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    Summary
    EudraCT Number:2021-002479-20
    Sponsor's Protocol Code Number:80202135MYG2001
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2022-05-02
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2021-002479-20
    A.3Full title of the trial
    An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to less than 18 years with Generalized Myasthenia Gravis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    PK, PD, Safety and Activity of Nipocalimab in Children with Generalized Myasthenia Gravis
    A.4.1Sponsor's protocol code number80202135MYG2001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05265273
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/117/2020
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorJanssen-Cilag Invernational N.V.
    B.1.3.4CountryBelgium
    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 supportJanssen Research & Development, LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJanssen-Cilag International N.V.
    B.5.2Functional name of contact pointClinical Registry Group
    B.5.3 Address:
    B.5.3.1Street AddressArchimedesweg 29
    B.5.3.2Town/ cityLeiden
    B.5.3.3Post code2333
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31715242166
    B.5.5Fax number+31715242110
    B.5.6E-mailClinicalTrialsEU@its.jnj.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.3.1Product nameNipocalimab
    D.3.2Product code JNJ-80202135
    D.3.4Pharmaceutical form 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 INNNipocalimab
    D.3.9.1CAS number 2211985-36-1
    D.3.9.2Current sponsor codeJNJ-80202135
    D.3.9.3Other descriptive nameM281
    D.3.9.4EV Substance CodeSUB195356
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    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
    Myasthenia gravis (MG)
    E.1.1.1Medical condition in easily understood language
    Myasthenia gravis, a neuromuscular disease.
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    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 evaluate the efficacy, safety, tolerability, and pharmacokinetics of nipocalimab in children and adolescents (2 to 17) with gMG who have an insufficient clinical response to ongoing, stable standard-of-care therapy
    E.2.2Secondary objectives of the trial
    To evaluate the treatment response, effects on quality of life, fatigue and pharmacodynamics of nipocalimab in children and adolescents (2 to 17) with gMG who have an insufficient clinical response to ongoing, stable standard-of-care therapy
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Boy or girl 2 to <18 years of age.
    - Diagnosis of MG with generalized muscle weakness meeting the clinical criteria for gMG as defined by the MGFA Clinical Classification Class IIa/b, IIIa/b, or IVa/b at screening
    - Has a documented positive serologic test for acetylcholine receptor (anti-AChR) antibodies or muscle-specific tyrosine kinase (anti-MuSK) antibodies that will be confirmed at screening.
    - Has suboptimal response to current stable therapy(as defined in the protocol) for gMG according to the investigator.
    - Participants who have undergone splenectomy (if local regulatory authority has not requested exclusion of participants with splenectomy) must be at least 3 months post resection prior to screening and must be vaccinated as per the United States Center for Disease Control and Prevention annual Recommended Immunization Schedule for Adults Aged 19 Years or Older, United States OR must be vaccinated as per country-specific guidelines or local regulations. Note: This criterion is not applicable if splenectomy is excluded by the local authority.
    - Is recommended to be up to date on all age-appropriate vaccinations (eg diphtheria and tetanus) prior to screening as per routine local medical guidelines. For study participants who received locally approved (including emergency-use-authorized) Coronavirus 2019 (COVID-19) vaccine(s) recently prior to study entry, follow applicable local vaccine labeling, guidelines, and standards-of-care for patients receiving immune-targeted therapy when determining an appropriate interval between vaccination and study enrollment
    - A female of childbearing potential must have a negative highly sensitive serum at Screening and a negative urine pregnancy test at Day 1 prior to administration of study intervention.
    Additional Inclusion criteria are included in the protocol
    E.4Principal exclusion criteria
    - Has a history of severe and/or uncontrolled liver, gastrointestinal, renal, pulmonary, cardiovascular, psychiatric, neurological or musculoskeletal disorder or any other medical disorder including laboratory abnormalities, that, in the opinion of the Investigator, might interfere with participant's full participation in the study, or might jeopardize the safety of the participant or the validity of the study results.
    - Has any confirmed or suspected clinical immunodeficiency syndrome not related to treatment of his/her gMG, or has a family history of congenital or hereditary immunodeficiency unless confirmed absent in the participant.
    - Has MGFA Class I disease or presence of MG crisis (MGFA Class V) at screening, history of MG crisis within 1 month of screening, or fixed weakness (and/or ‘burnt out’ MG).
    - Is dependent on gastric tube for nutritional needs or is ventilator-dependent.
    - Is actively undergoing radiation or chemotherapy for an unresected thymoma/malignant thymoma. Participants with stable, benign thymoma (stage I or IIa, for example) for which no treatment has been undertaken in the past 3 years may be allowed following discussion with the sponsor’s medical monitor.
