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    Summary
    EudraCT Number:2011-000241-21
    Sponsor's Protocol Code Number:BEL114870
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2011-12-07
    Trial results View 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 ConcernedGermany - PEI
    A.2EudraCT number2011-000241-21
    A.3Full title of the trial
    A clinical and mechanistic proof of efficacy study with belimumab in chronic immune thrombocytopenia (ITP) patients
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate belimumab for the treatment of chronic immune thrombocytopenia
    A.4.1Sponsor's protocol code numberBEL114870
    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 SponsorGlaxoSmithKline Research & Development Limited
    B.1.3.4CountryUnited Kingdom
    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 supportGlaxoSmithKline Research & Development Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGlaxoSmithKline
    B.5.2Functional name of contact pointGSK Clinical Support Helpdesk
    B.5.3 Address:
    B.5.3.1Street AddressIron Bridge Road, Stockley Park West
    B.5.3.2Town/ cityUxbridge, Middlesex
    B.5.3.3Post codeUB11 1BT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44(0)208990 4466
    B.5.5Fax number+44(0)208990 4968
    B.5.6E-mailGSKClinicalSupportHD@gsk.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 Yes
    D.2.1.1.1Trade name BENLYSTA® (belimumab)
    D.2.1.1.2Name of the Marketing Authorisation holderGlaxo Group Limited
    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 namebelimumab
    D.3.2Product code GSK1550188
    D.3.4Pharmaceutical form Powder 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 INNbelimumab
    D.3.9.1CAS number 356547-88-1
    D.3.9.2Current sponsor codeGSK1550188
    D.3.9.3Other descriptive nameLymphostat B
    D.3.9.4EV Substance CodeSUB25607
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    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 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) Yes
    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 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
    Chronic Immune Thrombocytopenia
    E.1.1.1Medical condition in easily understood language
    Immune thrombocytopenia (ITP)
    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 15.1
    E.1.2Level LLT
    E.1.2Classification code 10051057
    E.1.2Term Idiopathic 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
    The Primary Objective :
    To evaluate whether belimumab can demonstrate a clinical response in ITP, using a surrogate clinical measure of platelet count, to clarify mechanism ofaction of belimumab.
    E.2.2Secondary objectives of the trial
    - To evaluate whether belimumab can modulate anti-platelet
    autoantibodies in subjects with detectable baseline levels of these antibodies
    - To evaluate the safety and tolerability of belimumab 10mg/kg in ITP
    - To assess the pharmacokinetics of belimumab 10mg/kg in ITP
    - To evaluate whether belimumab can modulate antigen specific lymphocyte responses, general serum autoantibody levels, serum cytokine/chemokine levels and transcriptomic profiling.
    - To evaluate whether any demonstrated modulation of B cells and the immune system can, through selected pharmacodynamic (PD) assays, be directly linked to clinical response such that the PD assays could be used as biomarkers
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A subject will be eligible for inclusion in this study only if all of the following criteriaapply:
    1. Age & Gender: Male or female between 18 and 75 years of age inclusive, at the time of signing the informed consent.
    2. Disease severity: Diagnosis of chronic ITP for a minimum of 6 months (12 months for Germany) with a platelet count of <=75,000/µL (<=50,000/µL for Germany) at screening and a historical platelet count of <=75,000/µL (<=50,000/µL for Germany) 2 to 6 months prior to screening.
    3. Platelet autoantibody status: Subjects with platelet auto-antibody +ve by MAIPA. Subsequently, if decision is made after one of the 2 planned interim analyses, subjects with platelet auto-antibody +ve by PIFT (i.e. MAIPA –ve, PIFT +ve). Following a separate decision after one of the 2 planned interim analyses, autoantibody negative subjects (MAIPA –ve, PIFT –ve) may be recruited into a separate open-labelled belimumab cohort.
    4. ITP Treatment: ITP patients stable either on no treatment or on a stable dose of corticosteroids (10 mg/day prednisone or prednisone equivalent or less) and/or AZA
    (100mg/day or less) or on MMF (2 g/day or less) for a minimum of 30 days before screening.
