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    Summary
    EudraCT Number:2020-004032-21
    Sponsor's Protocol Code Number:ARGX-113-2004
    National Competent Authority:Romania - National Agency for Medicines and Medical Devices
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-05-25
    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 ConcernedRomania - National Agency for Medicines and Medical Devices
    A.2EudraCT number2020-004032-21
    A.3Full title of the trial
    A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and the Safety of Efgartigimod (ARGX-113) PH20 Subcutaneous 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 Phase 3 study to evaluate the efficacy and safety of efgartigimod PH 20 Subcutaneous in adult patients with primary immune thrombocytopenia.
    A.3.2Name or abbreviated title of the trial where available
    ADVANCE SC
    A.4.1Sponsor's protocol code numberARGX-113-2004
    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
    B.5.3.3Post codeB-9052
    B.5.3.4CountryBelgium
    B.5.4Telephone number+32 9 310 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/3/19/2230
    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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary immune thrombocytopenia (ITP)
    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] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 23.0
    E.1.2Level LLT
    E.1.2Classification code 10083843
    E.1.2Term Primary immune 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 efficacy of efgartigimod PH20 SC compared to placebo PH20 SC in achieving a sustained platelet count response in patients with chronic primary ITP, with a sustained platelet count response defined as platelet counts of ≥50×10^9/L for at least 4 of the 6 visits between week 19 and week 24 of the trial
    E.2.2Secondary objectives of the trial
    • To evaluate the efficacy of efgartigimod PH20 SC compared to placebo PH20 SC in overall platelet count response
    • To evaluate the incidence and severity of bleeding events while receiving treatment with efgartigimod PH20 SC compared to placebo PH20 SC
    •To evaluate the safety and tolerability of efgartigimod PH20 SC administered qw or every other week (q2w) compared to placebo PH20 SC
    • To evaluate the use of rescue treatment and changes in concurrent ITP therapy while receiving treatment with efgartigimod PH20 SC compared to placebo PH20 SC
    • To evaluate the effects of efgartigimod PH20 SC treatment on quality-of-life (QoL) measures and patient-reported outcomes (PRO) compared to placebo PH20 SC
    • To assess the immunogenicity of efgartigimod and rHuPH20
    • To assess the pharmacokinetics (PK) of efgartigimod PH20 SC
    • To assess the pharmacodynamic (PD) effects of efgartigimod PH20 SC
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Ability to understand the requirements of the trial and provide written informed consent (including consent for the use and disclosure of research-related health information), willing and able to comply with the trial protocol procedures (including attending the required trial visits)
    2. Male or female, aged ≥18 years at the time the informed consent form (ICF) is signed
    3. Confirmed diagnosis of primary ITP made at least 3 months before randomization and based on the American Society of Hematology Criteria, and no known etiology for thrombocytopenia
    4. Diagnosis supported by a response to a prior ITP therapy (other than TPO-RAs), in the opinion of the investigator
    5. Mean platelet count of <30×10^9/L from at least 3 documented, qualifying counts within the 3 preceding months where at least 2 of the qualifying counts must be taken during the screening period: 1 platelet count collected during the screening period and the predose platelet count on the day of randomization (visit 1). If the third count is not available from the 3 preceding months, this third platelet count can be obtained during the screening period.
    6. A documented history of a platelet count of <30×10^9/L before screening
    7. At the start of the trial, the participant either takes concurrent ITP treatment(s) and has received at least 1 prior therapy for ITP in the past, or the participant does not take treatment for ITP (see note) but has received at least 2 prior treatments for ITP. Participants receiving permitted concurrent ITP treatment(s) at baseline must have been stable in dose and frequency for at least 4 weeks before randomization.
    Permitted concurrent ITP medications include corticosteroids, danazol, vinca alkaloids, oral immunosuppressants, dapsone, fostamatinib, and/or oral TPO-RAs.
    Note: Participants not receiving concurrent ITP therapy are also eligible for the trial if they have not received prior ITP therapy for at least 4 weeks before baseline, and 6 months in case of prior ITP therapy with an anti-CD20 therapy (eg, rituximab).
    8. Women of childbearing potential:
    • As defined in Woman of Childbearing Potential in the protocol, women of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before trial medication can be administered
    • Must be on a stable regimen for at least 1 month of a highly effective or acceptable method of contraception (see Female Contraception in the protocol) during the trial and for 90 days after the last administration of IMP
    9. Non-sterilized male participants who are sexually active with a female partner of childbearing potential must use an acceptable method of contraception, ie, a condom (see Male Contraception) from signing the ICF through the last administration of the IMP. Male participants are also not allowed to donate sperm during this time.
