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    Summary
    EudraCT Number:2012-000658-67
    Sponsor's Protocol Code Number:DEEP-1
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2012-11-07
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2012-000658-67
    A.3Full title of the trial
    Multi-centre, oral single dose experimental and modelling study to evaluate the pharmacokinetics of deferiprone in patients aged from 1 month to less than 6 years of age affected by transfusion-dependent haemoglobinopathies
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Pharmacokinetic study of deferiprone in paediatric patients
    A.3.2Name or abbreviated title of the trial where available
    DEEP-1
    A.4.1Sponsor's protocol code numberDEEP-1
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/307/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorConsorzio per Valutazioni Biologiche e Farmacologiche (CVBF)
    B.1.3.4CountryItaly
    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 supportEuropean Commission - HEALTH-F4-2010 - SP1 - Cooperation Collaborative Project - Grant Agreement n° 261483
    B.4.2CountryEuropean Union
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationConsorzio per Valutazioni Biologiche e Farmacologiche (CVBF)
    B.5.2Functional name of contact pointDirezione Scientifica
    B.5.3 Address:
    B.5.3.1Street AddressVia Luigi Porta 14
    B.5.3.2Town/ cityPavia
    B.5.3.3Post code27100
    B.5.3.4CountryItaly
    B.5.4Telephone number39038225075
    B.5.5Fax number390382536544
    B.5.6E-maildeep-1@deep-project.net
    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 namedeferiprone
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    chronic iron overload
    E.1.1.1Medical condition in easily understood language
    chronic iron overload
    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 14.1
    E.1.2Level LLT
    E.1.2Classification code 10065974
    E.1.2Term Chronic iron overload
    E.1.2System Organ Class 100000004861
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the pharmacokinetics of deferiprone in paediatric patients aged from 1 month to less than 6 years
    E.2.2Secondary objectives of the trial
    1) To identify dose levels yielding deferiprone exposures comparable to adults and define the dose rationale in children aged from 1 month to
    less than 6 years.
    2) To evaluate safety and tolerability of deferiprone after single dose administration in children aged from 1 month to less than 6 years.
    3) To evaluate the effect of demographic covariates on DFP disposition
    and estimate the clearance distribution across the population.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    a) Patients in a chronic transfusional program who have received at
    least 150 ml/kg/year of packed red blood cells (corresponding
    approximately to 12 transfusions) and on current treatment with DFO,
    DFX, DFP; aged from 1 month to less than 6 years.
    b) Patients naïve to any chelation treatment who have received not less
    than 150 ml/kg of packed red blood cells (corresponding to
    approximately 12 transfusions) and have ferritin levels > 800 ng/mL,
    aged from 1 month to less than 6 years.
    c) Patients who meet the transfusion criteria (150 ml/kg/year
    corresponding approximately to 12 transfusions) and have known
    intolerance or contraindication to DFO
    And if all of the following criteria apply:
    d) Patients affected by any hereditary haemoglobinopathies requiring
    chronic transfusion therapy including but not limited to thalassaemia
    and sickle cell disease
    e) Written informed consent obtained from their legal guardian on the
    patient's behalf in accordance with the national legislations. According to his/her capability, patient's informed assent will be collected
    E.4Principal exclusion criteria
    a) Patient with known intolerance or contraindication to the trial
    treatment
    b) Patient with Hb levels less than 8g/dl (entry may be delayed until
    values return to normal)
    c) Patient with platelet count <100.000/mm3 or absolute neutrophil
    count <1.500/mm3 (entry may be delayed until values return to normal)
    d) Patient with evidence of abnormal liver function (ALT level >5 times
    the upper normal limit during six months preceding enrolment; entry
    may be delayed until values return to normal)
    e) iron overload from causes other than trasfusional haemosiderosis
    f) severe heart dysfunction secondary to iron overload defined as the
    occurrence of heart failure or severe arrhythmia or as indicated by
    cardiac T2* lower than 10 ms, if recent MRI data is available,
    g) Patient with serum creatinine level above the upper normal limit at
    screening; entry may be delayed until values return to normal.
    h) Serological evidence of chronic hepatitis B (presence of HBe Ag,
    HBsAg, HBcAb-IgM, in the absence of HBsAb).
    i) History of significant medical or psychiatric disorder that may impair
    compliance with the requirements of the protocol.
    j) The patient has received another investigational drug within 30 days
    prior to this study.
    k) Patient with a pre-existing condition or any other surgical or medical
    condition which might significantly interfere with normal
    gastrointestinal and hepatic function that could alter the absorption,
    metabolism, and/or excretion of the study drug.
    l) Patient with a known history of HIV seropositivity.
    m) Fever and other signs/symptoms of infection in the 10 days before
    drug administration(treatment day)
    n) Concomitant use of other iron chelators or trivalent cation-dependent
    medicinal products such as aluminium-based antacids.
    o) Patient with a chronic condition that does not allow suspension of
    related treatment from starting of washout until drug is administered.
    E.5 End points
    E.5.1Primary end point(s)
    Pharmacokinetic endpoints derived from the PK study will include:
    • Primary PK parameters: CL/F, Vd/F, Ka
    • Secondary PK parameters: AUC, Cmax,Tmax, Css and Cmin
    E.5.1.1Timepoint(s) of evaluation of this end point
    Samples will be collected from pre-dose up to 8 hours post-dose on a single day after drug administration
    E.5.2Secondary end point(s)
    Secondary pharmacodynamic/biomarker endpoints will include:
    Clinical safety and tolerability data including ferritin levels, spontaneous AE reporting, ECGs, vital signs, nursing/physician observation and clinical laboratory values (haematology and biochemistry).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Blood samples for clinical chemistry and haematology will be collected at screening and during follow up and early termination visit if a patient withdraws or is withdrawn from the study. Ferritin levels will be collected only at screening. Adverse events, vital signs and ECG will also be monitored 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 No
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Egypt
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial months8
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 30
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 25
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 30
    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)
    Subjects will receive any available chelation therapy upon his/her physician discretion
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: Italy
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