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    Summary
    EudraCT Number:2021-003302-50
    Sponsor's Protocol Code Number:ARGX-117-2002
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-11-19
    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 number2021-003302-50
    A.3Full title of the trial
    A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, Multicenter Trial to Evaluate the Safety and Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 2 Dose Regimens of ARGX-117 in Adults With Multifocal Motor Neuropathy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to investigate the safety and tolerability, efficacy pharmacokinetics, pharmacodynamics, and immunogenicity of 2 dose regimens of ARGX-117 in adults with multifocal motor neuropathy
    A.3.2Name or abbreviated title of the trial where available
    ARDA
    A.4.1Sponsor's protocol code numberARGX-117-2002
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05225675
    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 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 codeB-9052
    B.5.3.4CountryBelgium
    B.5.4Telephone number003293103400
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameARGX-117 IV
    D.3.2Product code ARGX-117 IV
    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 INNnot defined
    D.3.9.3Other descriptive nameARGX-117
    D.3.9.4EV Substance CodeSUB206651
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number120
    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 No
    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 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
    Multifocal Motor Neuropathy
    E.1.1.1Medical condition in easily understood language
    Multifocal Motor Neuropathy
    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 10065579
    E.1.2Term Multifocal motor neuropathy
    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 safety and tolerability of ARGX-117 compared to placebo in adult participants previously stabilized with IVIg
    E.2.2Secondary objectives of the trial
    - To evaluate the efficacy of ARGX-117 compared to placebo on muscle strength and/or motor function in adult participants previously stabilized with IVIg
    - To evaluate the efficacy of ARGX-117 on functional ability, arm and hand function, quality of life, and fatigue in adult participants with MMN
    - To evaluate the effect of ARGX-117 on health-related productivity and work productivity
    - To evaluate medication treatment satisfaction
    - To assess the PK, PD, and immunogenicity of ARGX-117
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol (including consent for the use and disclosure of research related health information). Participants must be able to read and write and be willing and able to comply with the trial protocol procedures (including attending the required trial visits)
    2. Male/female at least 18 years of age at the time the ICF is signed
    3. Probable or definite MMN according to the EFNS/PNS 2010 guidelines at screening confirmed by the MCC
    4.1. Receiving a stable IVIg regimen for at least 3 months before screening or recently initiated IVIg treatment (refer to inclusion criterion 5.1a) AND both of the following:
    a) IVIg treatment interval of 2 to 5 weeks
    b) IVIg dose of 0.4 to 2.0 g per kg body weight and infusion
    5.1. IVIg treatment dependency confirmation by the MCC at screening or after IVDP when applicable, based on 1 of the following:
    a) Recently initiated IVIg treatment (less than 3 months):
    - Clinical improvement following IVIg initiation documented in the participant’s medical record
    b) Maintenance therapy with IVIg (longer than 3 months), based on 1 of the following:
    - Clinical deterioration following IVIg withdrawal, IVIg dose reduction, or IVIg delayed administration within 12 months prior to screening (documented in the participant’s medical record)
    - Clinical deterioration following IVIg delayed administration during the IVDP
    6.2 Immunization with the first meningococcal vaccine and pneumococcal vaccine, and the single Haemophilus influenzae type B vaccine must be performed at least 14 days before IMP administration at V1 according to local country-specific immunization schedules. A documented history of vaccination against Neisseria meningitidis, Haemophilus influenzae type B, and Streptococcus pneumoniae will be permitted.
    • Germany only: Participants must be fully vaccinated against COVID-19 according to local country specific immunization schedules if required locally
    7.1 Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
    a) Male participants must agree to not donate sperm from the time the ICF is signed until 15 months after the last IMP administration
    b) Women of childbearing potential (WOCBP) 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
    1. Any coexisting condition which may interfere with the outcome assessments (eg, diabetic neuropathy, CIDP, inflammatory arthritis, or osteoarthritis affecting the hand)
    2. Clinical signs or symptoms suggestive for neuropathies other than MMN such as motor neuron disease (eg, bulbar signs or brisk reflexes) or other inflammatory neuropathies (eg, sensory neuropathy)
    3. Severe psychiatric disorder (such as severe depression, psychosis, bipolar disorder), history of suicide attempt, or current suicidal ideation that in the opinion of the investigator could create undue risk to the participant or could affect adherence with the trial protocol.
    4.2 Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection during the screening and/or IVMP.
    • Germany only: A SARS-CoV-2 rapid antigen test must be completed or a negative rapid antigen test not older than 48 hours (depending on local requirements) must be presented upon site entry during screening. Rapid antigen testing will only be mandatory while required locally.
    5. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of MMN or put the participant at undue risk (eg, SLE).
    6. History of malignancy unless resolved by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following carcinomas will be eligible:
    a. Adequately treated basal cell or squamous cell skin cancer
    b. Carcinoma in situ of the cervix
    c. Carcinoma in situ of the breast
    or
    d. Incidental histological finding of prostate cancer (TNM stage T1a or T1b)
    7.1 Clinical evidence of other significant serious diseases, have had a recent major surgery, (including a splenectomy at any time), or who have any other condition in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk
    8. Prior/concomitant therapy
    a. Cyclophosphamide and/or rituximab and/or eculizumab and/or mycophenolate mofetil within 3 months prior to screening
    b. Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP.
