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    Summary
    EudraCT Number:2021-003302-50
    Sponsor's Protocol Code Number:ARGX-117-2002
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-11-26
    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 ConcernedItaly - Italian Medicines Agency
    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
    Sperimentazione di Fase 2, randomizzata, in doppio cieco, controllata con placebo, a gruppi paralleli, multicentrica per valutare la sicurezza e la tollerabilità, l’efficacia, la farmacocinetica, la farmacodinamica e l’immunogenicità di 2 regimi di dosaggio di ARGX-117 in adulti affetti da neuropatia motoria multifocale
    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
    Studio clinico per valutare la sicurezza e la tollerabilità, l’efficacia, la farmacocinetica, la farmacodinamica e l’immunogenicità di 2 regimi di dosaggio di ARGX-117 in adulti affetti da neuropatia motoria multifocale
    A.3.2Name or abbreviated title of the trial where available
    ARDA
    ARDA
    A.4.1Sponsor's protocol code numberARGX-117-2002
    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.2.1.2Country which granted the Marketing AuthorisationItaly
    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 injection/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.3.9.2Current sponsor codeARGX-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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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.IMP: 2
    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 Act-HiB
    D.2.1.1.2Name of the Marketing Authorisation holderSanofi Pasteur Europe
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 nameHaemophilus type b vaccine (conjugated)
    D.3.2Product code [.]
    D.3.4Pharmaceutical form Powder and suspension for suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor code-
    D.3.9.3Other descriptive nameHAEMOPHILUS TYPE B CONJUGATE VACCINE (TETANUS TOXOID CONJUGATE)
    D.3.9.4EV Substance CodeSUB14050MIG
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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.IMP: 3
    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 Intratect
    D.2.1.1.2Name of the Marketing Authorisation holderBiotest Pharma GmbH
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 nameHuman normal immunoglobulin (IVIg)
    D.3.2Product code [-]
    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 INNHuman normal immunoglobulin (IVIg)
    D.3.9.2Current sponsor code-
    D.3.9.3Other descriptive nameHUMAN NORMAL IMMUNOGLOBULIN (IV)
    D.3.9.4EV Substance CodeSUB12041MIG
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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.IMP: 4
    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 Menjugate
    D.2.1.1.2Name of the Marketing Authorisation holderGSK Vaccines S.r.l.
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 nameGroup C meningococcal conjugate vaccine
    D.3.2Product code [J07AH07]
    D.3.4Pharmaceutical form Suspension for injection in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNGroup C meningococcal conjugate vaccine
    D.3.9.2Current sponsor codeGroup C meningococcal conjugate vaccine
    D.3.9.3Other descriptive nameN. MENINGITIDIS GROUP C POLYSACCHARIDE
    D.3.9.4EV Substance CodeSUB26071
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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.IMP: 5
    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 Prevenar 13 sospensione per iniezione
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Europe MA EEIG
    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 namepneumococcal polysaccharide conjugate vaccine (13-valent, adsorbed)
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVACCINO PNEUMOCOCCICO SACCARIDICO CONIUGATO ADSORBITO
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive namePNEUMOCOCCAL POLYSACCHARIDE CONJUGATE VACCINE (ADSORBED)
    D.3.9.4EV Substance CodeSUB25302
    D.3.10 Strength
    D.3.10.1Concentration unit µl microlitre(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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.IMP: 6
    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 Pneumovax 23
    D.2.1.1.2Name of the Marketing Authorisation holderMSD SHARP & DOHME GMBH
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 namePneumococcal Polysaccharide Vaccine
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Solution for injection in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVACCINO ANTIPNEUMOCOCCICO
    D.3.9.2Current sponsor codeN/A
    D.3.9.3Other descriptive namePNEUMOCOCCAL POLYSACCHARIDE VACCINE (23 VALENT)
    D.3.9.4EV Substance CodeSUB14922MIG
    D.3.10 Strength
    D.3.10.1Concentration unit µl microlitre(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    Neuropatia Motoria Multifocale
    E.1.1.1Medical condition in easily understood language
    Multifocal Motor Neuropathy
    neuropatia motoria multifocale
    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
    Valutare la sicurezza e la tollerabilità di ARGX-117 rispetto al placebo in partecipanti adulti precedentemente stabilizzati con 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
    - Valutare l’efficacia di ARGX-117 rispetto al placebo sulla forza muscolare e/o sulla funzione motoria in partecipanti adulti precedentemente stabilizzati con IVIg
    - Valutare l’efficacia di ARGX-117 sull’abilità funzionale, sulla funzione del braccio e della mano, sulla qualità della vita e sulla stanchezza nei partecipanti adulti con MMN
    - Valutare l’effetto di ARGX-117 sulla produttività correlata alla salute e Sulla produttività lavorativa
    - Valutare la soddisfazione in merito al trattamento farmacologico
    - Valutare PK, PD e immunogenicità di 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
    at screening confirmed by the MCC
    3. Probable or definite MMN according to the EFNS/PNS 2010 guidelines
    at screening confirmed by the MCC
    4. Receiving a stable IVIg regimen before screening 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. IVIg treatment dependency confirmation by the MCC at screening or
    at IMV1, 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. Immunization with the first meningococcal vaccine and pneumococcal
    vaccine, and the single Haemophilus influenza 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 meningitides, Haemophilus influenza type
    B, and streptococcus pneumonia will be permitted
    7. 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 12 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
    1. Capacità di fornire il consenso informato firmato come descritto nella Sezione 10.1.3 dell’Appendice 1, che include la conformità ai requisiti e alle restrizioni elencati nel modulo di consenso informato (Informed Consent Form, [ICF]) e nel presente protocollo (compreso il consenso all’uso e alla divulgazione di informazioni sanitarie correlate alla ricerca). I partecipanti devono essere in grado di leggere e scrivere ed essere disposti e in grado di attenersi alle procedure del protocollo dello studio (inclusa la partecipazione alle visite dello studio richieste).
