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    Summary
    EudraCT Number:2014-000174-19
    Sponsor's Protocol Code Number:9345
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2014-09-01
    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 ConcernedGermany - BfArM
    A.2EudraCT number2014-000174-19
    A.3Full title of the trial
    Efficacy and Safety of Eltrombopag in Patients with Acquired Moderate Aplastic Anemia (EMAA) who are treated with Ciclosporin A

    Prospective Randomized Multicenter Study comparing Thrombopoetin-Receptor agonist Eltrombopagwith Placebo in Patients with Acquired Moderate Aplastic Anemia who are treated with Ciclosporin A
    Wirksamkeit und Sicherheit von Eltrombopag in Kombination in Patienten mit Erworbener Moderater Aplastischer Anämie (EMAA), die mit Ciclosporin A behandelt werden

    Eine prospektive, randomisierte, multizentrische klinische Studie zum Vergleich des Thrombopoetinrezeptor-Agonist Eltrombopag mit Placebo in Patienten mit Erworbener Moderater Aplastischer Anämie (EMAA), die mit Ciclosporin behandelt werden
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy and Safety of Eltrombopag in Patients with Acquired Moderate Aplastic Anemia (EMAA) who are treated with Ciclosporin A

    Prospective Randomized Multicenter Study comparing Thrombopoetin-Receptor agonist Eltrombopag with Placebo in Patients with Acquired Moderate Aplastic Anemia who are treated with Ciclosporin A
    Wirksamkeit und Sicherheit von Eltrombopag in Kombination in Patienten mit Erworbener Moderater Aplastischer Anämie (EMAA), die mit Ciclosporin A behandelt werden

    Eine prospektive, randomisierte, multizentrische klinische Studie zum Vergleich des Thrombopoetinrezeptor-Agonist Eltrombopag mit Placebo in Patienten mit Erworbener Moderater Aplastischer Anämie (EMAA), die mit Ciclosporin behandelt werden
    A.3.2Name or abbreviated title of the trial where available
    EMAA
    A.4.1Sponsor's protocol code number9345
    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 SponsorUniversitätsklinikum Ulm
    B.1.3.4CountryGermany
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportNovartis Pharma GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversitätsklinikum Ulm
    B.5.2Functional name of contact pointBritta Höchsmann
    B.5.3 Address:
    B.5.3.1Street AddressHelmholtzstraße 10
    B.5.3.2Town/ cityUlm
    B.5.3.3Post code89081
    B.5.3.4CountryGermany
    B.5.4Telephone number0049731150560
    B.5.5Fax number0049731150500
    B.5.6E-mailb.hoechsmann@blutspende.de
    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 Revolade 75 mg
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Pharma GmbH
    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 nameEltrombopag
    D.3.2Product code SB497115
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEltrombopag
    D.3.9.1CAS number 496775-62-3
    D.3.9.2Current sponsor codeSB497115
    D.3.9.3Other descriptive nameELTROMBOPAG OLAMINE
    D.3.9.4EV Substance CodeSUB30141
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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 placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Moderate Aplastic Anemia (MAA)
    E.1.1.1Medical condition in easily understood language
    Moderate Aplastic Anemia (MAA) is a non-malignant blood disorder.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The aim of this trial is to improve treatment of Moderate Aplastic Anemia (MAA) by evaluating the safety and efficiency
    of Eltrombopag as a new treatment option in patients with MAA requiring therapy.

    The primary objective of this trial is the evaluation of the superiority of Eltrombopag on top of background treatment
    with Ciclosporin (CSA) regarding hematologic response (PR +CR) at 6 months in comparison with treatment with CSA alone in untreated MAA patient.
    E.2.2Secondary objectives of the trial
    The secondary objective of this trial is to investigate the impact of Eltrombopag added to background therapy with
    CSA on all outcome measures, safety and quality of life in untreated MAA patients. As well as the evaluation of telomere
    lengths and telomerase mutations as biomarkers for response to Eltrombopag therapy in MAA and the evaluation of the new Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (PNH) specific quality of life questionnaire QLQ-AA/PNH.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Current diagnosis of a Moderate Aplastic Anemia requiring standard treatment with CSA without prior
    specific therapy.

