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    Summary
    EudraCT Number:2011-004074-28
    Sponsor's Protocol Code Number:HEP001
    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:2013-01-14
    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 number2011-004074-28
    A.3Full title of the trial
    A prospective, open label, multicenter, partially randomized, safety study of one cycle of Promethera HepaStem in Urea Cycle Disorders and Crigler-Najjar Syndrome Patients
    Uno studio sperimentale prospettico, multicentrico, in aperto, parzialmente randomizzato, per valutare la sicurezza di un ciclo con Promethera HepaStem su pazienti pediatrici affetti da disturbi del ciclo dell'™urea (UCD) e da sindrome di Crigler-Najjar (CN).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase I/II trial for evaluating both safety and preliminary efficacy of one cycle of Promethera Hepastem in Urea Cycle Disorders and Crigler-Najjar Syndrome patients
    Uno studio sperimentale di fase I/II per valutare la sicurezza e l’efficacia a livello preliminare di un ciclo con Promethera HepaStem su pazienti affetti da disturbi del ciclo dell’urea e da sindrome di Crigler-Najjar.
    A.3.2Name or abbreviated title of the trial where available
    HEP 001
    HEP 001
    A.4.1Sponsor's protocol code numberHEP001
    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 SponsorPROMETHERA BIOSCIENCES
    B.1.3.4CountryBelgium
    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 supportPromethera Biosciences
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPromethera Biosciences
    B.5.2Functional name of contact pointWouters Vinciane
    B.5.3 Address:
    B.5.3.1Street AddressRUE GRANBONPRE, 11
    B.5.3.2Town/ cityMONT-SAINT-GUIBERT
    B.5.3.3Post code1435
    B.5.3.4CountryBelgium
    B.5.4Telephone number32 10 394311
    B.5.5Fax number32 10 394301
    B.5.6E-mailvinciane.wouters@promethera.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/3/07/506 and EU/3/08/530
    D.3 Description of the IMP
    D.3.1Product namePromethera HepaStem
    D.3.2Product code HHALPC
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOther use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHeterologous Human Adult Liver-derived Progenitor Cells (HHALPC)
    D.3.9.2Current sponsor codeHHALPC
    D.3.10 Strength
    D.3.10.1Concentration unit million organisms/ml million organisms/millilitre
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number5
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product Yes
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberSomatic Cell Therapy Product EMA/391931/2011
    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
    Crigle-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucoronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are six desorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia, argininosuccinic aciduria and argininemia.
    La sindrome di Crigler-Najjar si associa a un deficit epatico dell’attività di glucuronil-transferasi della bilirubina e nel periodo neonatale si evidenzia con intense manifestazioni d’itterizia. I disturbi del ciclo dell’urea sono errori innati del metabolismo associati alla mancanza di sei enzimi.
    E.1.1.1Medical condition in easily understood language
    Liver meabolic diseases: Crigler-Najjar disease and Urea Cycle disorders
    Malattie Metaboliche del fegato: La sindrome di Crigler-Najjar e i disturbi del ciclo dell’urea
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.1
    E.1.2Level LLT
    E.1.2Classification code 10013373
    E.1.2Term Disorders of urea cycle metabolism
    E.1.2System Organ Class 100000004861
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.1
    E.1.2Level PT
    E.1.2Classification code 10011386
    E.1.2Term Crigler-Najjar syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.1
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.1
    E.1.2Level LLT
    E.1.2Classification code 10021601
    E.1.2Term Inborn error of metabolism NOS
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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 safety of one cycle of HHALPC infusions up to six months in paediatric patients suffering from CN or UCD in terms of clinical status, portal-vein hemodynamics, morphology of the liver, de novo detection of circulating anti-HLA antibodies, and/or other immune related markers as well as Serious Adverse Events (SAEs) and clinically significant Adverse Events (AEs) related to infusion.
    Determinare la sicurezza di un ciclo di infusione di cellule HHALPC, entro un periodo di sei mesi, in pazienti pediatrici affetti da sindrome di CN o disturbi UCD, nell’ambito della valutazione dello stato clinico, dell’emodinamica della vena porta, della morfologia del fegato, della presenza de novo di anticorpi anti-HLA in circolo, e/o di altri marcatori immunologici associati, nonché della valutazione di eventi avversi gravi [Serious Adverse Events (SAE)] e di eventi avversi significativi [Adverse Events (AE)] associati all’infusione.
