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    Summary
    EudraCT Number:2020-000949-14
    Sponsor's Protocol Code Number:VIB0551.P3.S1
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-12-08
    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 ConcernedGermany - PEI
    A.2EudraCT number2020-000949-14
    A.3Full title of the trial
    A RANDOMIZED, DOUBLE-BLIND, MULTICENTER,
    PLACEBO-CONTROLLED PHASE 3 STUDY WITH
    OPEN-LABEL PERIOD TO EVALUATE THE EFFICACY
    AND SAFETY OF INEBILIZUMAB IN ADULTS WITH
    MYASTHENIA GRAVIS
    Eine randomisierte, doppelblinde, multizentrische, placebokontrollierte Phase-III-Studie mit einer unverblindeten Phase zur Beurteilung der Wirksamkeit und Sicherheit von Inebilizumab bei Erwachsenen mit Myasthenia gravis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Inebilizumab efficacy and safety in adults with myasthenia gravis
    A.3.2Name or abbreviated title of the trial where available
    Myasthenia Gravis INebilizumab Trial (MINT)
    A.4.1Sponsor's protocol code numberVIB0551.P3.S1
    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 SponsorViela Bio, Inc. /Horizon Therapeutics Ireland DAC
    B.1.3.4CountryIreland
    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 supportViela Bio, Inc. / Horizon Therapeutics Ireland DAC
    B.4.2CountryIreland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationViela Bio, Inc. /Horizon Therapeutics Ireland DAC
    B.5.2Functional name of contact pointDave Caraher
    B.5.3 Address:
    B.5.3.1Street Address70 St. Stephen's Green
    B.5.3.2Town/ cityDublin 2
    B.5.3.3Post codeD02 E2X4
    B.5.3.4CountryIreland
    B.5.4Telephone number+35317722201
    B.5.6E-mailDCaraher@horizontherapeutics.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 Uplizna
    D.2.1.1.2Name of the Marketing Authorisation holderHorizon Therapeutics Ireland DAC
    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 nameInebilizumab
    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 INNINEBILIZUMAB
    D.3.9.2Current sponsor codeVIB0551
    D.3.9.3Other descriptive nameCD19-directed humanised afucosylated monoclonal antibody
    D.3.9.4EV Substance CodeSUB189141
    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 Yes
    D.3.11.13.1Other medicinal product typeInebilizumab is a CD19-directed humanised, afucosylated IgG1 monoclonal antibody.
    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
    Myasthenia Gravis which is either due to acetylcholine receptor antibodies (AChR) or muscle specific kinase antibodies (MuSK).
    E.1.1.1Medical condition in easily understood language
    Myasthenia gravis (MG) is a rare autoimmune disorder, caused by binding of antibodies to the acetylcholine receptor (AChR) or functionally related molecules on the postsynaptic neuromuscular junction.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    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 whether inebilizumab can reduce MG-related
    disability.
    E.2.2Secondary objectives of the trial
    - To evaluate whether inebilizumab can reduce the frequency of MG exacerbations.
    - To evaluate whether inebilizumab can improve MG-related quality of life.
    - To evaluate the safety and tolerability of inebilizumab in MG.
    - To characterize the pharmacokinetic (PK) profile and immunogenicity of inebilizumab in patients with MG.
    -To evaluate the effect of inebilizumab on corticosteroid usage.
    - To evaluate the ability of inebilizumab to elicit minimal symptom expression.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    B-cell sub-study:
    To better understand the effect of inebilizumab, B-cell repertoire profiling prior to and following inebilizumab treatment will be performed
    E.3Principal inclusion criteria
    1. Diagnosis of MG with anti-AChR or anti-MuSK antibody.
    2. MGFA Clinical Classification Class II, III, or IV.
    3.MG-ADL score at the time of screening and randomization between 6 and 10 with > 50% of this score attributed to non-ocular items, or an
    MG-ADL score ≥ 11.
    4.QMG score of ≥ 11 or greater at the time of screening and at randomization.
    5. Subjects must be on:

