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    EudraCT Number:2016-003038-26
    Sponsor's Protocol Code Number:ARGX-113-1603
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-12-08
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedGermany - PEI
    A.2EudraCT number2016-003038-26
    A.3Full title of the trial
    A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients with Primary Immune Thrombocytopenia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of the safety and effectiveness of ARGX-113 in Patients with Primary Immune Thrombocytopenia
    A.4.1Sponsor's protocol code numberARGX-113-1603
    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.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.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, Building C
    B.5.3.2Town/ cityZwijnaarde
    B.5.3.3Post codeB-9052
    B.5.4Telephone number+3293103400
    B.5.5Fax number+3293103499
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameARGX-113
    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 INNN/A
    D.3.9.1CAS number 1821402-21-4
    D.3.9.2Current sponsor codeARGX-113
    D.3.9.3Other descriptive nameARGX-113
    D.3.9.4EV Substance CodeSUB180001
    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
    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
    Primary Immune Thrombocytopenia
    E.1.1.1Medical condition in easily understood language
    Primary Immune Thrombocytopenia
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10074678
    E.1.2Term Primary immune thrombocytopenic purpura
    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 safety and tolerability of ARGX-113 (short and long term)
    To evaluate the safety of repeated use of ARGX-113
    E.2.2Secondary objectives of the trial
    To evaluate the patients with initial response i.e., platelet count ≥ 30 × 109/L and/or at least doubling of the Baseline count and absence of bleeding.
    - To evaluate the clinical effect of ARGX-113 ARGX-113 (short term and long term) on:
    - platelet counts;
     - use of rescue treatment;
     - bleeding events.
    - To evaluate the efficacy of repeated use of ARGX-113.
    - To assess the effect of ARGX-113 on quality of life.
    - To assess the PK of ARGX-113.
    - To assess the PD effects of ARGX-113.
    - To evaluate the immunogenicity of ARGX-113.

    Specifically for the extended FU period:
    To assess the duration of the treatment effect for all patients who did
    not relapse during the treatment period. This will be based on:
    - local platelet counts;
    - use of rescue treatment;
    - bleeding events.To assess the PK of ARGX-113.
    To assess the PD effects of ARGX-113.
    To evaluate the immunogenicity of ARGX-113.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Main study
    1. Ability to understand the requirements of the study, and comply
    withthe study protocol procedures (including required study visits).
    2. Male or female patients aged ≥ 18 to ≤ 85 years.
    3. Eligible patients must receive standard-of-care treatment for ITP
    following the ASH guidelines and International Working Group (IWG)
    stable in dose and frequency for at least 4 weeks prior to Screening.
    4. Confirmed diagnosis of ITP according to the American Society of
    Hematology Criteria 2011 with (average) blood platelet counts < 30 ×
    10E9/L and who have not experienced major bleeding in the last 4
    weeks prior to Screening.
    5. Women of childbearing potential must have a negative serum
    pregnancy test at Screening and a negative urine pregnancy test at
    Baseline prior to administration of IMP.
    6. Female participants of childbearing potential must agree to use a
    highly effective method of birth control (i.e., pregnancy rate of less than
    1% per year) during the study and for 90 days after the discontinuation
    of IMP.
    7. Non-sterilized male patients who are sexually active with a female
    partner of childbearing potential must use effective double contraception
    Additional Inclusion Criteria for the Extended Follow-up Period
    - Sign the amended ICF of the main study including its extended
    - Completed Visit 16 of the FU period of the protocol with a platelet
    count ≥ 30 × 10E9/L and/or at least doubling of the Baseline platelet
    count and absence of bleeding and remained on the same SoC
    Eligibility criteria for the open-label treatment period (first treatment
    1. Please refer to inclusion criteria 5, 6 and 7 from the main study.
    2. Provide written informed consent
    3. Received at least 3 doses of the IMP and had at least 2 weeks of
    follow-up in the main study.
    4. Patient is at the same SoC as in the main study. Dose and/or
    frequency increase is allowed, changing or stopping the SoC is not
    5. During up to 21 weeks of FU, the patient is relapsing i.e. platelet count
    decreases to below 30 x 10E9/L or the patient's platelets never went up
    to 30 x 10E9/L and are still below 30 x 10E9/L, and absence of bleeding.