    E.2.2Secondary objectives of the trial
    1. To assess the long term safety (up to 12 months) of one cycle of HHALPC infusions in paediatric patients suffering from CN or UCD in terms of clinical status, portal-vein hemodynamics, morphology of the liver, de novo detection of circulating anti-HLA antibodies, and/or other immune related markers as well as SAE's and clinically significant AEs related to infusion. 2. To appraise the efficacy post-infusion of one cycle of HHALPC for each individual patient and for all patients for a minimum of six and up to twelve months as compared to his/her prior medical condition. 3. To investigate the engraftment of HHALPC in the liver of all patients (at 6 month, and optional at 12 month) by quantitative measurement of enzymatic activity on biopsies and/or establishing donor sequences by real-time PCR or in situ hybridization (FISH) or immunohistochemistry.
    1.Determintare la sicurezza a lungo termine (fino a 12 mesi) di un ciclo di infusione di HHALPC in pazienti pediatrici affetti da sindrome di CN o UCD, per valutare lo stato clinico, l’emodinamica della vena porta, la morfologia del fegato, la presenza de novo di anticorpi anti-HLA in circolo e/o di altri marcatori immunologici associati, nonché gli SAE e AE clinicamente significativi associati all’infusione. 2.Valutare l’efficacia post-infusione di un ciclo di HHALPC su ciascun paziente e su tutti i pazienti, per un minimo di sei e fino a dodici mesi, da un confronto con le condizioni cliniche precedenti. 3. Esaminando l’attecchimento di HHALPC nel fegato di tutti i pazienti (dopo 6 mesi e se possibile dopo 12 mesi) misurando l’attività enzimatica su biopsie e/o stabilendo le sequenze del donatore con PCR in tempo reale o con ibridizzazione in sito o immunoistochimica.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    OTHER SUBSTUDIES:
    Sub-study PBMC_Version 2.0_01 August 2012. Ref: HEP001 PBMC ''Peripheral Blood Mononuclear Cells'' Objective: To investigate the kinetics of an immune response following infusion of HHALPC.

    ALTRI SOTTOSTUDI:
    Sottostudio PBMC_versione 2.0_01 Aug 2012. Ref: HEP001 PBMC ''Peripheral Blood Mononuclear Cells''. Obiettivo: esaminare la cinetica di una risposta immune a seguito di iniezione di HHALPC

    E.3Principal inclusion criteria
    General: 1. Subject shows patency of the portal vein and branches, with normal flow velocity in the main portal vein and normal portal vein pressure as confirmed by Doppler ultrasound and accessibility of the portal vein, or respectively, accessibility of the umbilical vein. 2. Subject (if capable of signing) and parents or legal representative have provided a written informed assent/consent. 3. Female subjects of childbearing potential need to have a negative pregnancy test and must agree to use an acceptable method of contraception throughout the study. Crigler-Najjar Syndrome specific: Patient presents with Crigler-Najjar syndrome type 1 and diagnosis must be confirmed by genetic mutation analysis if not available. 4. Patient presents with Crigler-Najjar syndrome type 2 - poorly controlled under phenobarbital treatment, or - experiencing serious impairment in quality of life. Diagnosis must be confirmed by genetic mutation analysis if not available. Urea Cycle Disorders specific : 5. Diagnosis of one of the urea cycle disorders (CPS I D, OCTD, ASSD, ASLD, Arginase deficiency, and NAGSD) - of which the disease is of such severity to warrant liver transplantation or alternatives despite full conservative therapy, or - subject experiencing serious impairment in quality of life despite full conservative therapy. Diagnosis must be confirmed by genetic mutation analysis if not available.