    2. Soggetti ambosesso di almeno 18 anni di età al momento della firma dell’ICF
    3. NMM probabile o accertata secondo le linee guida EFNS/PNS 2010 (Appendice 8: Sezione 10.8) allo screening, confermata dall’NCC
    4. Ricezione di un regime stabile con IVIg prima dello screening ed entrambi i seguenti punti:
    a. intervallo di trattamento con IVIg da 2 a 5 settimane;
    b. dose di IVIg da 0,4 a 2,0 grammi per kg di peso corporeo e infusione.
    5. Conferma della dipendenza dal trattamento con IVIg da parte dell’NCC allo screening o alla IMV1, sulla base di 1 dei seguenti:
    a. Trattamento con IVIg avviato di recente (meno di 3 mesi):
    - miglioramento clinico successivo all’avvio del trattamento con IVIg documentato nella cartella clinica del partecipante.
    b. Terapia di mantenimento con IVIg (di durata superiore a 3 mesi), in base a 1 dei seguenti:
    - deterioramento clinico in seguito a sospensione del trattamento con IVIg, riduzione della dose di IVIg o somministrazione ritardata di IVIg nei 12 mesi precedenti lo screening (documentata nella cartella clinica del partecipante);
    - deterioramento clinico in seguito al ritardo nella somministrazione di IVIg durante il periodo di dipendenza dal trattamento con IVIg (IVIg Dependency Period, [IVDP]).
    6. L’immunizzazione con il primo vaccino meningococcico e il vaccino pneumococcico e il singolo vaccino contro Haemophilus influenzae di tipo B devono essere eseguiti almeno 14 giorni prima della somministrazione dell’IMP alla V1 in base ai programmi di immunizzazione locali specifici per il Paese. Sarà consentita un’anamnesi documentata di vaccinazione contro Neisseria meningitides, Haemophilus influenzae di tipo B e polmonite da streptococco.
    7. L’uso di contraccettivi da parte di uomini e donne deve essere coerente con le normative locali riguardanti i metodi contraccettivi per coloro che partecipano a studi clinici.
    a. I partecipanti di sesso maschile devono acconsentire a non donare sperma dal momento della firma dell’ICF fino a 12 mesi dopo l’ultima somministrazione dell’IMP.
    b. Le donne in età fertile (Women Of Child Bearing Potential, [WOCBP]) (come definito nella Sezione 10.4.1.1 dell’Appendice 4) devono risultare negative a un test di gravidanza sierico allo screening e a un test di gravidanza sulle urine al basale prima che l’IMP possa essere somministrato.
    I requisiti contraccettivi per le WOCBP sono descritti nella Sezione 10.4.2 dell’Appendice 4).
    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. Clinically significant uncontrolled active or chronic bacterial, viral, or
    fungal infection during the screening and/or IVMP.
    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. Clinical evidence of other significant serious diseases, have had a
    recent major surgery, 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. 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
    c. HIV based on test results that are associated with an AIDS-defining
    1.Qualsiasi condizione coesistente che possa interferire con le valutazioni degli esiti (ad es., neuropatia diabetica, polineuropatia demielinizzante infiammatoria cronica [CIDP], artrite infiammatoria od osteoartrite a carico della mano)
    2.Segni o sintomi clinici indicativi di neuropatie diverse da NMM, come neuropatia motoria (per es., segni bulbari o riflessi attivi) o altre neuropatie infiammatorie
    3.Disturbo psichiatrico grave (quali forme gravi di depressione, psicosi, disturbo bipolare), anamnesi di tentativo di suicidio, o ideazione suicidaria in atto che, a giudizio dello sperimentatore, potrebbe comportare un rischio eccessivo per il partecipante o potrebbe compromettere l’aderenza al protocollo dello studio clinico.