    9.1 Positive serum test at screening for an active viral infection with any of the following conditions:
    a. Hepatitis B virus (HBV) that is indicative of an acute or chronic infection (https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf)
    b. Hepatitis C virus (HCV) based on HCV antibody assay, confirmed by HCV RNA
    c. HIV based on test results that are associated with an AIDS-defining condition or a CD4 count <200 cells/mm3
    10. Current or history of (ie, within 12 months of screening) alcohol, drug, or medication abuse
    11. Known hypersensitivity reaction to 1 of the components of the IMP or any of its excipients
    12.1 Female participants with a positive serum or urine pregnancy test, lactating females, and those who intend to become pregnant during the trial or within 15 months after last dose of the IMP
    13. ALT or AST ≥2 × upper limit of normal and total bilirubin ≥1.5 × upper limit of normal of the central laboratory reference range, or any other clinically significant laboratory abnormality. These tests will be performed by the central laboratory
    14. An estimated glomerular filtration rate of ≤60 mL/min/1.73m2 calculated by the central laboratory using the 4-variable Modification of
    Diet in the Renal-Disease equation
    E.5 End points
    E.5.1Primary end point(s)
    Safety outcomes based on adverse event (AE) monitoring and other safety assessments
    E.5.1.1Timepoint(s) of evaluation of this end point
    Days 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113 + continuous monitoring (CM)
    E.5.2Secondary end point(s)
    A. Time to the first retreatment with IVIg since the final IVIg treatment of the IVIg monitoring period
    B. Time-to-relapse
    C. AUC of the change from baseline in mMRC-10 sum score
    D. Value and change from baseline in the average score of the 2 most important muscle groups as assessed by the mMRC-14 sum score
    E. Value and change from baseline in the mMRC-14 sum score
    F. Proportion of participants showing a deterioration of 1 or more points in at least 2 muscle groups as assessed by the mMRC-14 sum score
    G. Proportion of participants with no deterioration in 2 or more muscle groups as assessed by mMRC-14 sum score
    H. AUC of the change from baseline in GS
    I. Proportion of participants with a GS decrease of 8 kilopascal (kPa) or more over 3 consecutive days
    J. Values, change, and percent change from baseline in GS
    K. Values and change from baseline in the Rasch-built overall disability scale for MMN (MMN-RODS©)
    L Values and change from baseline in the average time for the upper extremity (arm and hand) function (9 Hole Peg Test [9-HPT], or timed pegboard test)
    M Proportion of participants by level of severity on each dimension of the EQ-5D-5L scale
    N Value and change from baseline in EQ-5D-5L visual analog scale (VAS)
    O Values and change from baseline in the chronic acquired polyneuropathy patient reported index (CAP-PRI)
    P Values of the Patient Global Impression Change (PGIC) scale
    Q Values and change from baseline in the 9-item Fatigue Severity Scale (FSS)
    R Values for work-related and household chore activities of the Health-Related Productivity Questionnaire (HRPQ) at each visit:
    − Hours lost because of absenteeism
    − Hours lost because of presenteeism
    − Total hours lost (absenteeism + presenteeism)
    − Percentage of scheduled hours lost because of absenteeism
    − Percentage of scheduled hours lost because of presenteeism
    − Percentage of scheduled hours lost in total (absenteeism +
    presenteeism)
    S Effectiveness, side effects, convenience, and overall satisfaction scores as assessed by the Treatment Satisfaction 14-Item Questionnaire for Medication (TSQM)
    T Serum concentrations and PK parameters for ARGX-117
    U Values and change from baseline in free C2, total C2, functional complement activity (CH50)
    V Incidence and prevalence of ADA against ARGX-117
    E.5.2.1Timepoint(s) of evaluation of this end point
    A & T: Days 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113
    B: Days 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113
    C, D, E, F, G, K, L: Days 1, 8, 15, 22, 29, 43, 57, 71, 85, 99 and 113
    H, I & J: Days 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113 + cm
    M, N, O & R: Days 1, 15, 29, 57, 85 and 113
    Q, P & S: Days 1, 15, 29, 43, 57, 71, 85, 99 and 113
    U: Days 1, 8, 15, 22, 29, 43, 57, 85 and 113
    V: Days 1 and 113
    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 No
    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 Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity, Quality of Life
    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) No
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA23
    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 Kingdom
    United States
    Austria
    Belgium
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    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.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    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: 36
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 12
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 32
    F.4.2.2In the whole clinical trial 48
    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 completing the double-blinded treatment period, participants who meet the inclusion and exclusion criteria of may enroll in a longterm extension study ARGX-117-2003, and receive ARGX-117.
    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 Decision2022-04-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-09-14
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2024-06-04
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