    5. ECG: Single QTc, < 450 msec; or QTc < 480 msec in subjects with Bundle Branch Block.
    6. Female Subjects: A female subject is eligible to participate if she is not pregnant or nursing and at least one of the following conditions apply: a. Non-childbearing potential defined as pre-menopausal females with a documented tubal ligation or hysterectomy; or postmenopausal defined as 12
    months of spontaneous amenorrhea [in questionable cases a blood sample with simultaneous follicle stimulating hormone (FSH) > 40 MlU/ml and estradiol < 40 pg/ml (<147 pmol/L) is confirmatory]. Females on hormone replacement therapy (HRT) and whose menopausal status is in doubt will be required to use one of the contraception methods in Section 8.1.1 if they wish to continue their HRT during the study. Otherwise, they must discontinue HRT to allow confirmation of post-menopausal status prior to study
    enrollment. For most forms of HRT, at least 2-4 weeks will elapse between the cessation of therapy and the blood draw; this interval depends on the type and dosage of HRT. Following confirmation of their post-menopausal status,
    they can resume use of HRT during the study without use of a contraceptive method.
    b. Child-bearing potential and agrees to use one of the contraception methods listed in Section 8.1.1 for one month (2 weeks for abstinence) prior to the start of dosing to sufficiently minimize the risk of pregnancy at that point.
    Female subjects must agree to use contraception until 16 weeks after the last dose.
    7. Informed Consent: Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the consent form.
    E.4Principal exclusion criteria
    1. Secondary ITP: If the diagnosis of ITP is secondary to other conditions.
    2. B-cell Prior Therapy: Have received treatment with any B cell targeted therapy at any time.
    3. 364 Day Prior Therapy: Have received any of the following within 364 days prior to Day 0:
    • Abatacept.
    • A biologic investigational agent other than B cell targeted therapy.
    4. 180 Day Prior Therapy: Have received any of the following within 180 days prior to Day 0:
    • Intravenous (IV) cyclophosphamide.
    • 3 or more courses of systemic corticosteroids for concomitant conditions (eg, asthma, atopic dermatitis. Topical or inhaled steroids are permitted).
    5. 90 Day Prior Therapy: Have received any of the following within 90 days prior to Day 0:
    • High dose corticosteroid for treatment of ITP (> 100 mg/day prednisone or equivalent).
    • Splenectomy, plasmapheresis.
    6. 60 Day Prior Therapy: Have received any of the following within 60 days, 5 halflives or twice the duration of the biological effect of the investigational product (whichever is longer) prior to Day 0:
    • A non-biologic investigational agent.
    • Any other immunosuppressive/immunomodulatory agent (e,g, ciclosporin A, dapsone, vincristine) with the exception of AZA, corticosteroids and MMF.
    • Eltrombopag, romiplostim.
    • Any steroid injection (intramuscular, intraarticular or intravenous).
    Note: Inhaled steroids and Topical immunosuppressive agents (e.g. eye drops, topical creams) are allowed.
    7. 30 Day Prior to Screening Therapy: Have received any of the following within 30 days prior to Screening:
    • Intravenous immunoglobulin.
    • Corticosteroids greater than 10mg/day or azathioprine more than 100 mg/day.
    • Changes to corticosteroid or azathioprine therapy or MMF therapy.
    8. 30 Day Prior Therapy: Have received any of the following within 30 days prior to Day 0:
    • A live vaccine.
    9. Haemorrhage: Disease status where, in the opinion of the investigator, the subject could be at risk of haemorrhage that threatens a vital organ.
    10. Transplantation: Have a history of a major organ transplant or hematopoietic stem cell/marrow transplant.
    11. Cancer: Have a history of malignant neoplasm within the last 5 years, except for adequately treated cancers of the skin or carcinoma in situ of the uterine cervix.
    12. Acute or chronic infection: Have required management of acute or chronic infections, as follows:
    • Currently on any suppressive therapy for a chronic infection
    • Hospitalisation for treatment of infection within 60 days prior to Day 0.
