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    Summary
    EudraCT Number:2012-000658-67
    Sponsor's Protocol Code Number:DEEP-1
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-08-07
    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 ConcernedItaly - Italian Medicines Agency
    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
    Studio sperimentale e di modelling, multi-centro per la valutazione della farmacocinetica del deferiprone dopo singola somministrazione per via orale in pazienti di età compresa tra 1 mese e meno di 6 anni, affetti da emoglobinopatie trasfusione dipendenti
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Pharmacokinetic study of deferiprone in paediatric patients
    Studio della farmacocinetica del deferiprone in pazienti pediatrici
    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
    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
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/10/832
    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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDEFERIPRONE
    D.3.9.1CAS number 30652-11-0
    D.3.9.4EV Substance CodeSUB06941MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number80
    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
    Accumulo di ferro cronico
    E.1.1.1Medical condition in easily understood language
    chronic iron overload
    Accumulo di ferro cronico
    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 17.1
    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
    Definire la farmacocinetica del DFP in pazienti pediatrici di età compresa tra 1 mese e meno di 6 anni
    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.
    1) Identificare il dosaggio che fornisca un’esposizione al deferiprone paragonabile a quella nell’adulto e definire il dosaggio nei bambini di età compresa tra 1 mese e meno di 6 anni.
    2) Valutare sicurezza e tollerabilità del DFP dopo somministrazione singola in questo gruppo di pazienti pediatrici.
    3) Valutare l’effetto dei fattori demografici sulla disposizione del DFP e stimare la distribuzione della clearance nella popolazione di interesse.
    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
    I pazienti pediatrici saranno inclusi nello studio se almeno uno dei seguenti criteri è riscontrato:
    - Pazienti di età compresa tra 1 mese e meno di 6 anni in un programma trasfusionale cronico che hanno ricevuto almeno 150 ml/kg/anno di globuli rossi (corrispondenti approssimativamente a 12 trasfusioni) e attualmente in trattamento con DFO, DFX, DFP.
    - Pazienti di età compresa tra 1 mese e meno di 6 anni non trattati in precedenza con qualsiasi trattamento chelante che hanno ricevuto non meno di 150 ml/kg di globuli rossi (corrispondenti approssimativamente a 12 trasfusioni) e presentano livelli di ferritina > 800 ng/mL.
    - Pazienti che soddisfano i criteri trasfusionali (150 ml/kg/anno corrispondenti approssimativamente a 12 trasfusioni) e che sono intolleranti alla DFO o per i quali la DFO è controindicata.
    E se tutti i criteri seguenti vengono soddisfatti:
    - Pazienti affetti da qualsiasi emoglobinopatia ereditaria che richieda un trattamento trasfusionale cronico, incluse ma non limitato esclusivamente a thalassaemia ed anemia falciforme (“sickle cell disease”).
    - Consenso informato scritto, ottenuto dal tutore legale per conto del paziente in accordo con la legislazione nazionale. Se appropriato, in base alla capacità del bambino, verrà richiesto anche l’assenso informato del paziente.
    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.
    - Paziente intollerante al trattamento dello studio o per il quale il trattamento è controindicato
    - Paziente con livelli di emoglobina inferiori a 8 g/dl (l’inclusione può essere rinviata fino a quando i valori non rientrano nella normalità)
    - Paziente con conta piastrinica < 100000/mm3 o conta neutrofila assoluta < 1500/mm3 (l’inclusione può essere rinviata fino a quando i valori non rientrano nella normalità)
    - Paziente con funzione epatica anormale (ALT > 5 volte il limite superiore di normalità durante i sei mesi precedenti il reclutamento; l’inclusione può essere rinviata fino a quando i valori non rientrano nella normalità)
    - Sovraccarico di ferro dovuto a cause diverse dall’emosiderosi trasfusionale
    - Grave disfunzione cardiaca secondaria al sovraccarico di ferro definita come l’insorgenza di insufficienza cardiaca o aritmia grave, o come indicato da T2* cardiaco inferiore a 10 ms, se dati recenti di MRI sono disponibili
    - Pazienti con livelli sierici di creatinina più alti del limite superiore di normalità durante lo screening; l’inclusione può essere rinviata fino a quando i valori non rientrano nella normalità
    - Evidenza sierologica di epatite B cronica (presenza di HBe Ag, HBsAg, HBcAb-IgM, in assenza di HBsAb)
    - Storia di significativo disturbo medico o psichiatrico che possa pregiudicare l’aderenza ai requisiti del protocollo
    - Il paziente ha ricevuto un altro farmaco in studio clinico nei 30 giorni precedenti questo studio
    - Paziente con una condizione pre-esistente o con una qualsiasi altra condizione medicochirurgica che potrebbe interferire in modo significativo con la normale funzione epatica e gastrointestinale e di conseguenza potrebbe alterare l’assorbimento, il metabolismo, e/o l’escrezione del farmaco in studio.
    - Paziente con una storia conosciuta di sieropositività (HIV)
    - Febbre ed altri segni/sintomi di infezione nei 10 giorni precedenti la somministrazione del farmaco (giorno del trattamento)
    - Uso concomitante di altri chelanti del ferro o prodotti medicinali cationici trivalenti dipendenti come ad esempio antiacidi a base di alluminio
    - Pazienti che presentano una condizione cronica che non consente la sospensione del relativo trattamento dall’inizio del “washout” fino alla somministrazione del farmaco in studio
    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
    Gli endpoint di farmacocinetica che verranno valutati nello studio, includono:
    Parametri primari di farmacocinetica: CL/F, Vd/F, Ka
    Parametri secondari di farmacocinetica: 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. A maximum of 5 samples (post-dose; each 2 ml) will be collected per patient according a predefined sampling matrix.
    I campioni verranno raccolti da pre-dose fino a 8 ore post-dose. Un massimo di 5 campioni (post-dose; ognuno di 2 ml) verrà raccolto per paziente secondo una matrice di campionamento predefinita.
    E.5.2Secondary end point(s)
    Secondary safety and tolerability endpoints will include: adverse events , vital signs and ECG will be monitored during the study. At day of treatment, clinical safety and tolerability data will be collected, including spontaneous adverse events reporting, ECGs, vital signs and all observations reported by nursing/physicians.
    Endpoint secondari di sicurezza e tollerabilità includeranno: Effetti avversi, segni vitali ed ECG verranno monitorati durante lo studio. Nel giorno del trattamento, verranno raccolti dati di sicurezza e tollerabilità clinica, inclusa la segnalazione spontanea di effetti avversi, ECG, segni vitali e qualsiasi sintomo riportato da personale medico/infermieristico.
    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.
    I campioni di sangue per analisi chimico cliniche (ematologia e biochimica) saranno raccolti allo screening e durante la visita di follow up,e durantela visita anticipata di fine trattamento se il paziente si ritira o viene ritirato dallo studio. I livelli di ferritina verranno misurati solo durante lo screening.
    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 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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Cyprus
    Egypt
    Italy
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    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.4.1In the member state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 23
    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.
    I soggetti riceveranno una qualsiasi terapia chelante a discrezione del medico curante
    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 Decision2012-07-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-04-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-02-17
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