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    Summary
    EudraCT Number:2016-000786-24
    Sponsor's Protocol Code Number:NET-2013-02355002
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-11-30
    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 number2016-000786-24
    A.3Full title of the trial
    Thalidomide, a novel immunological treatment to modify the natural history of paediatric Crohn's disease: a new proposal from a well-established paediatric research network
    Talidomide: un nuovo trattamento immunologico per modificare la storia naturale del morbo di Crohn a esordio pediatrico: una nuova proposta da un gruppo di ricerca pediatrico consolidato
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy and safety of thalidomide in pediatric patients with Crohn's disease
    Efficacia e sicurezza della talidomide in pazienti pediatrici affetti da morbo di Crohn
    A.3.2Name or abbreviated title of the trial where available
    Talidomide in Crohn disease in Pediatrics
    Talidomide nel Morbo di Crohn in pediatria
    A.4.1Sponsor's protocol code numberNET-2013-02355002
    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 SponsorIRCCS Burlo Garofolo
    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 supportMinistry of Health
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIRCCS materno infantile Burlo Garofolo
    B.5.2Functional name of contact pointAlessandra Maestro
    B.5.3 Address:
    B.5.3.1Street Addressvia dell'Istria 65/1
    B.5.3.2Town/ cityTRIESTE
    B.5.3.3Post code34137
    B.5.3.4CountryItaly
    B.5.4Telephone number00390403785418
    B.5.5Fax number00390403785411
    B.5.6E-mailsperimentazioni.cliniche@burlo.trieste.it
    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 THALIDOMIDE CELGENE - 50 MG CAPSULA RIGIDA - USO ORALE - BLISTER (PVC/PE/ACLAR/ALU) 28 CAPSULE
    D.2.1.1.2Name of the Marketing Authorisation holderCELGENE EUROPE LIMITED
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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.2.5The 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 nameINFLIXIMAB
    D.3.4Pharmaceutical form Powder for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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
    Crohn's Disease
    Morbo di Crohn
    E.1.1.1Medical condition in easily understood language
    Inflammatory bowel disease
    Malattia infiammatoria cronica dell'intestino
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10011402
    E.1.2Term Crohn's disease (colon)
    E.1.2System Organ Class 100000004856
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of thalidomide compared with standard therapy in inducing mucosal healing and 52 weeks
    Valutare l’efficacia della talidomide rispetto alla terapia standard nell’ indurre la guarigione mucosale a 52 settimane dall’avvio del trattamento.
    E.2.2Secondary objectives of the trial
    To assess the efficacy of thalidomide
    compared with standard therapy in:
    - inducing a clinical response amd remission(12 and 52 weeks)
    - reducing the need of therapeutic changes at 12 and 52 weeks
    - reducing the number of hospitalizations up to 52 weeks.
    - reducing the number of patients who undergo surgery up to 52 weeks
    - changing inflammation laboratory indices at 12 and 52 weeks .
    - changing nutritional indicators at12 to 52 weeks and bone mineralizaztion at 52 weeks.
    To assess the safety of thalidomide compared with standard therapy evaluating the number and type of adverse effects at 12 and
    52 weeks.
    - direct and indirect costs of thalidomide compared with standard therapy up to 52 weeks
    - results from genetic analysis from patient with Crohn’s disease;
    - results from immunological analysis from patient with CD;
    - information on the mechanisms of action of thalidomide in CD;
    - pharmacogenetic data of thalidomide in CD;
    - pharmacogenomic data
    Valutare la talidomide rispetto alla terapia standard in termini di:
    - l’efficacia nell’indurre una risposta clinica e remissione a 12 e a 52 settimane
    - l’efficacia nel ridurre la necessità di cambiamenti terapeutici a 12 e 52 settimane.
    - l’efficacia nel ridurre il numero di ospedalizzazioni a 52 settimane.
    - l’efficacia nel ridurre il numero di pazienti con necessità di chirurgia a 52 settimane.
    - l’efficacia sull’andamento degli indici laboratoristici di infiammazione a 12 e a 52 settimane.
    - l’efficacia sull'andamento sigli indici nutrizionali e di crescita a 12 e a 52 settimane, e di mineralizzazione ossea a 52 sett
    - la sicurezza in base agli eventi avversi della terapia a 12 e 52 settimane
    - costi diretti ed indiretti delle terapie
    - ottenere dati sulla genetica, sui meccanismi immunologici, sui meccanismi d'azione sulla farmacogenetica, genomica e
    metabolomica
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Pharmacoeconomics study
    Version: 1
    Data: 15/11/2017
    Genetic and immunology of Crohn's disease
    Versione: 1
    Data: 15/11/2016
    Farmacoeconomia
    Versione: 1
    Data: 15/11/2017
    Farmacogenetica:
    Versione: 1
    Data: 15/11/2016
    E.3Principal inclusion criteria
    - Age at diagnosis between 6 and 18 years
    - New diagnosis of CD, defined on the basis of the Porto criteria after thus excluding infectious causes of enteritis.
    - No complicated CD (not stenosing not fistulizing) or not needing surgery for the treatment of simple perianal fistulizing disease.
    - Presence of at least one risk factor of negative prognosis (pernala fistulizing disease, panenteric disease, extention > 60 cm,
    severe growth retardation, severe osteoporosis, hypoalbuminemia or raised CRP (normal valuse x2)
    - Acceptance of contraceptive measures by patients in reproductive ages for reducing the risk of teratogenicity
    - Età alla diagnosi compresa tra i 6 e i 18 anni
    - Nuova diagnosi di MC, definita sulla base dei criteri di Porto, previa pertanto esclusione di cause infettive di enterite.
