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    Summary
    EudraCT Number:2019-003107-35
    Sponsor's Protocol Code Number:ARGX-113-1902
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2020-05-01
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2019-003107-35
    A.3Full title of the trial
    Open-label Extension of the ARGX-113-1802 Trial to Investigate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
    Een open-label uitbreiding van het ARGX-113-1802-onderzoek om de
    veiligheid, verdraagbaarheid en doeltreffendheid van efgartigimod PH20 SC
    op lange termijn te onderzoeken bij patiënten met chronische
    inflammatoire demyeliniserende polyneuropathie (CIDP)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the long-term safety and efficacy of a subcutaneous formulation of efgartigimod in adults with chronic inflammatory demyelinating polyneuropathy (an autoimmune disorder that affects the peripheral nerves)
    Een studie om de veiligheid en doeltreffendheid op lange termijn te
    ondezoeken van een onderhuidse toediening van efgartigimod bij
    volwassenen met chronische inflammatoire demyeliniserende
    polyneuropathie (een autoimmuun ziekte dat de perifere zenuwen aantast)
    A.3.2Name or abbreviated title of the trial where available
    ADHERE+
    CIDP Studie 1902 (ADHERE+)
    A.4.1Sponsor's protocol code numberARGX-113-1902
    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 BVBA
    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 BVBA
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationargenx BVBA
    B.5.2Functional name of contact pointRegulatory
    B.5.3 Address:
    B.5.3.1Street AddressIndustriepark Zwijnaarde 7
    B.5.3.2Town/ cityZwijnaarde
    B.5.3.3Post codeB-9052
    B.5.3.4CountryBelgium
    B.5.4Telephone number00329310 3400
    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 numberEU/3/21/2555
    D.3 Description of the IMP
    D.3.1Product nameEfgartigimod PH20 SC
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous 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 number165
    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.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 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 numberEU/3/21/2555
    D.3 Description of the IMP
    D.3.1Product nameEfgartigimod PH20 SC
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEFGARTIGIMOD ALFA
    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 number180
    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
    Chronic Inflammatory Demyelinating Polyneuropathy
    Chronicsche Inflammatoire Demyelinating Polyneuropathie
    E.1.1.1Medical condition in easily understood language
    Inflammation of peripheral nervous system
    Ontsteking aan het perifere zenuwstelsel
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10077384
    E.1.2Term Chronic inflammatory demyelinating polyneuropathy
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the long-term safety and tolerability of efgartigimod PH20 SC (efgartigimod co-formulated with recombinant human hyaluronidase PH20 [rHuPH20] for subcutaneous [SC] administration)
    E.2.2Secondary objectives of the trial
    - To determine the long-term efficacy
    - To evaluate the immunogenicity (antidrug antibodies [ADA] against efgartigimod during the first 48 week treatment cycle [followed by a safety follow-up period, if applicable])
    - To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC (during the first 48 week treatment cycle [followed by a safety follow-up period, if applicable])
    - To evaluate the pharmacodynamic (PD) effect of efgartigimod PH20 SC (ie, total immunoglobulin G [IgG] levels)
    - To evaluate additional patient-reported outcomes (PROs) (including patient-reported quality of life and satisfaction with treatment)
    - To explore self-administration of the treatment
    - To explore administration of the treatment by caregivers.

    Note that there are other objectives for patients with less frequent than weekly dosing of efgartigimod PH20 SC which are described in Section 11.9. of the protocol.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Optional Dosing Frequency Substudy:
    The optional dosing frequency substudy offers patients who are stabilized on weekly dosing for at least 12 weeks the option to receive 2 less frequent efgartigimod dosing regimens sequentially (see separate protocol appendix for a dosing frequency substudy).
    E.3Principal inclusion criteria
    1. Ability to understand the requirements of the trial, provide written informed consent (including consent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including required trial visits) of this trial.

    2. a. Male or female patient with 1 of the following options:
    - Have completed the Week-48 visit of Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC; or
    - Have deteriorated during Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC, or
    - Have been offered the participation in the OLE trial due to early termination of the ARGX-113-1802 trial (because sufficient events for the primary endpoint analysis of the that trial have been reached and it is stopped) and are considered to be eligible for treatment with efgartigimod PH20 SC treatment; or
    - Have completed the Week-48 visit of the previous cycle of the OLE trial and are considered to be eligible to continue with efgartigimod PH20 SC treatment.