    - Has had a thymectomy within 12 months prior to screening, or thymectomy is planned during the Active treatment Phase of the study.
    - Has current or a history of any neurologic disorder other than MG that might interfere with the accuracy of study assessments, including but not limited to any chronic neurodegenerative disease, altered level of consciousness, dementia, abnormal mental status, major congenital neurologic defect, Lambert-Eaton myasthenic syndrome, drug induced MG, or hereditary forms of myasthenic syndrome.
    - Currently has a malignancy or has a history of malignancy within 3 years before screening (with the exception of localized basal cell carcinoma and/or squamous cell carcinoma skin cancer that has been adequately treated with no evidence of recurrence for at least 3 months [defined as a minimum of 12 weeks] before the first study intervention administration or cervical carcinoma in situ that has been treated with no evidence of recurrence for at least 3 months before the first study intervention administration).
    - Has known allergies, hypersensitivity, or intolerance to nipocalimab or its excipients (refer to the IB).
    - Has shown a previous severe immediate hypersensitivity reaction, such as anaphylaxis to therapeutic proteins (eg, monoclonal antibodies).
    - Has experienced myocardial infarction, unstable ischemic heart disease, or stroke within 12 weeks of screening.
    - History of moderate or severe substance or alcohol use disorder according to Diagnostic and Statistical Manual of Mental Disorders (5th edition) (DSM-5) criteria, except nicotine or caffeine, within 1 year before Screening.
    - Is currently taking IgG Fc-related protein therapeutics, or Fc-conjugated therapeutic agents, including factor or enzyme replacement.
    - Has received, rituximab within 6 months prior to first administration of study intervention.
    - Has received a live vaccine within 3 months prior to screening or has a known need to receive a live vaccine during the study, or within at least 3 months after the last administration of study intervention. Participants are allowed to receive a vaccine conditionally approved by their regional health advisory for emergency use for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), unless it is a live vaccine. Concomitant enrollment in an investigational trial for any SARS-CoV-2 (COVID-19) vaccine while participating in this study is not permitted.
    Additional Exclusion criteria are included in the protocol
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoints include:
    The effect of nipocalimab on total serum IgG in pediatric participants 2 to <18 years of age with gMG who have an insufficient clinical response to ongoing, stable standard-of-care therapy.
    The safety and tolerability of treatment with nipocalimab in pediatric participants 2 to <18 years of age with gMG who have an insufficient clinical response to ongoing, stable standardof- care therapy.
    The pharmacokinetics of nipocalimab in pediatric participants 2 to <18 years of age with gMG who have an insufficient clinical response to ongoing, stable standard of care therapy.
    All primary PK and IgG endpoints will be summarized descriptively over time for the evaluable population, and for each age cohort (2 to <12, or 12 to <18 years old).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the Active Treatment phase of the study. The maintenance of nipocalimab’s effect, as well as the long-term safety of nipocalimab will be further evaluated in the LTE phase.
    E.5.2Secondary end point(s)
    The secondary endpoints include:
    The activity of nipocalimab in gMG as measured by the change from baseline in Myasthenia Gravis –Activities of Daily Living (MG-ADL) efficacy score
    The activity of nipocalimab in gMG as measured by the change from baseline in Quantitative Myasthenia Gravis (QMG) efficacy score
    The effect on quality of life as measured by the European Quality of Life 5-Dimemsion Youth (EQ-5D-Y) tool
    The effect on fatigue as measured by the Neurological Quality of Life (Neuro-QoL) pediatric fatigue score
    All secondary endpoints will be summarized descriptively over time for the evaluable population, and for each age cohort (2 to < 12, or 12 to < 18 years old).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the Active Treatment phase of the study. he maintenance of nipocalimab’s effect, as well as the long-term safety of nipocalimab will be further evaluated in the LTE phase.
    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) Yes
    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.8.3 Will this trial be conducted at a single site globally? Yes
    E.8.4 Will this trial be conducted at multiple sites globally? No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Canada
    Japan
    United States
    Belgium
    France
    United Kingdom
    Netherlands
    Germany
    Italy
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months6
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    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)
    All Cohort 1 and Cohort 2 participants will have the option to enroll in the Long Term Extension (LTE) phase. LTE Phase will last until 2 years after marketing authorization in a participant's local country or until nipocalimab becomes available commercially or via other continued access program, whichever comes first. After that no further treatment and medical supervision of the patients after the end of the clinical trial is planned for the above clinical trial.
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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