    E.4Principal exclusion criteria
    1. Secondary ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, thrombocytopenia associated with myeloid dysplasia, or hematopoietic stem cell transplant
    2. Use of anticoagulants (eg, vitamin K antagonists, direct oral anticoagulants) within 4 weeks prior to randomization
    3. Use of any transfusions within 4 weeks prior to randomization
    4. Use of Ig (IV, SC, or intramuscular route) or plasmapheresis (PLEX) within 4 weeks prior to randomization
    5. Use of romiplostim within 4 weeks prior to randomization
    6. Undergone splenectomy less than 4 weeks prior to randomization
    7. Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP
    8. Use of any monoclonal antibody or Fc fusion proteins, other than those previously indicated, within 6 months before the first dose of the IMP (eg, anti-CD20)
    9. At the screening visit, clinically significant laboratory abnormalities as follows:
    • Hemoglobin ≤9 g/dL
    - OR –
    • International normalized ratio >1.5 or activated partial thromboplastin time >1.5×upper limit of normal
    - OR –
    • total IgG level <6 g/L
    10. History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of IMP. Participants with the following cancer can be included at any time:
    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)
    11. Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments
    12. History of any major thrombotic or embolic event (eg, myocardial infarction, stroke, deep venous thrombosis, or pulmonary embolism) within 12 months prior to randomization
    13. History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia
    14. 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
    15. 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 (unless associated with a negative HCV RNA test)
    c. Human immunodeficiency virus (HIV) based on test results that are associated with an acquired immunodeficiency syndrome (AIDS)-defining condition or a CD4 count <200 cells/mm3
    16. Known hypersensitivity reaction to efgartigimod, rHuPH20, or 1 of its excipients
    17. Previously participated in a clinical trial with efgartigimod and have received at least 1 administration of the IMP
    18. Pregnant or lactating females and those who intend to become pregnant during the trial or within 90 days after last dose of the IMP
    19. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
    20. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of ITP or put the participant at undue risk
    21. Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITP (eg, cardiovascular, pulmonary, hematologic, gastrointestinal, endocrine, hepatic, renal, neurological, malignancy, infectious diseases, uncontrolled diabetes) despite appropriate treatments which could put the participant at undue risk
    22. Current or history of (ie, within 12 months of screening) alcohol, drug, or medication abuse
    23. Received a live/live-attenuated vaccine less than 4 weeks before screening. The receipt of any inactivated, sub-unit, polysaccharide, or conjugate vaccine at any time before screening is not considered an exclusion criterion.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patients with chronic ITP with a sustained platelet count response defined as achieving platelet counts of ≥50×10^9/L for at least 4 of the 6 visits between week 19 and week 24 of the trial
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 5 weeks (between week 19 –24)
    E.5.2Secondary end point(s)
    1. Extent of disease control defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥50×10^9/L in the chronic ITP population
    2. Proportion of patients in the overall population (chronic and persistent ITP) with a sustained platelet count response defined as achieving platelet counts of ≥50×10^9/L for at least 4 of the 6 visits between week 19 and week 24
    3. Proportion of patients in the overall population achieving platelet counts of ≥50×10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial
    4. Proportion of patients in the overall population with overall platelet response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time during the 24-week treatment period
    5. Extent of disease control defined as the number of cumulative weeks until week 12 with platelet counts of ≥50×10^9/L in the overall population
    6. Proportion of patients in the overall population with overall platelet response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time until week 12
    7. Mean change from baseline in platelet count at each visit in the overall population
    8. Time to response defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L in the overall population
    9. The number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥30×10^9/L and ≥20×10^9/L above baseline in the overall population
    10. In patients with baseline platelet count of <15×10^9/L, the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥30×10^9/L and ≥20×10^9/L above baseline in the overall population
    11. Incidence and severity of the World Health Organization (WHO)-classified bleeding events in the overall population
    12. Incidence and severity of AEs, AEs of special interest (AESIs), and SAEs in the overall population
    13. Vital signs, ECG, and laboratory assessments in the overall population
    14. Rate of receipt of rescue therapy (rescue per patient per month) in the overall population
    15. Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have increased at week 12 or later in the overall population
    16. Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-Fatigue], Functional Assessment of Cancer Therapy questionnaire-Th6 [Fact-Th6]) and QoL (Short Form-36 [SF-36]) at planned visits in the overall population
    17. Incidence and prevalence of antibodies to efgartigimod and/or rHuPH20 in the overall population
    18. Titers of antibodies to efgartigimod and/or rHuPH20 in the overall population
    19. Presence of neutralizing antibodies (NAb) against efgartigimod and/or rHuPH20, and titers of NAb against efgartigimod and/or rHuPH20 in the overall population
    20. Serum efgartigimod concentration observed predose (Ctrough) in the overall population
    21. Pharmacodynamics markers: total IgG and antiplatelet antibody levels in the overall population
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Up to 24 weeks
    2. Up to 5 weeks (between week 19-24)
    3. Up to 7 weeks (between week 17-24)
    4. Up to 24 weeks
    5. Up to 12 weeks
    6. Up to 12 weeks
    7. Up to 35 weeks
    8. Up to 35 weeks
    9. Up to 24 weeks
    10. Up to 24 weeks
    11. Up to 35 weeks
    12.Up to 35 weeks
    13. Up to 35 weeks
    14. Up to 35 weeks
    15. Up to 23 weeks (between week 12-35)
    16. Up to 24 weeks
    17. Up to 35 weeks
    18. Up to 35 weeks
    19. Up to 35 weeks
    20. Up to 35 weeks
    21. Up to 35 weeks
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy 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
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA55
    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
    Argentina
    Australia
    Chile
    China
    Colombia
    Israel
    Japan
    Jordan
    Korea, Republic of
    Mexico
    New Zealand
    Peru
    South Africa
    Taiwan
    Thailand
    Tunisia
    United States
    France
    Latvia
    Poland
    Bulgaria
    Romania
    Spain
    Germany
    Greece
    Italy
    Denmark
    Georgia
    Ireland
    Norway
    Portugal
    Russian Federation
    Serbia
    Turkey
    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
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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: 136
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 49
    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)
    Participants completing the 24-week randomized trial period will perform the end-of-treatment visit and can enter the open-label extension trial (ARGX-113-2005).
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-06-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-29
    P. End of Trial
    P.End of Trial StatusOngoing
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