    MAA is defined as Aplastic Anemia fulfilling the following criteria:
    • no evidence for other disease, causing marrow failure
    • hypocellular bone marrow for age
    • depression of at least two out of three peripheral blood counts below the normal values in two different blood samples in a time span from at least two weeks:
    • absolute neutrophil count (ANC) < 1.2 G/L
    • platelet count < 70 G/L
    • absolute reticulocyte count < 60 G/L21
    • without fulfilling the criteria for SAA (hypocellularity of bone marrow 25 % and depression of two of the three peripheral counts: ANC < 0.5 G/L, platelet count < 20 G/L, reticulocyte count < 20 G/L)

    1. In this study need for treatment with CSA is defined as:
    a. transfusion-independent MAA and:
    • ANC < 1.0 G/L
    • or hemoglobin < 8.5 g/dl and reticulocyte count < 60 G/L
    • or platelet count < 30 G/L
    • or significant clinical symptoms (infections, bleeding, anemia)
    b. transfusion-dependent MAA:
    • Platelet transfusion dependency is defined as prophylactic transfusion (platelet counts < 10 G/L with no bleeding) or therapeutic transfusion in the 12 weeks prior to study entry.
    • Red blood cell transfusion dependency is defined as transfusion of at least 4 units of packed red blood cell
    concentrates (PRBC) in the 12 weeks prior to study entry.
    2. A signed and dated informed consent is necessary before the conduct of any study-specific procedure.
    E.4Principal exclusion criteria
    1. Age < 18 years
    2. Constitutional Aplastic Anemia (i.e. Fanconi anemia or Dyskeratosis congenita)
    3. Clonal myeloid disorders based on cytogenetic findings performed within 12 weeks of study entry. Especially patients with cytogenetic abnormalities which are recurrent in MDS are not eligible for the
    study.
    4. Bone marrow reticulin fibrosis of grade 3 or greater
    5. Severe concurrent diseases precluding the patient's ability to tolerate protocol therapy
    6. ALT > 3 times the upper limit of normal if this elevation is progressive, or persistent during the 4 weeks before study entry or accompanied by increased direct bilirubin, or accompanied by clinical symptoms of liver injury or evidence for hepatic decompensation
    7. Infection not adequately responding to appropriate therapy
    8. HIV-positivity (patients with Hepatitis B or Hepatitis C are only in combination with hepatic failure - see
    criteria 7- excluded)
    9. Moribund status with a likely death within 3 months.
    10. History of malignancy other than localized tumors diagnosed more than one year previously and treated surgically with curative intent (for instance squamous cell or other skin cancers, stage 1, breast cancer in
    situ, cervical carcinoma in situ...).
    11. Prior specific treatment of Aplastic Anemia with immuno-suppression or androgens or interleukin2- receptor-antibodies. The use of these drugs in context with other disorders before diagnosis of aplastic anemia is not an exclusion criteria if these treatments were finished longer than 6 months before study entry.
    12. Treatment with other hematological effective drugs (including erythropoetin) within 3 months before study
    entry as well as treatment with corticosteroids and GCSF within 3 weeks before enrollment.
    13. Known hypersensitivity to Eltrombopag or its components
    14. Known hypersensitivity to Ciclosporin.
    15. Current nursing, pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth
    control to refrain from pregnancy as well as a missing or positive pregnancy test within the last 14 days before inclusion for women of childbearing potential during the course of this study.
    16. Inability to understand the investigational nature of the study or to give informed consent.
    17. Renal failure with creatinine > 2× upper limit of normal.
    18. Uncontrolled hypertension.
    19. Participation in any study using an investigational drug or treatment with an investigational drug within 30 days preceding the first dose of study medication.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of the study is the hematologic response rate (CR + PR) at 6 months.
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 months
    E.5.2Secondary end point(s)
    Secondary endpoints are:
    • Trilineage (CR and PR) and single lineage hematological response rate at 3, 6, 12 and 18 months.
    • cumulative incidence of response
    • time to best hematological trilineage and single lineage response
    • Proportion of patients with need for transfusions and number of units transfused (PRBC and PC) since start of treatment
    • cumulative incidence of progress to SAA/VSAA or intensive immunosuppressive treatment with ATG
    • toxicity profile as measured using the CTCAE criteria
    • relapse rate at 6, 12 and 18 months
    • cumulative incidence of relapse (from best trilineage hematological response)
    • overall survival
    • failure-free survival
    • telomere lengths and presence of telomerase mutations as biomarkers for response.
    • quality of life as assessed by quality of life instruments (FACIT-F SCALE and EORTC QLQ-C30, QLQAA/PNH)

    Pharmacokinetic studies for assessment of dose dependency regarding efficiency and safety in a subgroup of patients.
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Trilineage hematological response rate (CR and PR) at 3, 6, 12 and 18 months.
    • single lineage response at 3, 6, 12 and 18 months
    • relapse rate at 6, 12 and 18 months
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) 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 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 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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    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
    France
    Germany
    Italy
    Netherlands
    Switzerland
    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 years4
    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 years4
    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: 90
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 90
    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 state60
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 90
    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 conclusion of the trial patients will receive further standard medical care as usual for this kind of disease.
    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 Decision2015-01-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-11-10
    P. End of Trial
    P.End of Trial StatusOngoing
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
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