    Eligibility criteria for subsequent open-label retreatment cycles
    The patient has the right to receive more than 1 retreatment cycle if:
    1. Patient reached a platelet count of at least twice the platelet count
    measured on the day of the first IMP administration during the previous
    (re)treatment cycle, confirmed on at least 2 separate consecutive
    occasions (at least 1 day apart but with maximum 7 days in between the
    2 measurements), and measured during the treatment period up to
    minimum 4 weeks of follow-up.
    2. Patient received at least 3 doses of the IMP and had at least 4 weeks
    follow-up in the previous treatment cycle.
    3. Patient is at the same SoC as in the previous treatment cycle. Dose
    and/or frequency increase is allowed, changing or stopping the SoC is
    not allowed.
    4. Patient is relapsing i.e. platelet count decreases to below 30 x 10E9/L
    or the patient's platelets never went up to 30 x 10E9/L and are still
    below 30 x 10E9/L, and absence of bleeding.
    E.4Principal exclusion criteria
    1. Use of anticoagulants, or any drug with antiplatelet effect (e.g., acetylsalicylic acid [aspirin] or other salicylate containing medications, cyclooxygenase inhibitors, adenosine diphosphate (ADP) receptor inhibitors, phosphodiesterase inhibitors, glycoprotein IIB/IIIA inhibitors, adenosine reuptake inhibitors, thromboxane inhibitors, antimalarials, and prostacyclins) during the study and within 3 weeks prior to Screening.
    2. Patients who have received any blood support or transfusion within 4 weeks prior to Screening.
    3. Use of IVIg or anti-D immunoglobulin treatment within 4 weeks prior to Screening.
    4. Use of recombinant thrombopoietin at any time.
    5. Use of rituximab within 6 months prior to Screening. Use of any anti-CD20 other than rituximab at any time is not permitted.
    6. Use of corticosteroids which has not been stable for at least 4 weeks prior to Screening.
    7. Use of immunosuppressants is not permitted within 4 weeks prior to Screening, with the exception of the following oral immunosuppressants: azathioprine [up to 2.5 mg/kg/day], danazol [up to 15 mg/kg/day], mycophenolate mofetil [up to 3 g/day], mycophenolate sodium [up to 2160 mg/day]) which must have been stable for at least 4 weeks prior to Screening.
    8. Use of any other biological therapy or investigational drug than those previously indicated within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening.
    9. Received vaccinations within 4 weeks prior to Screening or planned during the study.
    10. At Screening, have clinically significant laboratory abnormalities given as below:
    a. Aspartate aminotransferase (AST) or ALT > 3 × upper limit of normal (ULN).
    b. Total serum bilirubin of > 1.5 x ULN (except for Grade 1 hyperbilirubinemia as defined by the National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE], due solely to a documented medical diagnosis of Gilbert’s syndrome).
    c. Serum creatinine of > 1.5 mg/dL or creatinine clearance < 50 mL/min (using Chronic Kidney Disease Epidemiology - Creatinine formula).
    d. Hemoglobin ≤ 9 g/dL.
    e. Thyroid stimulating hormone or thyroglobulin outside of the central laboratory normal range.
    f. International normalized ratio (INR) > 1.5 or activated partial thromboplastin time > 1.5 × ULN.
    g. Total immunoglobulin G (IgG) level < 6 g/L.
    h. Presence of > 1+ proteinuria
    Note: A patient with isolated laboratory values that meet the thresholds listed above may be considered eligible. The agreed upon medical rationale, discussed between the investigator and sponsor, must be documented in the patient's chart prior to patient enrolment in the study
    11. History of myeloproliferative or lymphoproliferative disorders at any time; or have a history of malignancy at any time unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 5 years prior to Screening. Patients with completely excised nonmelanoma skin cancers or cervical carcinoma in situ would be permitted at any time.
    12. History of cerebrovascular accident or myocardial infarction within the last 12 months, before Screening, or current severe/unstable angina, arrhythmia, or at risk of ventricular arrhythmia, symptomatic congestive heart failure (New York Heart Association Class III or IV), or uncontrolled hypertension (Grade 3 to 4 CTCAE). Uncontrolled hypertension is defined as a repeated elevation in the blood pressure exceeding 140 mmHg and over 90 mmHg despite appropriate treatments.