    - Malattia non complicata (non stenosante non fistolizzante) ovvero che non necessità di intervento chirurgico ad eccezione che
    per il trattamento delle fistole perianali.
    - Presenza di almeno un fattore di rischio per prognosi negativa (malattia perinale fistolizzante,- malattia panenterica, malattia
    estesa per una lunghezza > 60 cm, compromissione grave della crescita (z-score per altezza < -2 DS) dovuta al MC, grave
    osteoporosi (z score < -2 DS), ipoalbuminemia (< 3g/dL) o aumento della proteina C reattiva (2 volte superiore al range di
    normalità).
    - accettazione da parte dei pazienti in età riproduttiva delle misure contraccettive previste per la riduzione del rischio teratogenico
    E.4Principal exclusion criteria
    - infectious enteritis
    - Pregnancy in progress
    - Patients who are breast-feeding
    - Presence of peripheral neuropathy
    - Diagnosis of HIV
    - Diagnosis of tuberculosis
    - Diagnosis of Hepatitis HBV or HCV
    - other infections such as sepsis, abscesses, opportunistic infections in progress or other serious diseases not controlled.
    - Patients undergone treatment with a live vaccine during the 30 days prior to enrollment in the study.
    - Presence of moderate to severe heart failure (Class III / IV NYHA - New York Heart Association).
    - Patients with organ transplants
    - involvement in other clinical trials
    - Riscontro di cause infettive di enterite
    - Gravidanza in corso
    - Pazienti che stanno allattando al seno
    - Presenza di neuropatia periferica
    - Diagnosi di HIV
    - Diagnosi di tubercolosi
    - Diagnosi di Epatite da virus HBV o HCV
    - Infezioni maggiori come sepsi, ascessi, infezioni opportunistiche in corso o altre malattie gravi non controllate.
    - Pazienti sottoposti a trattamento con vaccini vivi nei 30 giorni precedenti l’arruolamento allo studio.
    - Presenza di insufficienza cardiaca da moderata a grave (Classe III/IV NYHA – New York Heart Association).
    - Pazienti con trapianto d’organo
    - Partecipazione ad altri studi sperimentali
    E.5 End points
    E.5.1Primary end point(s)
    - number of patient with mucosal healing at 52 weeks of treatment in the two groups.
    Mucosal healing will be defined by a SES-CD) ≤ 2
    Il numero di pazienti in guarigione mucosale a 52 settimane dall’avvio della terapia nei due gruppi di trattamento (talidomide versus terapia standard).
    La guarigione mucosale verrà definita in base ad un Simplified Endoscopic Activity Index for CD (SES-CD) ≤ 2
    E.5.1.1Timepoint(s) of evaluation of this end point
    52 weeks
    52 settimane
    E.5.2Secondary end point(s)
    - number of patient who exit from the protocol and need to modify or increase drug treatment at 12 and 52 weeks
    - Number of hospitalization at 52 weeks
    - number of patient who need to undergo chirurgic procedures at 52 weeks
    - flogosis indexes at 12 and 52 weeks
    - body mass index; height-for-age z score; weight-for-age z score
    - growth and nutritional indexes at 12 and 52 weeks (body mass index; height-for-age z score; weight-for-age z score)
    - Bone mineralization at 52 weeks
    - Adverse events at 12 and 52 weeks
    - Direct and indirect costs at 52 weeks
    assessment of genetics of CD,
    immunological mechanisms of CD, mechanisma of action of thalidomide, pharmacokinetics, pharmacogenomics and metabolomics
    of thalidomide
    - Numero di pazienti con risposta clinica (riduzione wPCDAI > 50% rispetto ai valori basali) e in remissione (wPCDAI < 12.5) a 12 e a 52 settimane o oltre in caso di prosecuzione del follow up, tramite wPCDAI (allegato 3) (65,66,67)
    - Numero di pazienti con necessità di uscita dal protocollo e modifica o aumento del numero di farmaci impiegati a 12 e 52 settimane.
    - Numero di ospedalizzazioni a 52 settimane.
    - Numero di pazienti con necessità di chirurgia a 52 settimane.
    - Andamento degli indici laboratoristici di infiammazione a 12 e a 52 settimane.
    - Andamento degli indicatori nutrizionali e di crescita a 12 e a 52 settimane.Tra i parametri rilevati verranno inclusi: body mass index; height-or-age z score; weight-for-age z score
    - Andamento dello stato di mineralizzazione ossea all’esordio e a 52 settimane.
    - Numero e tipo di effetti avversi delle terapie a 12 e 52 settimane.
    - Costi diretti e indiretti delle terapie a 52 settimane.
    - Valutazione dati sulla genetica del MC, i dati sui meccanismi immunologici del MC, dati sui
    meccanismi d’azione della talidomide nel MC, i dati di farmacocinetica della talidomide nel MC, i dati di farmacogenomica della talidomide nel MC, dati di metabolomica della talidomide nel MC
    E.5.2.1Timepoint(s) of evaluation of this end point
    52 weeks
    52 settimane
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic Yes
    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 Yes
    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 Yes
    E.8.1.7.1Other trial design description
    In singolo cieco per cecità del valutatore degli esiti
    blindness of outcome assessor
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    Ultima visita dell' ultimo soggetto
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    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: 124
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 62
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 62
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Pediatric patients
    Pazienti pediatrici
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state124
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 124
    F.4.2.2In the whole clinical trial 124
    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)
    Patients, who had befefits with talidomide, will continue the therapy
    Si provvederà a continuare la terapia con talidomide nei pazienti che ne hanno tratto beneficio
    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 Decision2017-03-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-03-22
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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