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    Summary
    EudraCT Number:2008-000972-25
    Sponsor's Protocol Code Number:AB06006
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-02-24
    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 ConcernedBulgarian Drug Agency
    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
    A.4.1Sponsor's protocol code numberAB06006
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Information not present in EudraCT
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10026891
    E.1.2Term Mastocytosis
    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 is to compare the safety and efficacy of masitinib to placebo in patients with mastocytosis with handicap.

    Primary endpoint:
     Responder rate at week 24
    E.2.2Secondary objectives of the trial
    Secondary endpoints:
     Response in at least two out of three variables among pruritus, flushes, depression or asthenia, at week 12 and week 24
     Responder rate at week 12
     Pruritus score at week 12 and week 24
     Number of flushes per week at week 12 and week 24
     Hamilton score at week 12 and week 24
     Fatigue Impact Scale at week 12 and week 24
     Number of mictions per day at week 12 and week 24
     Number of stools per day at week 12 and week 24
     QLQ-C30 scores at week 12 and week 24
     Overall Patient Assessment (OPA) at week 12 and week 24
     AFIRMM V2 score at week 12 and week 24
     Percentage of patient without handicap at week 12 and 24
     Mast cell infiltration in the skin or bone marrow at week 24
     Serum tryptase level at week 24
    Safety profile of masitinib: Occurrence of Adverse Events, vital signs, EKG, Chest X-Ray, cardiac ultrasonography and biological parameters.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.
    1. Patient with one of the following documented mastocytosis as per WHO classification:
     Smouldering Systemic Mastocytosis
     Indolent Systemic Mastocytosis
     Cutaneous 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 asthenia:
     pruritus score ≥ 6
     number of flushes per week ≥ 7
     Hamilton rating scale (depression) ≥ 10
     number of stools per day ≥ 4 ,
     number of mictions per day ≥ 8 ,
     Fatigue Impact Scale total score (asthenia) ≥ 40
    5. Patients with OPA > 2 (moderate to intolerable general handicap)
    6. ECOG ≤ 2
    7. Patient with adequate organ function :
     absolute neutrophil count (ANC) ≥ 2.0 x 109/L,
     platelets (PTL) ≥ 100 x 109/L
     AST/ALT ≤ 2.5x ULN (≤ 5 x ULN in case of liver mast cell involvement),
     bilirubin ≤ 1.5x ULN
     creatinin clearance ≥50 mL/min (Cockrot and Gault formula)
     albumin > 0.75 x LLN
     urea ≤ 1.5 x ULN
     proteinuria < 30mg/dL on the dipstick;; in case of proteinuria ≥ 30 mg/dL, 24 hours proteinuria should be < 1.5g/24 hours
    8. Male or female patient with age ≥ 18 years
    9. Men and women of child bearing potential (entering the study after a confirmed menstrual period and who have a negative pregnancy test) must agree to use 2 methods of medically acceptable forms of contraception during the study. Men and women of childbearing potential must agree upon using contraception for three months following their participation in the study
    10. Patient should be able and willing to comply with study visits and procedures per protocol
    11. Patient should understand, sign, and date the written voluntary informed consent form at the screening visit prior to any protocol-specific procedures performed
    12. Patient must be covered by insurance for routine costs
    E.4Principal exclusion criteria
    1. Patient with one of the following mastocytosis:
     Systemic Mastocytosis with an Associated clonal Hematologic Non Mast cell lineage Disease (SM-AHNMD)
     Mast cell leukemia (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 condition:
    a. Ischemic heart disease, defined by at least one of the following condition:
    i. Medical history of ischemic heart disease
    ii. Clinical symptoms of ischemic heart disease
    iii. Q wave > 3 mm on the electrocardiogram
    iv. ST elevation or depression > 2 mm on the electrocardiogram
    v. Negative T wave in at least 2 leads of the electrocardiogram
    b. Cardiac failure, defined by at least one of the following condition:
    i. Medical history of cardiac failure defined by a previous left ventricular ejection fraction ≤ 50%
    ii. Clinical symptoms of cardiac failure
    iii. NT proBNP ≥ 300 pg/mL and/or troponin T > 0.1 ng/mL
    c. Conduction disorders or arrhythmia, defined by at least one of the following and confirmed by electrocardiogram:
    i. Severe ventricular arrhythmia (frequent premature ventricular beats)
    ii. Atrioventricular block at second or third level
    iii. Left bundle branch block
    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
    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
    Previous treatment
    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 variable: Responder rate
    A patient is classified as responder if he/she presents an improvement of ≥ 50% in at least one handicap, handicap being defined as a score above the cut in any of the 4 variables:
    - pruritus score ≥ 6,
    - number of flushes per week ≥ 7,
    - Hamilton score ≥ 10,
    - Fatigue Impact Scale score ≥ 40.
    Any patient with a worsening of more than 50% of any handicap, and/or with emergence of a new handicap with an increase of more than 50% from baseline, will be considered a non responder
    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.8.3 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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 / 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 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.1.3Elderly (>=65 years) Yes
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 150
    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 Decision2011-04-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-08-04
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-11-02
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