    a. Corticosteroids only, with no dose increase within 4 weeks prior to randomization, or
    b. One allowed non-steroidal IST, with continuous use for ≥ 6 months prior to randomization and no dose increase within 4 months prior to randomization, or
    c. Combination of (1) corticosteroids with no dose increase within 4 weeks prior to randomization and (2) one allowed nonsteroidal IST with continuous use for ≥ 6 months prior to randomization and no dose increase within 4 months prior to randomization.

    Allowed ISTs, alone or in combination with corticosteroids, are azathioprine, mycophenolate mofetil, and mycophenolic acid.

    9.. Females of childbearing potential who are sexually active with a non-sterilized male partner must use at least one highly effective contraception method from the time of screening and for 6 months after the final dose of IP. Periodic abstinence, the rhythm method, or the withdrawal method is not an acceptable methods of contraception.
    E.4Principal exclusion criteria
    8. Thymectomy within the 12 months prior to baseline (Day 1) visit or planned thymectomy during the duration of the RCP.
    9. Receipt of the following medications or treatments at any time prior to randomization:
    a. Alemtuzumab
    b. Total lymphoid irradiation
    c. Bone marrow transplant
    d. T-cell vaccination therapy
    e. Natalizumab
    10. Receipt of rituximab, ocrelizumab, ofatumumab, obinutuzumab, inebilizumab, or any experimental B-cell depleting agent within the 6 months prior to Day 1, unless the subject has a CD19+ B-cell count ≥ 40 cells/µL according to the central laboratory at screening.
    11. Receipt of Leflunomide within 1 year prior to Day 1.
    12. Receipt of the following within the 3 months prior to Day 1:
    a. Tocilizumab
    b. Belimumab
    c. Eculizumab
    d. Cyclophosphamide
    e. Ravulizumab
    f. Neonatal Fc receptor blockers (efgartigimod alfa)
    g. Abatacept
    h. Etanercept
    i. Mitoxantrone
    j. Sirolimus
    13. Receipt of the following within the 4 weeks prior to Day 1:
    a. Cyclosporine (except eye drops)
    b. Tacrolimus (except topical) (tacrolimus ≤ 3 mg/day is allowed in Japan only; see inclusion criterion 7C)
    c. Methotrexate
    d. Intravenous immunoglobulin (IVIg) or SC Ig
    e. PLEX treatment
    f. Thalidomide
    g. Tofacitinib

    2. Current use of:
    a. Corticosteroids (Prednisone > 40 mg/day or > 80 mg over a 2-day period, or equivalent dose of other corticosteroids
    b. Acetylcholinesterase inhibitors (pyridostigmine > 480 mg/day) or unstable dose in the 2 weeks prior to Day 1
    c. Azathioprine > 3 mg/kg/day
    d. Mycophenolate mofetil > 3 g/day or mycophenolic acid > 1440 mg/day
    e. Any IST, alone or in combination with corticosteroids, except for azathioprine, mycophenolate mofetil, and mycophenolic acid.
    15. Concurrent/previous enrollment in another clinical study involving an investigational treatment within 4 weeks or 5 half-lives of the investigational treatment, whichever is longer, prior to Day 1.
    16. Receipt of a live attenuated vaccine within 4 weeks prior to randomization. Administration of inactivated (killed) vaccines is acceptable.

