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    Summary
    EudraCT Number:2008-000972-25
    Sponsor's Protocol Code Number:AB06006
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-05-16
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2008-000972-25
    A.3Full title of the trial
    A 24-week with possible extension, prospective, multicentre, randomized, double blind, placebo-controlled, 2-parallel group with a randomization 1:1, Phase III study to compare efficacy and safety of masitinib at 6 mg/kg/day to placebo in treatment of patients with Smouldering Systemic, Indolent Systemic or Cutaneous Mastocytosis with handicap
    24 hetes, meghosszabbítható, prospektív, multicentrikus, randomizált, kettős-vak, placebo-kontrollos, két párhuzamos csoportos, 1:1 arányú randomizálást alkalmazó, III. fázisú vizsgálat, melynek célja a napi 6 mg/kg adagban alkalmazott masitinib hatásosságának és biztonságosságának összehasonlítása placebóéval, tünetekkel járó lappangó szisztémás, indolens szisztémás vagy cutan mastocytosisban szenvedő betegeknél
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to compare efficacy and safety of masitinib at 6 mg/kg/day to placebo in treatment of patients with Indolent with handicap
    A.4.1Sponsor's protocol code numberAB06006
    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 SponsorAB SCIENCE
    B.1.3.4CountryFrance
    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 supportAB Science
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHungarotrial Zrt
    B.5.2Functional name of contact pointDr. László Nagy
    B.5.3 Address:
    B.5.3.1Street AddressFehérvári út 89-95.
    B.5.3.2Town/ cityBudapest
    B.5.3.3Post code1119
    B.5.3.4CountryHungary
    B.5.4Telephone number3612032134
    B.5.5Fax number3612033985
    B.5.6E-maillnagy@hungarotrial.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/3/04/242
    D.3 Description of the IMP
    D.3.1Product nameAB1010 Tablets
    D.3.2Product code AB1010 Tablets
    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 INNMasitinib mesylate
    D.3.9.1CAS number 790-299-79-5
    D.3.9.2Current sponsor codeAB1010
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMasitinib mesylate
    D.3.9.1CAS number 790-299-79-5
    D.3.9.2Current sponsor codeAB1010
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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
    Smouldering Systemic, Indolent Systemic or Cutaneous Mastocytosis with handicaps
    Tünetekkel járó lappangó szisztémás, indolens szisztémás vagy cutan mastocytosis
    E.1.1.1Medical condition in easily understood language
    Indolent Mastocytosis
    Lappangó Mastocytosis
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    E.1.2Level PT
    E.1.2Classification code 10026891
    E.1.2Term Mastocytosis
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objective of this study is to compare the efficacy and safety of masitinib at 6 mg/kg/day to
    placebo in the treatment of patients with documented Smouldering or Indolent Systemic mastocytosis
    with severe handicap based on treatment effect on the pruritus score, the number of flushes per week,
    the HAMD-17 score, and the Fatigue Impact Scale score.

    Primary endpoint:
    - Cumulative response by patient*handicap
    E.2.2Secondary objectives of the trial
    Secondary endpoints:
    - Cumulative response on pruritus among patients with the handicap at Baseline
    - Cumulative response on OPA score among patients with “severe” or “intolerable”
    handicap at Baseline
    - Quality of Life (QoL) : QLQ-C30 global score, functional scores and symptom scores at
    each visit
    - AFIRMM questionnaire :
    · global score
    · for each of the 52 items : cumulative response among patients with “severe” or
    “intolerable” handicap at Baseline
    - Cumulative response on micturitions among patients with the handicap at Baseline
    - Cumulative response on stools among patients with the handicap at Baseline
    - Urticaria Pigmentosa (UP) evaluation at week 12, 24 and then every 12 weeks
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient with one of the following documented mastocytosis as per WHO classification:
    Smouldering Systemic Mastocytosis
    Indolent Systemic Mastocytosis
    2. Patient with documented mastocytosis and evaluable disease based upon histological criteria:
    typical infiltrates of mast cells in a multifocal or diffuse pattern in skin and/or bone marrow biopsy
    3. Patient with documented treatment failure of his/her handicap(s) with at least one of the following
    therapy used at optimized dose (refer to table 2):
    Anti H1
    Anti H2
    Proton pump inhibitor
    Osteoclast inhibitor
    Cromoglycate Sodium
    Antileukotriene
    4. Handicapped status defined as at least two of the following handicaps, including at least one
    among pruritus, flushes, depression and fatigue:
    Pruritus score ≥ 9
    Number of flushes per week ≥ 8
    Hamilton rating scale for depression(HAMD-17) score ≥ 19
    Number of stools per day ≥ 4
    Number of micturition per day ≥ 8
    Fatigue Impact Scale total score (asthenia) ≥ 75
    5. Patients with OPA > 2 (moderate to intolerable general handicap)
    6. ECOG ≤ 2
    7. Patient with adequate organ function :
    Absolute neutrophils count (ANC) ≥ 2.0 x 109/L,
    Haemoglobin ≥ 10 g/dL
    Platelets (PTL) ≥ 100 x 109/L
    AST/ALT ≤ 3x ULN (≤ 5 x ULN in case of liver mast cell involvement),
    Bilirubin ≤ 1.5x ULN
    Creatinine clearance >60mL/min (Cockcroft and Gault formula)
    Albumin >1 x LLN
    Proteinuria < 30mg/dL on the dipstick; in case of proteinuria ≥ 1+ on dipstick, 24 hours
    proteinuria should be <1.5g/24 hours
    8. Male or female patient aged 18 to 75 years, weight > 50 kg, BMI between 18 and 35 kg/m²
    9. Male or female patient of child bearing potential ust agree to use two methods (one for the patient
    and one for the partner) of medically acceptable forms of contraception during the study and for
    three months after the last treatment intake. Female patients must have a negative result in the
    pregnancy tests at screening and baseline.