    Nota: allo screening la suicidalità sarà valutata utilizzando la scala della Columbia University per la valutazione della gravità del rischio di suicidio; vedere Sezione 8.2.6.1); i partecipanti con un alto rischio di suicidio saranno esclusi dallo studio clinico (ovvero, saranno esclusi i partecipanti con risposta positiva alle domande n. 4 e/o n. 5 della sottoscala relativa all’ideazione suicidaria [negli ultimi 3 mesi]; e/o con qualsiasi risposta positiva alla sottoscala relativa al comportamento suicidario [durante l’ultimo anno]). Qualsiasi risposta positiva a queste domande sotto la dicitura “Nel corso della vita, periodo in cui il soggetto ha avuto più tendenze suicide” deve essere valutata attentamente per un eventuale rischio di suicidio attuale da parte dello sperimentatore prima dell’ingresso nello studio clinico.4.Infezione batterica, virale o fungina attiva o cronica non controllata clinicamente significativa durante lo screening e/o il periodo di monitoraggio con IVIg 5.Qualsiasi altra malattia autoimmune nota che, a giudizio dello sperimentatore, interferirebbe con una valutazione accurata dei sintomi clinici di NMM o esporrebbe il partecipante a un rischio inopportuno (per es. lupus eritematoso sistemico [LES]).
    6.Anamnesi di neoplasia a meno che non si sia risolta con un trattamento adeguato senza alcuna evidenza di recidiva per =3 anni prima della prima somministrazione dell’IMP. Saranno idonei i partecipanti con i seguenti carcinomi:
    a.tumore cutaneo basocellulare o squamocellulare adeguatamente trattato;
    b.carcinoma della cervice uterina in situ;
    c.carcinoma mammario in situ; o
    d.risultato istologico casuale di tumore prostatico
    7.Evidenza clinica di altre malattie gravi e significative, recente intervento di chirurgia maggiore o presenza di qualsiasi altra patologia che, a parere dello sperimentatore, potrebbe confondere i risultati della sperimentazione o esporre il partecipante a un rischio inopportuno.
    8.Terapia pregressa/concomitante
    a.Ciclofosfamide e/o rituximab e/o eculizumab e/o micofenolato mofetile nei 3 mesi precedenti lo screening
    b.Uso di prodotto sperimentale nei 3 mesi o nelle 5 emivite (a seconda di quale sia il periodo più lungo) precedenti alla prima dose dell’IMP
    9.Positività del test sierico per una infezione virale attiva allo screening con una qualsiasi delle seguenti condizioni:
    a.virus dell’epatite B (Hepatitis B Virus, [HBV]) che è indicativo di un’infezione acuta o cronica
    b.virus dell’epatite C (Hepatitis C Virus, [HCV]) in base al test degli anticorpi anti-HCV;
    c.virus dell’immunodeficienza umana (Human Immunodeficiency Virus, [HIV]) in base a risultati del test che sono associati a una condizione che definisce la sindrome da immunodeficienza acquisita (Acquired Immunodeficiency Syndrome, [AIDS]) o una conta dei CD4 <200 cellule/mm3.
    10.Anamnesi attuale o pregressa di abuso di alcol, farmaci o sostanze stupefacenti
    11.Nota reazione di ipersensibilità a 1 dei componenti dell’IMP o a uno qualsiasi dei suoi eccip
    12.Partecipanti di sesso femminile con test di gravid. su siero o urine positivo, donne in fase di allattamento e donne che intendono avviare una gravidanza durante la sperimentazione o entro 9 mesi dopo l’ultima dose dell’IMP
    E.5 End points
    E.5.1Primary end point(s)
    Safety outcomes based on adverse event (AE) monitoring and other
    safety assessments
    Esiti di sicurezza basati sul monitoraggio degli eventi avversi (EA) e altre valutazioni di sicurezza
    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)
    giorni 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 e 113 + continuo monitoraggio (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. Difference between the time to the first retreatment with IVIg (cycle)
    and the second time to retreatment with IVIg
    C. AUC of the change from baseline in mMRC-10 sum score
    D. Change from baseline in 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. Percentage of time 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 upper extremity (arm and hand)
    function (9-Hole Peg Test [9-HPT], or timed Peg Board Test)
    M. Values and change from baseline in quality of life using the Euro-
    Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) scale
    N. Values and change from baseline in the chronic acquired
    polyneuropathy patient-reported index (CAP-PRI)
    O. Values and change from baseline in the Patient Global Impression
    Change (PGIC) scale
    P. Values and change from baseline in the 9-item Fatigue Severity Scale
    (FSS)
    Q. Values and change from baseline in the Health-Related Productivity
