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    Summary
    EudraCT Number:2020-005841-18
    Sponsor's Protocol Code Number:ARGX-113-2006
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-09-08
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2020-005841-18
    A.3Full title of the trial
    Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
    A.3.2Name or abbreviated title of the trial where available
    ADAPT JR
    A.4.1Sponsor's protocol code numberARGX-113-2006
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/097/2020
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of Sponsorargenx BV
    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 supportargenx BV
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationargenx BV
    B.5.2Functional name of contact pointRegulatory
    B.5.3 Address:
    B.5.3.1Street AddressIndustriepark Zwijnaarde 7
    B.5.3.2Town/ cityZwijnaarde (Ghent)
    B.5.3.3Post code9052
    B.5.3.4CountryBelgium
    B.5.6E-mailregulatory@argenx.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 Yes
    D.2.5.1Orphan drug designation numberEMA/OD/245/17
    D.3 Description of the IMP
    D.3.1Product nameefgartigimod
    D.3.2Product code argx-113
    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 INNEFGARTIGIMOD ALFA
    D.3.9.1CAS number 1821402-21-4
    D.3.9.2Current sponsor codeARGX-113
    D.3.9.4EV Substance CodeSUB198780
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    Generalized Myasthenia Gravis
    E.1.1.1Medical condition in easily understood language
    Myasthenia Gravis in patients aged 2 to 18 years who have generalized Muscle Weakness
    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 confirm an age-adjusted optimum dose of efgartigimod IV and provide (model-predicted) evidence for a treatment response
    E.2.2Secondary objectives of the trial
    -To evaluate the safety and tolerability of efgartigimod IV
    -To evaluate the PK and PD of efgartigimod IV
    -To evaluate the immunogenicity of efgartigimod IV
    -To evaluate the activity and impact on quality of life of efgartigimod IV
    -To evaluate the effect of efgartigimod treatment on antibody response to vaccines
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Participants are eligible to be included in the trial only if all of the following criteria apply:
    1. Ability of the participant and/or his/her legally authorized representative to understand the requirements of the trial and provide written informed consent/assent, if applicable (including consent/assent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including attending the required trial visits).
    2. Male or female participants between 2 to less than 18 years of age at the time of providing informed consent/assent. Age groups are enrolled in a staggered fashion respectively: 6 participants in the 12 to less than 18 years of age group followed by 6 participants in the 2 to less than 12 years of age group at the time of providing informed consent/assent.
    3. Diagnosed with gMG with confirmed documentation
    4. Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa.
    5. Eligible participants should have an unsatisfactory response (efficacy and/or safety) to immunosuppressants, steroids or AChE inhibitors and should be on stable concomitant gMG therapy of adequate duration before screening.
    6. Positive serologic test for anti-AChR antibodies at screening (for younger participants (<15kg) historical values can be used).
    7. Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical trials. A subject is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children and is sexually active.
    a. Male participants: Male participants must agree to not donate sperm from the time the ICF was signed until the end of the trial.
    b. Female participants:
    Female adolescents of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before IMP can be administered.
    E.4Principal exclusion criteria
    Participants are excluded from the trial if any of the following criteria apply:
    1. Participants with MGFA class I, IVb, and V.
    2. Female adolescents of childbearing potential (FAOCBP): Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of IMP.
    3. Has any of the following medical conditions:
    a) Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening.
    b) Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk.
    c) History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time:
    -Adequately treated basal cell or squamous cell skin cancer
    -Carcinoma in situ of the cervix
    -Carcinoma in situ of the breast
    -Incidental histological findings of prostate cancer (TNM Classification of Malignant Tumors stage T1a or T1b)
    d) Clinical evidence of other significant serious diseases, or have had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the trial or put the participant at undue risk.
    4. Worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoro-quinolones, beta-blockers, etc).
    5. A documented lack of clinical response to plasma exchange (PLEX).
    6. Received a live or live-attenuated vaccine fewer than 28 days before screening. Receiving an inactivated, subunit, polysaccharide, or conjugate vaccine any time before screening is not exclusionary.
    7. Received a thymectomy <3 months before screening or 1 is planned to be performed during the trial period.
    8. The following results from these diagnostic assessments will be considered exclusionary:
    a. Positive serum test at screening for an active viral infection with any of the following conditions:
    -Hepatitis B virus (HBV) that is indicative of an acute or chronic infection (https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf)
    -Hepatitis C virus (HCV) based on HCV antibody assay
    -Human immunodeficiency virus (HIV) associated with a CD4 count <200 cells/mm3 with an acquired immunodeficiency syndrome (AIDS)-defining condition, such as: Cytomegalovirus retinitis with loss of vision, Pneumocystis jiroveci pneumonia, chronic intestinal cryptosporidiosis, HIV-related encephalopathy, Mycobacterium tuberculosis (pulmonary or extrapulmonary), or invasive cervical cancer
    b. Positive nasopharyngeal swab PCR test for SARS-CoV-2 at screening .
    9. Using the following prior or concomitant therapies:
    a. Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP.
    b. Use of any monoclonal antibody within the 6 months before the first dose of the IMP.
    c. Use of IVIg, immunoglobulins administered SC or intramuscularly, or PLEX within 4 weeks before screening.
    10. Total IgG levels below the lower limit of normal (LLN) according to the reference ranges of the central laboratory for participant by sex and age at screening.
    11. A known hypersensitivity reaction to efgartigimod or any of its excipients.
    E.5 End points
    E.5.1Primary end point(s)
    - Efgartigimod concentrations as input for compartmental, model-driven analysis to determine (age and size dependency of) clearance (CL) and volume of distribution (Vd)
    - PD parameters: total IgG levels and anti-acetylcholine receptors antibodies (AChR-Ab) as input for PK/PD modeling analysis
    E.5.1.1Timepoint(s) of evaluation of this end point
    26 weeks
    E.5.2Secondary end point(s)
    -Incidence and severity of adverse events (AEs), incidence of serious adverse events (SAEs)
    - Efgartigimod serum concentrations
    -Levels of total IgG and anti-acetylcholine receptor antibodies (AChR-Ab): absolute values, change from baseline and percent (%) change from baseline
    -Incidence and prevalence of anti-drug antibodies (ADAs) against efgartigimod
    -MG-ADL: absolute value and change from baseline of total MG-ADL score
    -Total QMG score: absolute value and change from baseline of total QMG score
    -Total score EQ-5D-Y: absolute value and change from baseline
    -Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score: values and change from baseline
    -Change in protective antibody titers to vaccines received before or received during the trial
    E.5.2.1Timepoint(s) of evaluation of this end point
    2nd endpoint 1,4,8 = 28 weeks
    endpoint 2,3,5,6,7 = 26 weeks
    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 Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    - Dose confirmation for paediatric patients aged 2 to 18 years old.
    - Quality of Life
    - Tolerability
    - Biomarkers
    - Immunogenicity
    - Health Economics
    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 No
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    United States
    Georgia
    Austria
    Belgium
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    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 trial is defined as the date of the last visit of the last participant in the ARGX-113-2006 trial. A participant is considered to have completed the trial if he/she completed the end of trial (EoT) visit at day 182 ±3 days (trial entry in Part A) or day 126 ±3 days (trial entry in Part B), even if the participant did not complete treatment.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    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.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 22
    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)
    After the end of the trial participants will have the option to roll over into a long-term safety extension trial, ARGX-113-2008, to assess long-term safety and will be provided continued access until efgartigimod IV becomes commercially available or another option to access efgartigimod IV is available. Treatment may be continued based on investigator discretion provided that there is clear evidence of clinical benefit.
    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 Decision2021-11-16
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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