    25. History of untreated hepatitis C infection, or positive antibody test for hepatitis C virus (HCV) unless patient is considered to be cured following antiviral therapy and has a HCV viral load below the limit of detection ≥ 24 weeks after completion of treatment at site or central lab

    30. Hospitalization for any reason < 30 days prior to randomization
    31. Current or recent myasthenia gravis exacerbationdeterioration that has not returned to baseline/resolved within at least 30 days prior to randomization.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Profile score at end of the RCP
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 26 over all study population ( AChR-Ab+ population and MuSK-Ab+ population)
    E.5.2Secondary end point(s)
    Key secondary endpoints:
    1. Change from baseline in Quantitative Myasthenia Gravis (QMG) scores at week 26 in the overall study population.
    2. Change from baseline in MG-ADL score at Week 26 in the AChR-Ab+ population.
    3. Change from baseline in QMG score at Week 26 in the AChR-Ab+ population.
    4. Change from baseline in MG-ADL score at Week 26 in the MuSK-Ab+ population.
    5. Change from baseline in QMG score at Week 26 in the MuSK-Ab+ population.

    Additional secondary endpoints:
    1. The Proportion of subjects with ≥ 3-point improvement in MG-ADL score at week 26 and no use of rescue therapy between Day 28 and Week 26 in
    the overall study population, the AChR-Ab+ population, and the MuSK-Ab+ population and at Week 52 and no use of rescue therapy between Day 28
    and Week 52 in the AChR-Ab+ population.
    2. Change from baseline in MG-ADL score at Week 52 in the AChR-Ab+ population.
    3. Change from baseline in QMG score at Week 52 in the AChR-Ab+ population.
    4. Change from baseline in Myasthenia Gravis Composite (MGC) score at Week 26 in the overall study population, the AChR-Ab+ population, and the MuSK-Ab+ population and at Week 52 in the AChR-Ab+ population.
    5. Change from baseline in Myasthenia Gravis Quality of Life-15, revised score at Week 26 in the overall study population, the AChR-Ab+ population, and the MuSK-Ab+ population and at Week 52 in the AChR-Ab+ population.
    6. Patient Global Impression of Change score at Week 26 in the overall study population, the AChR-Ab+ population, and the MuSK-Ab+ population and
    at Week 52 in the AChR-Ab+ population.
    7. Time to first MG exacerbation by Week 26 in the overall study population, the AChR-Ab+ population, and the MuSK-Ab+ population and by Week 52 in the AChR-Ab+ population.
    8. The safety and tolerability of inebilizumab as measured by the incidence of treatment-emergent adverse events, adverse events of special interest,
    and treatment-emergent serious adverse events. Laboratory measurements will also be evaluated as part of safety.

    9. The proportion of subjects with steroid tapered to ≤ 5 mg/day at Week 26 for the overall study population, the AChR-Ab+ population, and the MuSK-Ab+ population and at Week 52 for the AChR-Ab+ population.
    10. The proportion of subjects in whom steroid dose was reduced by ≥ 50% by Week 26 for the overall study population, the AChR-Ab+ population, and the MuSK-Ab+ population and by Week 52 for the AChR-Ab+ population.
    11. The proportion of subjects achieving minimal symptom expression, defined as MG-ADL = 0 or 1, at Week 26.
    12. Anti-drug antibody status and titer during the study in the overall study population, the AChR-Ab+ population, and the MuSK-Ab+ population.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 52 for AChR-Ab+ population and Week 26 for over all population (AcHR-Ab+ and MuSK-Ab+ population)
    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 Yes
    E.8.1.7.1Other trial design description
    The second part of trial is optional and open label
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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
    Ukraine
    Taiwan
    Belarus
    Brazil
    Canada
    China
    India
    Israel
    Japan
    Korea, Republic of
    Russian Federation
    United Kingdom
    United States
    Denmark
    France
    Germany
    Italy
    Poland
    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 study will be complete when the last active subject completes the Day 547 visit of the OLP, discontinues the OLP, or (for patients who have opted out of the OLP) completes the RCP.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years7
    E.8.9.2In all countries concerned by the trial months0
    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: 210
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 126
    F.4.2.2In the whole clinical trial 230
    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)
    None
    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 Decision2021-02-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-07-30
    P. End of Trial
    P.End of Trial StatusOngoing
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