    Optional Dosing Frequency Substudy: Note that after a minimum period of weekly dosing in this OLE trial, as specified in a separate protocol appendix for a dosing frequency substudy, patients who have a stable clinical condition for at least 12 weeks will be offered the option to receive less frequent dosing to evaluate the maintenance of the clinical condition by administration of efgartigimod PH20 SC 1000 mg at 2 lower dosing frequencies.

    3. Women of childbearing potential who have a negative urine pregnancy test at baseline before IMP administration.

    4. a. Women of childbearing potential must use an acceptable method of contraception from signing the ICF until the date of the last dose of IMP

    5. Inclusion criterion removed via protocol amendment #4.
    E.4Principal exclusion criteria
    1. Week-48/ED visit in the ARGX-113-1802 trial or the Week-48 visit of the previous OLE participation occurred more than 14 days prior to SD1 of the OLE trial or the start of a new treatment cycle in the OLE trial and more than 21 days since the last dose of IMP.

    2. a. Pregnant and lactating women and those intending to become pregnant during the trial.

    3. a. Patients with clinical evidence of other significant serious disease or patients who underwent a recent or have a planned major surgery, or patients who (intend to) use prohibited medications and therapies during the trial, or any other reason which could confound the results of the trial or put the patient at undue risk.
    E.5 End points
    E.5.1Primary end point(s)
    - Incidence of TEAEs and SAEs by system organ class (SOC) and preferred term (PT).
    - Incidence of clinically significant laboratory abnormalities.
    E.5.1.1Timepoint(s) of evaluation of this end point
    TEAEs and SAEs are monitored throughout the entire study duration
    Blood sampling for laboratory analysis is taken at every study visit
    E.5.2Secondary end point(s)
    Efficacy;
    Change from baseline over time in the following scores and measurements:
    − Adjusted INCAT score;
    − MRC Sum score;
    − 24-item I-RODS disability scores;
    − Mean grip strength assessed by Martin vigorimeter;
    − TUG score.
    Percentage of patients without clinical deterioration over time, defined by adjusted INCAT increase ≥1 point compared to baseline.

    Immunogenicity;
    - Percentage of patients with and titers of binding antibodies (BAb) towards efgartigimod and/or rHuPH20; and the presence of neutralizing antibodies (NAb) against efgartigimod and titers of NAb against rHuPH20.

    Pharmacokinetics;
    - Efgartigimod serum concentrations (during the first 48-week treatmentcycle [followed by a safety follow-up period, if applicable]).

    Pharmacodynamics;
    - Changes from baseline over time of serum IgG levels (total)

    Additional Patient-reported Outcome;
    Change from baseline over time in:
    − Health-related quality-of-life questionnaire (EQ-5D-5L);
    − Brief Pain Inventory – Short Form (BPI-SF);
    − 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9);
    − Rasch-transformed-Fatigue Severity Scale (RT-FSS);
    − Hospital Anxiety and Depression Scale (HADS).

    Percentage of patients performing self-administration over time.

    Percentage of patients with treatment administered by caregiver over time.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy, immunogenicity, pharmacokinetic and pharmacodynamic endpoints are assessed at every study visit
    Patient report outcomes are assessed at every study visit with the exception of visit 2 (week 4)
    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 No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity
    immunogeniteit
    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 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.8.2.4Number 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA61
    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
    China
    Israel
    Japan
    Taiwan
    United States
    Georgia
    Russian Federation
    Turkey
    Ukraine
    Serbia
    Belgium
    Bulgaria
    Czechia
    Denmark
    France
    Germany
    Hungary
    Italy
    Latvia
    Netherlands
    Poland
    Romania
    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
    LVLS
    Laatste visite laatste patient
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 216
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 144
    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 155
    F.4.2.2In the whole clinical trial 360
    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, patients will no longer receive IMP and will be treated as considered appropriate by the patient's treating physician
    Na einde studie, ontvangen patienten niet langer IMP en zullen ze door
    de behandelend arts zo goed mogelijk behandeld worden.
    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 Decision2020-05-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-09-25
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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