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    EudraCT Number:2019-002101-21
    Sponsor's Protocol Code Number:ARGX-113-1803
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-03-03
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2019-002101-21
    A.3Full title of the trial
    A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A long-term study to assess the safety and efficacy of efgartigimod in adult patients with primary immune thrombocytopenia (an autoimmune disorder that destructs platelets, blood cells that help with clotting, and can lead to easy or excessive bruising and bleeding)
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberARGX-113-1803
    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.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.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
    B.5.3.3Post codeB-9052
    B.5.4Telephone number+329310 3400
    B.5.5Fax number+329310 3499
    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/3/19/2230
    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
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Primary immune thrombocytopenia
    E.1.1.1Medical condition in easily understood language
    Disorder that can lead to easy or excessive bruising and bleeding due to low levels of the cells that help blood clot
    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 23.0
    E.1.2Level LLT
    E.1.2Classification code 10050245
    E.1.2Term Autoimmune thrombocytopenia
    E.1.2System Organ Class 100000004851
    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 long-term safety of efgartigimod in adult patients with primary immune thrombocytopenia (ITP).
    E.2.2Secondary objectives of the trial
    First 52-week treatment period only:
    • To evaluate the long-term efficacy of efgartigimod on overall platelet count response.
    • To explore the potential for reduction in concurrent ITP therapy.
    • To evaluate the effects of efgartigimod treatment on quality-of-life (QoL) measures and patient-reported outcomes (PRO).
    • To evaluate the incidence and severity of bleeding events while receiving treatment with efgartigimod.
    • To evaluate the use of rescue treatment while receiving tretment with efgartigimod.
    • To assess the pharmacodynamic (PD) effects of efgartigimod.
    • To evaluate the pharmacokinetics (PK) of efgartigimod.
    First 52-week treatment period and additional 52-week treatment periods:
    • To assess the immunogenicity of efgartigimod.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).
    2. Patients enrolled in the ARGX-113-1801 trial who completed the 24-week trial period.
    3. Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered. Women are considered of childbearing potential unless they are post-menopausal (defined by continuous amenorrhea) for at least 1 year with a FSH of >40 IU/L or are surgically sterilized (ie, women who had a hysterectomy, a bilateral salpingectomy, both ovaries surgically removed, or have a documented permanent female sterilization procedure including tubal ligation). Follicle-stimulating hormone can be used to confirm post-menopausal status in amenorrheic patients not on hormonal replacement therapy.
    4. Women of childbearing potential should use a highly effective or acceptable method of contraception during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month:
    • combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation
    - oral
    - intravaginal
    - transdermal
    • progestogen-only hormonal contraception associated with inhibition of ovulation:
    - oral
    - injectable
    - implantable
    • intrauterine device (IUD)
    • intrauterine hormone-releasing system
    • bilateral tubal occlusion
    • vasectomized partner(provided that the partner is the sole sexual partner of the trial participant and that aspermia was documented post procedure)
    • continuous abstinence from heterosexual sexual contact. Sexual abstinence is only allowable if it is the preferred and usual lifestyle of the patient. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not acceptable.
    • male or female condom with or without spermicide
    • cap, diaphragm, or sponge with spermicide
    5. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use an acceptable method of contraception, ie, condom. Male patients practicing true sexual abstinence (when this is in line with the preferred and usual lifestyle of the participant) can be included. Sterilized male patients who have had a vasectomy with documented aspermia post procedure can be included. In addition, male patients are not allowed to donate sperm during this period from signing of informed consent form, throughout the duration of the trial, and for 90 days after the last administration of IMP.

    In addition to the above criteria, for patients who want to continue receiving efgartigimod during an additional 52-week treatment period (only applicable in case efgartigimod is not yet commercially available for patients with primary ITP, or becomes available through another patient program for patients with primary ITP)
    6. Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research- related health information), and to comply with the trial protocol procedures (including required trial visits)
    7. Patient has completed a 52-week treatment period.
    E.4Principal exclusion criteria
    1. Introduction or continuation of non-permitted medications during the ARGX-113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins or live/live-attenuated vaccines).
    2. Pregnant or lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.
    3. Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod.
    4. Use of any other investigational drug or participation in any other investigational trial.
    E.5 End points
    E.5.1Primary end point(s)
    Frequency and severity of AEs, vital signs, and laboratory assessments.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Frequency and severity of AEs - at each visit
    Vital signs - at baseline visit (week 1), followed by every even week (week 2 to week 52) and Early discontinuation, follow-up 1 and 2, unscheduled visits.
    Laboratory assessments - please refer to protocol Table 1 Schedule of Assessments for various laboratory assessments
    E.5.2Secondary end point(s)
    First 52-week treatment period only:
    1. Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10^9/L.
    2. Percentage of patients with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time during the 52-week treatment period.
    3. Mean change from baseline in platelet count at each visit.
    4. For patients rolling-over from the ARGX-113-1801 trial with a platelet count of <30×10^9/L: time to response is defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L.
    5. The percentage of weeks in the trial with platelet counts of ≥30×10^9/L and at least 20×10^9/L above baseline.
    6. In patients with baseline platelet count of <15×10^9/L in the current trial (ARGX-113-1803), the percentage of weeks in the trial with platelet counts of ≥30×10^9/L and at least 20×10^9/L above baseline.
    7. In patients with first exposure to efgartigimod: proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial.
    8. In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50×10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial.
    9. Rate of receipt of rescue therapy (rescue per patient per month).
    10. Reduction in concurrent ITP therapy.
    11. Incidence and severity of the WHO-classified bleeding events.
    12. Change from baseline in PRO (FACIT-Fatigue, Fact-Th6) and QoL (SF-36) at planned visits.
    13. Pharmacokinetic parameter of efgartigimod: serum concentration observed predose (Ctrough).
    14. Pharmacodynamics markers: total IgG.
    First 52-week treatment period and additional 52-weeek treatment periods:
    15. Incidence of anti-drug antibodies (ADA) to efgartigimod.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Over the 52-week treatment period
    2. Over the 52-week treatment period
    3. At each visit
    4. At each visit
    5. Over the 52-week treatment period
    6. Over the 52-week treatment period
    7. Between week 19 and 24
    8. Between week 17 and 24
    9. At each visit
    10. At each visit
    11. At each visit
    12. PRO: Visits 1, 5, 9, 13, 17, 21, 25, 33, 41, 49, 52 of Treatment period, Early Discontinuation
    QoL: Weeks 1, 9, 17, 25, 33, 41, 49, 52 of Treatment period, Early Discontinuation
    13 and 14. Visits 1, 5, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 of Treatment period, Early Discontinuation, Follow-up 1/2, Unscheduled Visit
    First 52-week treatment period and additional 52-week treatment periods:
    15. At planned visits per schedule of assessments in the protocol
    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 of efgartigimod
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number 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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA75
    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
    Russian Federation
    United States
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    E.8.9.2In all countries concerned by the trial months8
    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: 140
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 16
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 140
    F.4.2.2In the whole clinical trial 156
    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 a patient has completed the trial or has withdrawn/discontinued early, usual treatment will be administered if required, in accordance with the trial sites' standard of care and generally accepted medical practice depending on the patient's individual needs.
    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 Decision2020-04-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-28
    P. End of Trial
    P.End of Trial StatusCompleted
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