France
    Germany
    Italy
    Netherlands
    Poland
    Portugal
    Spain
    Türkiye
    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.
    Das Ende der Studie ist definiert als das Datum des letzten Besuchs des letzten Studienteilnehmers oder die letzte geplante Prozedur für einen Studienteilnehmer wie in der "Schedule of Activities" gezeigt
    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 months1
    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 months11
    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: 228
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 26
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 85
    F.4.2.2In the whole clinical trial 254
    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 their treating physician at the
    completion of study participation.
    Am Ende der Studie erhalten die Teilnehmer die Prüfmedikation nicht mehr und erhalten weiterhin ihre Standardbehandlung
    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 Decision2023-01-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-02-09
    P. End of Trial
    P.End of Trial StatusOngoing
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