    10. Patient must be able and willing to comply with study visits and procedures per protocol
    11. Patient must understand, sign, and date the written voluntary informed consent form at the
    screening visit prior to any protocol-specific procedures performed
    12. Patient must understand the patient card and follow the patient card procedures in case of signs or
    symptoms of severe neutropenia or severe cutaneous toxicity during the first 2 months of
    treatment
    13. Patient affiliated to a social security regimen
    E.4Principal exclusion criteria
    1. Patient with one of the following mastocytosis:
    Cutaneous Mastocytosis
    Not documented Smouldering Systemic Mastocytosis or Indolent Systemic Mastocytosis
    Systemic Mastocytosis with an Associated clonal Hematologic Non Mast cell lineage
    Disease (SM-AHNMD)
    Mast cell leukaemia (MCL)
    Aggressive systemic mastocytosis (ASM)
    2. Previous treatment with any Tyrosine Kinase Inhibitor
    3. Patient presenting with cardiac disorders defined by at least one of the following conditions:
    Patient with recent cardiac history (within 6 months) of:
    - Acute coronary syndrome
    - Acute heart failure (class III or IV of the NYHA classification)
    - Significant ventricular arrhythmia (persistent ventricular tachycardia, ventricular
    fibrillation, resuscitated sudden death)
    Patient with cardiac failure class III or IV of the NYHA classification
    Patient with severe conduction disorders which are not prevented by permanent pacing
    (atrio-ventricular block 2 and 3, sino-atrial block)
    Syncope without known aetiology within 3 months
    Uncontrolled severe hypertension, according to the judgment of the investigator, or
    symptomatic hypertension
    4. Patient who had major surgery within 2 weeks prior to screening visit
    5. Vulnerable population defined as:
    Life expectancy < 6 months
    Patient with < 5 years free of malignancy, except treated basal cell skin cancer or cervical
    carcinoma in situ
    Patient with any severe and/or uncontrolled medical condition
    Patient with known diagnosis of human immunodeficiency virus (HIV) infection
    6. Patient with history of poor compliance or history of drug/alcohol abuse, or excessive alcohol
    beverage consumption that would interfere with the ability to comply with the study protocol, or
    current or past psychiatric disease that might interfere with the ability to comply with the study
    protocol or give informed consent, or institutionalized by court decision
    7. Patient with any condition that the physician judges could be detrimental to subjects participating
    in this study; including any clinically important deviations from normal clinical laboratory values
    or concurrent medical events
    8. Change in the symptomatic treatment of mastocytosis or administration of any new treatment of
    mastocytosis within 4 weeks prior to baseline
    9. Treatment with any investigational agent within 4 weeks prior to baseline
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoint:
    - Cumulative response by patient*handicap
    E.5.1.1Timepoint(s) of evaluation of this end point
    Secondary endpoints:
    - Cumulative response on pruritus among patients with the handicap at Baseline
    - Cumulative response on OPA score among patients with “severe” or “intolerable” handicap at
    Baseline
    - Quality of Life (QoL) : QLQ-C30 global score, functional scores and symptom scores at each
    visit
    - AFIRMM questionnaire :
    · global score
    · for each of the 52 items : cumulative response among patients with “severe” or “intolerable”
    handicap at Baseline
    - Cumulative response on micturitions among patients with the handicap at Baseline
    - Cumulative response on stools among patients with the handicap at Baseline
    - Urticaria Pigmentosa (UP) evaluation at week 12, 24 and then every 12 weeks
    E.5.2Secondary end point(s)
    Response on at least one handicap out of 4 among pruritus, flushes, depression and fatigue
    - Overall cumulative response
    - Sustained response rate (two consecutive response)
    - Responder rate at week 12
    - Confirmed responder rate (response at week 20 then week 24)
    Response on at least one handicap out of 3 among pruritus, flushes and depression/fatigue (for depression/fatigue, a patient will be considered as responder if response on both depression and fatigue as those variables are partially overlapping)
    Responder rate at week 24
    - Overall cumulative response
    - Sustained response rate (two consecutive response)
    - Responder rate at week 12
    - Confirmed responder rate (response at week 20 then week 24)
    Response on at least 2 handicaps out of 4 among pruritus, flushes, depression and fatigue (among patients with at least 2 handicaps at baseline)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 24
    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 No
    E.6.7Pharmacodynamic No
    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) 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 concerned100
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA160
    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
    Bulgaria
    Czech Republic
    France
    Germany
    India
    Italy
    Latvia
    Poland
    Russian Federation
    Serbia
    Slovakia
    Ukraine
    United Kingdom
    United States
    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
    End of the trial is defined as last visit of the last patient at week 24.

    Possible extension visits will not be taken in consideration for end of trial definition.
    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 months0
    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.1.3Elderly (>=65 years) Information not present in EudraCT
    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 state130
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 170
    F.4.2.2In the whole clinical trial 200
    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)
    Possibility will be offered to the patients who respond to the IMP at week 24 to continue their treatment (AB1010/placebo) untill database lock. Then afer unblinding patients who were treated with AB1010 and who were still responders upon database lock will be allowed to continue their treatment with AB1010 till market authorisation or development cessation whichever occurs first
    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 Decision2013-10-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-09-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-11-02
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