    13. History of any thrombotic or embolic event within 12 months prior to Screening.
    14. History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia.
    15. Known history or symptoms of systemic lupus erythematosus, antiphospholipid antibody syndrome or any other clinically documented
    auto-immune disease other than ITP.
    16. Prior history or symptoms suggestive of untreated Helicobacter pylori infection (recurrent nausea, vomiting, stomach burn, excessive burping, feeling bloated, and unexplained weight loss).
    17. History of a recent major surgery (that involve major organs e.g., brain, heart, lung liver, bladder, or gastrointestinal tract) within 4 weeks of Screening or that in the opinion of the investigator may compromise patient’s participation.
    18. Active infection; a recent serious infection (i.e., requiring injectable antimicrobial therapy or hospitalization) within the 8 weeks prior to Screening;
    Known history or known infection with viral infection with human immunodeficiency virus (HIV 1 and 2 antibodies). Known seropositive or active infection with hepatitis C virus (HCV) hepatitis. Known active or chronic viral infection with hepatitis B virus (HBV). Patients must have a negative TB Quantiferon test at Screening. Patients with an undetermined Quantiferon TB result will be allowed one retest, if not negative on retesting, the patient will be excluded.
    Please refer to the protocol on page 41 for the listed points 19 to 23.
    E.5 End points
    E.5.1Primary end point(s)
    -Incidence and severity of of treatment-emergent AEs (TEAEs) and serious AEs (SAEs).
    - Changes from Baseline (mean, median, minimum and maximum values, shifts) in vital signs, electrocardiogram parameters (ECGs), physical examination abnormalities, and clinical laboratory assessments.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Various time points throughout the study
    E.5.2Secondary end point(s)
    -Frequency and proportion of patients with initial response i.e., platelet count ≥ 30 × 109/L and/or at least doubling of the Baseline count and absence of bleeding at any time during the study.
    - Mean change in platelet counts compared to Baseline.
    -Frequency and proportion of patients with following response at any time during the study 5,1:
    o Complete response (CR): Platelet count ≥ 100×109/L, confirmed on at least 2 separate consecutive occasions ≥ 7 days apart, and the absence of bleeding;
    o Response (R): Platelet count ≥ 30 and < 100×109/L, and a greater than 2-fold increase in platelet count from Baseline, confirmed on at least 2 separate consecutive occasions ≥ 7 days apart, and the absence of bleeding;
    o No response (NR): Platelet count < 30 × 109/L or less than doubling of the Baseline count or bleeding;
    -Time to initial response: time from starting treatment to time to reach CR or R;
    -Duration of response: time from the achievement of CR or R to loss of CR or R;
    -Frequency and proportion of patients with response to ≥ 50×10E9/L:
    Platelet count increase to at least ≥ 50×10E9/L at any time during the
    - Frequency and proportion of patients requiring rescue therapy.
    -General bleeding assessment by the World Health Organization (WHO)
    bleeding scale and SMOG index of the ITP specific bleeding assessment
    tool (ITP-BAT)*.
    - Change from Baseline in the Short Form-36 item Health Status
    Questionnaire (SF-36) and the Functional Assessment of Cancer Therapy
    - PK parameters of ARGX-113 including maximum observed
    concentration (Cmax), time of maximum concentration (tmax), plasma
    concentration prior to dosing (Ctrough), apparent terminal half-life
    (t1/2,λz), and accumulation ratio (Rac).*
    - Evaluation of pharmacodynamic markers*: Total IgG, IgG isotypes**
    IgG1, IgG2, IgG3, IgG4; and antiplatelet antibody levels. In addition,
    IgA, IgD, IgE, and IgM will also be assessed**.
    - Evaluate the incidence of antidrug antibodies (ADA) to ARGX-113*.
    *not for the extended FU period (except WHO bleeding scale)
    **IgG isotypes and IgA, IgA, IgE, IgM will not be assessed in the
    treatment period
    E.5.2.1Timepoint(s) of evaluation of this end point
    Various time points throughout the study
    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 Yes
    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 Yes
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Long term efficacy
    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) 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 No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E. trial design description
    open label treatment after main study
    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 trial3
    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 EEA35
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years2
    E.8.9.1In the Member State concerned months0
    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 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: 22
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 14
    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 36
    F.4.2.2In the whole clinical trial 36
    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)
    None. Subject will continue with routine care.
    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 Decision2017-03-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-04-27
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-04-09
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