    Generali: 1.Nel soggetto si evidenzia la pervietà della vena porta e dei dotti arteriosi, con velocità di flusso nella norma e valori della pressione della vena porta anch’essi nella norma, così come confermato dall’esame ecodoppler, nonché la possibilità di accedere rispettivamente alla vena porta o alla vena ombelicale. 2.Il soggetto (se in grado di firmare) e i genitori o i rappresentanti legali hanno sottoscritto e fornito un modulo di assenso/consenso informato. 3.I soggetti di sesso femminile e potenzialmente fertili devono presentare un test di gravidanza con risultato negativo e devono acconsentire a utilizzare un adeguato metodo di contraccezione per l’intera durata dello studio. Criteri specifici per la sindrome di Crigler-Najjar: Il paziente presenta una sindrome di Crigler Najjar di tipo 1 e la diagnosi deve essere confermata da un’analisi della mutazione genetica, qualora non disponibile. 4. Il paziente presenta una sindrome di Crigler-Najjar di tipo 2 - non sufficientemente controllata mediante terapia con fenobarbital, oppure - che sia causa di una notevole riduzione della qualità della vita. La diagnosi deve essere confermata da un’analisi della mutazione genetica, qualora non disponibile. Criteri specifici per i disturbi del ciclo dell’urea 5.Diagnosi di uno dei disturbi del ciclo dell’urea (Deficit di CPS I, Deficit di OCT, Deficit di ASS, Deficit di ASL, Deficit di arginase e Deficit di NAGS) - che si presentano con sintomi di gravità tale da giustificare un trapianto di fegato o procedure alternative malgrado la terapia conservativa completa, oppure - che causano al paziente una seria riduzione della qualità della vita malgrado la terapia conservativa completa. La diagnosi deve essere confermata da un’analisi della mutazione genetica, qualora non disponibile.
    E.4Principal exclusion criteria
    1. The subject is 18 years or older at time of screening. 2. The subject presents acute liver failure. 3. The subject presents clinical or radiological evidence of liver fibrosis or cirrhosis 4. The subject presents or has a history of hepatic or extrahepatic malignancy 5. The patient has a non-corrected cardiac malformation. 6. The subject has a known medical or family history of coagulopathy. 7. The subject participates currently in another clinical trial – except disease registry and observational HepaStem study. 8. The subject underwent previous mature liver cell or stem cell transplantation or received an organ liver transplant. 9. The subject has a contraindication to immunosuppressive therapy. 10. The subject has a known hypersensitivity or allergy to the recommended antibiotics to prevent post-operative infections according to institutional guidelines, and basiliximab, solumedrol or tacrolimus unless alternative drugs can be used without risk for the patient. 11. The subject has a known hypersensitivity or allergy to bivalirudin. 12. The subject had or has a renal insufficiency treated by dialysis. 13. The subject requires valproate therapy. 14. The subject has a known hypersensitivity or allergy to contrast agents that cannot be treated adequately. 15. The subject has a thrombosis of the portal vein or persisting impairment of anterograde portal blood flow. 16. The subject has a porto systemic shunt or fistula assessed by Doppler US. 17. For umbilical vein access: The subject has any contraindication for umbilical vein catheterization (eg omphalitis, peritonitis, necrotizing enterocolitis, etc) 18. Any significant condition which in the Investigator’s opinion may interfere with the subject’s optimal participation in the study. 19. Patients with disease of such severity that liver transplantation is an absolute indication. 20. Patients with mild disease severity, easily controlled under standard of care therapy with no recurrent metabolic crises.
    I pazienti non sono ammessi a partecipare allo studio se: 1. Al momento dello screening il paziente ha un’età di 18 anni o più. 2. Il paziente presenta un’insufficienza epatica acuta. 3. Il paziente presenta un’evidenza clinica o radiologica di fibrosi o cirrosi epatica 4. Il paziente presenta o ha un’anamnesi di neoplasia maligna epatica o extraepatica 5. Il paziente presenta una malformazione cardiaca non risolta. 6. Il paziente presenta un’anamnesi clinica riconosciuta o un’anamnesi familiare di coagulopatia. 7. Il paziente sta partecipando al momento a un’altra sperimentazione clinica – fatta eccezione per un registro della malattia e uno studio osservazionale su HepaStem. 8. Il paziente è stato precedentemente sottoposto a trapianto di cellule epatiche mature o di cellule staminali o ha ricevuto un trapianto dell’organo del fegato. 9. Il paziente presenta controindicazioni per la terapia immunosoppressiva. 10. Il paziente presenta un’ipersensibilità o allergia riconosciuta agli antibiotici indicati a prevenzione delle infezioni post-operatorie, così come sancito dalle linee guida istituzionali, e allo basiliximab, solumedrol o tacrolimus a meno che non possano essere utilizzati farmaci alternativi senza alcun rischio per il paziente. 11. Il paziente presenta un’ipersensibilità riconosciuta o un’allergia al bivalirudin. 12. Il paziente ha avuto o ha al momento un’insufficienza renale curata con dialisi. 13. Il paziente necessita di terapia con valproato. 14. Il paziente presenta un’ipersensibilità riconosciuta o un’allergia ad agenti di contrasto che non può essere curata adeguatamente. 15. Il paziente presenta una trombosi della vena porta o un deficit persistente del flusso anterogrado della vena porta. 16. Il paziente presenta uno shunt porto sistemico o fistola, accertato con esame ecodoppler. 17. Per l’accesso alla vena ombelicale: Il paziente presenta una qualche controindicazione per la cateterizzazione della vena ombelicale (ad es. onfalite, peritonite, enterocolite necrotizzante etc.). 18. Una qualsiasi condizione che secondo l’opinione dello sperimentatore potrebbe compromettere una partecipazione ottimale del paziente allo studio. 19. Pazienti affetti da patologie gravi per le quali è assolutamente indicato il trapianto di fegato. 20. Pazienti affetti da patologie di media entità, che possono essere tenute sotto controllo con terapie di tipo standard, in assenza di ricorrenti crisi metaboliche.