    Questionnaire (HRPQ)
    R. Values and change from baseline in the Treatment Satisfaction 14-I
    tem Questionnaire for Medication (TSQM)
    S. Serum concentrations and PK parameters for ARGX-117
    T. Values and change from baseline in free C2, total C2, functional
    complement activity (CH50)
    U. Serum titer levels of binding antibodies (BAbs) against ARGX-117
    A. Tempo al primo ritrattamento con IVIg dal trattamento IVIg finale del periodo di monitoraggio IVIg
    B. Differenza tra il tempo al primo ritrattamento con IVIg (ciclo) e il secondo tempo al ritrattamento con IVIg
    C. AUC della variazione dal basale nel punteggio mMRC-10-sum
    D. Variazione rispetto al basale nei 2 gruppi muscolari più importanti valutato mediante il punteggio mMRC-14-sum
    E. Valore e variazione rispetto al basale nel punteggio mMRC-14-sum
    F. Percentuale di partecipanti che mostra un deterioramento di 1 o più punti in almeno 2 gruppi muscolari valutato mediante il punteggio mMRC 14-sum
    G. Percentuale di tempo senza deterioramento in 2 o più gruppi muscolari valutato mediante il punteggio mMRC-14-sum
    H. AUC della variazione dal basale nella GS
    I. Percentuale di partecipanti con una diminuzione della GS di 8 kilopascal (kPa) o più in 3 giorni consecutivi
    J. Valori, variazione e variazione percentuale rispetto al basale nella GS
    K. Valori e variazione rispetto al basale nella scala di disabilità complessiva di Rasch per MMN (MMN-RODS©)
    L. Valori e variazione rispetto al basale nella funzione degli arti superiori (braccio e mano) (9-Hole Peg Test [9-HPT] o Peg Board Test a tempo)
    M. Valori e variazione rispetto al basale della qualità della vita utilizzando la scala Euro-Quality of Life 5 Dimensions 5 Levels (EQ 5D 5L)
    N. Valori e variazione rispetto al basale dell’indice riferito dal paziente di polineuropatia cronica acquisita (CAP-PRI)
    O. Valori e variazione rispetto al basale nella scala Patient Global Impression Change (PGIC)
    P. Valori e variazione rispetto al basale nella Fatigue Severity Scale (FSS) a 9 item
    D. Valori e variazione rispetto al basale nel questionario sulla produttività correlata alla salute (HRPQ)
    R. Valori e variazione rispetto al basale nel Treatment Satisfaction 14 Item Questionnaire for Medication (TSQM)
    S. Concentrazioni sieriche e parametri farmacocinetici per ARGX-117
    T. Valori e variazione rispetto al basale di C2 libero, C2 totale, attività del complemento funzionale (CH50)
    U. Livelli sierici di anticorpi leganti (BAbs) contro ARGX-117
    E.5.2.1Timepoint(s) of evaluation of this end point
    vA & S: 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: 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
    K & L: Days 1, 8, 15, 22, 29, 43, 57, 71, 85, 99, and 113
    M, N & Q: Days 1, 15, 29, 57, 85 and 113
    O: Days 1, 15, 29, 43, 57, 71, 85, 99 and 113
    P: Days 1, 15, 29, 43, 57, 71, 85, 99 and 113
    R: Days 1, 15, 29, 43, 57, 71, 85, 99, 113
    T: Days 1, 8, 15, 22, 29, 43, 57, 85 and 113
    U: Days 1 and 113
    A & S: giorni 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 e113
    B: giorni 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 e 113
    C, D, E, F, G & P: Days 1, 4, 8, 15, 22, 29, 43, 57, 71, 85, 99 e 113

    H, I & J: giorni 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 e 113 +
    cm
    K & L: giorni 1, 4, 8, 15, 22, 29, 43, 57, 71, 85, 99, e 113
    M, N & Q: giorni 1, 15, 29, 57, 85 e 113
    O: giorni 1, 4, 15, 29, 43, 57, 71, 85, 99, 102 e 113
    R: giorni 1, 15, 29, 43, 57, 71, 85, 99, 113
    T: giorni 1, 8, 15, 22, 29, 43, 57, 85 e 113
    U: giorni 1 e 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
    immunogenicità, qualità della vita
    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 concerned3
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Austria
    Belgium
    Canada
    Denmark
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    United Kingdom
    United States
    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
    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 months8
    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.
    Dopo aver completato il periodo di trattamento in doppio cieco, i partecipanti che
    soddisfare i criteri di inclusione ed esclusione di può iscriversi a lungo termine
    studio di estensione ARGX-117-2003 e ricevere 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 Decision2022-02-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-15
    P. End of Trial
    P.End of Trial StatusOngoing
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