    • Use of parenteral antibiotics within 60 days prior to Day 0.
    13. Other diseases/conditions: Have clinical evidence of significant unstable or uncontrolled acute or chronic diseases not due to ITP which, in the opinion of the investigator, could confound the results of the study or put the subject at undue risk. or Have a planned surgical procedure or a history of any other medical disease, laboratory abnormality, or condition that, in the opinion of the investigator, makes the subject unsuitable for the study.
    14. Hepatitis: A positive screening Hepatitis C antibody result or serologic evidence of Hepatitis B (HB) infection based on the results of testing for HBsAg, anti-HBc and anti-HBs as follows:
    • Patients positive for HBsAg are excluded.
    • Patients negative for HBsAg and anti-HBc antibody but positive for anti-HBs antibody and with no history of Hepatitis B vaccination are excluded.
    • Patients negative for HBsAg but positive for both anti-HBc and anti-HBs
    antibodies are excluded.
    • Patients negative for HBsAg and anti-HBs antibody but positive for anti-HBc antibody are excluded.
    15. HIV: A positive test for HIV antibody at screening or historically.
    16. Liver Function Tests: Aspartate aminotransferase (AST) and alanine
    aminotransferase (ALT) ≥ 2x upper limit of normal (ULN); alkaline phosphatase and bilirubin > 1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%).
    17. Immunodeficiency: Have an IgA deficiency (IgA < 10mg/dL).
    18. Laboratory Abnormalities: Abnormal laboratory assessments which are judged clinically significant by the investigator.
    19. Lymphopenia: Have a lymphocyte count <500 per mm3.
    20. Drug Sensitivity: History of sensitivity to any of the study medications, or components thereof or a history of drug or other allergy including a previous anaphalactic reaction to parenteral administration contrast agents, human or murine proteins or monoclonal antibodies, that, in the opinion of the investigator or GSK Medical Monitor, contraindicates their participation.
    21. Suicidality: Subjects, who have evidence of serious suicide risk including any history of suicidal behavior in the last 6 months and/or any suicidal ideation of type 4 or 5 on the C-SSRS in the last 2 months or who in the investigator's judgment, pose a significant suicide risk.

    For more information please refer Protocol page no. 39
    E.5 End points
    E.5.1Primary end point(s)
    - Change from baseline in platelet count at 12 weeks
    Data from other visits for these endpoints will also be provided as supporting/descriptive analyses.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 12
    E.5.2Secondary end point(s)
    - Efficacy
    • Time to first response (platelet count increase >20,000/μL from baseline)
    • Incidence of response of platelet count increase >20,000/μL from baseline (Day 0) over all timepoints
    • Incidence of complete response as defined by platelet count to >100,000/μL over all timepoints
    • Incidence of subjects with ≥ 2 times baseline platelet count over all timepoints
    - Mechanistic
    • Change in platelet bound GPIIb/IIIa and/or GPIb/IX anti-platelet antibodies at 12 weeks and by time
    • Change in relevant B cell and T cell sub-populations and BLyS receptor expression (by FACS analysis) by time
    • Change in antigen-specific B cells and T cells by time
    •Serum cytokine/chemokine and autoantibody profile by time
    • Change in transcriptomic profile by time
    - Safety and tolerability
    • Adverse Events (AEs)
    • Change from baseline and number of subjects outside the normal range for blood pressure, heart rate, temperature
    • Change from baseline in clinical chemistry and haematology parameters
    • Immunogenicity
    - Pharmacokinetics
    • Individual serum concentrations of belimumab and data permitting summary PK parameters
    E.5.2.1Timepoint(s) of evaluation of this end point
    Weeks 0, 2, 4, 8, 12, 16, 20, 24, 28, 40, 52 (dependent on endpoint)
    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 No
    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 Yes
    E.8.1.4Double blind No
    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 EEA5
    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 No
    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
    last subject’s last safety visit (16 week post last dose)
    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.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 35
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 40
    F.4.2.2In the whole clinical trial 40
    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)
    No, see protocol section 10.4
    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 Decision2012-02-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-03-30
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2013-03-07
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