    E.5 End points
    E.5.1Primary end point(s)
    Safety of the technical intervention (infusion of HepaStem in portal vein) common to both indications and to different weight cohorts (short-and mid-term safety).
    Sicurezza della procedura tecnica (infusione di HepaStem nella vena porta) sia dal punto di vista delle indicazioni, sia dal punto di vista delle diverse coorti di peso (sicurezza a lungo e medio termine):
    E.5.1.1Timepoint(s) of evaluation of this end point
    Four time windows will be assessed: 1. Inpatient period : Vo-Vdischarge: reactogenicity of infusion 2. Outpatient period: Vdischarge-V1month: short-term safety of infusion 3. Outpatient period: V1m-V6m: mid-term safety of infusion 4. Overall period: Vo-V6m
    Saranno presi in esame quattro intervalli di tempo: 1. Periodo del ricovero: Vo-Vdimissione: reattogenicità dell’infusione 2. Periodo ambulatoriale: Vdimissione-V1mese: sicurezza a breve termine dell’infusione 3. Periodo ambulatoriale: V1m-V6m: sicurezza a medio termine dell’infusione 4. Periodo complessivo: Vo-V6m
    E.5.2Secondary end point(s)
    - To characterize the safety profile of HepaStem for both indications and for different weight cohorts (long-term safety). - To characterize the preliminary efficacy of HepaStem for both indications and for different weight cohorts (0 -6 months ; 6 -12 months).
    - Delineare il profilo di sicurezza del metodo HepaStem sia riguardo alle indicazioni, sia riguardo alle differenti coorti di peso (sicurezza a lungo termine). - Delineare l’efficacia preliminare del metodo HepaStem sia riguardo alle indicazioni, sia riguardo alle differenti coorti di peso (0-6 mesi; 6-12 mesi).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Assessment of reactogenicity and safety of the treatment during 6 to 12 months post infusion (long-term safety) will be evaluated
    Vagliare la reattogenicità e la sicurezza del trattamento nel corso dei 6 - 12 mesi successivi all’infusione (sicurezza a lungo termine).
    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 Yes
    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) Yes
    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) 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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial3
    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 No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    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
    last patient, last visit
    ultimo paziente, ultima visita
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months12
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months12
    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 Yes
    F.1.1Number of subjects for this age range: 18
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 1
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 9
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    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
    paediatric patients (<18 years old)
    pazienti pediatrici (< 18 anni)
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 18
    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)
    A long term (5years) safety surveillance of the patients following infusion with HepaStem is planned. The surveillance will mimic as much as possible the standard follow-up of the respective diseases (standard of care). The surveillance in the remit of this trial, will end when the patient is undergoing an organ transplant or takes part in another research study.
    Per i pazienti che hanno subito un’infusione con il metodo HepaStem è stato programmato un periodo di monitoraggio di sicurezza di lunga durata (5 anni). Questo monitoraggio vuole simulare per quanto possibile la procedura standard di follow up prevista per i rispettivi disturbi (standard del trattamento). Il monitoraggio nell’ambito del presente studio sperimentale avrà termine qualora il paziente sarà sottoposto a un trapianto d’organo o parteciperà a un altro studio di ricerca.
    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 Decision2012-11-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-12-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-11-04
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