Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43857   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2021-000824-36
    Sponsor's Protocol Code Number:HLSC-UCD-01
    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:2021-08-16
    Trial 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 number2021-000824-36
    A.3Full title of the trial
    An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in newborns and infants up to the age of 12 months with neonatal and infantile onset of urea cycle disorders (UCD) using a 15N ammonium chloride tracer compared to newborns and infants without UCD.
    Studio clinico di fase I in aperto, controllato e multicentrico volto a valutare la capacità di ureogenesi nei neonati e nei bambini fino a 12 mesi di età affetti da disturbi del ciclo dell'urea (UCD) a esordio neonatale e infantile mediante un tracciante a base di cloruro di ammonio 15N rispetto ai neonati e ai bambini senza UCD.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    15N ammonium chloride ureagenesis validation clinical trial
    Studio clinico di convalida dell'ureogenesi mediante cloruro di ammonio 15N
    A.3.2Name or abbreviated title of the trial where available
    reLiver-1
    reLiver-1
    A.4.1Sponsor's protocol code numberHLSC-UCD-01
    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 SponsorUnicyte AG
    B.1.3.4CountrySwitzerland
    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 organisationUnicyte AG
    B.5.2Functional name of contact pointCEO
    B.5.3 Address:
    B.5.3.1Street AddressAawasserstrasse 2
    B.5.3.2Town/ cityOberdorf NW
    B.5.3.3Post code6370
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number0041416198949
    B.5.6E-mailf.jehle@unicyte.ch
    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 name15N ammonium chloride (15NH4Cl)
    D.3.2Product code [na]
    D.3.4Pharmaceutical form Tablet and powder for oral solution
    D.3.4.1Specific paediatric formulation No
    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 INNAmmonium (15N) chloride
    D.3.9.1CAS number 39466-62-1
    D.3.9.2Current sponsor codeAmmonium (15N) chloride
    D.3.9.3Other descriptive nameAmmonium (15N) chloride
    D.3.9.4EV Substance CodeSUB222811
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    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 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 Yes
    D.3.11.13.1Other medicinal product typeStable isotope diagnostic tracer 15N ammonium chloride (15NH4Cl)
    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
    Subject has a genetically confirmed diagnosis of any of the following
    urea cycle disorders: ASS, CPS1, ASL, OTC
    Subjects without UCD can have other stable illness that not interfere
    with the clinical trial according to the investigator judgement
    Il soggetto ha diagnosi geneticamente confermata di uno qualunque dei seguenti disturbi del ciclo dell'urea: ASS, CPS1, ASL, OTC
    I soggetti senza UCD possono avere un'altra malattia stabile che, secondo il parere dello sperimentatore, non interferisce con la sperimentazione clinica
    E.1.1.1Medical condition in easily understood language
    Urea formation disorders
    Disturbi della formazione 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 20.1
    E.1.2Level PT
    E.1.2Classification code 10080020
    E.1.2Term Urea cycle disorder
    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 intra-subject inter-occasion variability of ureagenesis inneonates with a confirmed diagnosis of UCD over a period of up to 36 weeks using a 15NH4Cl diagnostic tracer.
    Valutare la variabilità intra-individuale e inter-occasione dell'ureogenesi in neonati con diagnosi confermata di UCD nell'arco di un periodo massimo di 36 settimane con l'ausilio di un tracciante per uso diagnostico a base di 15NH4CI
    E.2.2Secondary objectives of the trial
    To assess the intra-subject inter-occasion
    variability of ureagenesis in infants with a confirmed diagnosis of UCD
    over a period of up to 36 weeks using a 15NH4Cl diagnostic tracer. •To
    assess disease stability in neonates and infants with a confirmed
    diagnosis of UCD over a period of up to 36 weeks.
    Secondary Objcs: •To assess the ureagenesis and its variability in
    neonates and infants without UCD over a period of 12 weeks using a
    15NH4Cl diagnostic tracer. •To compare the inter-subject variability and
    extent of impairment of ureagenesis in subjects with UCD with the
    ureagenesis capacity in neonates and infants without UCD using a
    15NH4Cl diagnostic tracer. •To correlate the ureagenesis, in both
    subjects with and without UCD, as determined by the 15NH4Cl tracer
    assay, with blood levels of ammonia, citrulline and glutamine
    Safety Objectives: To assess the safety and tolerability of 15NH4Cl
    diagnostic tracer in neonates and infants with and without UCD.
    Valutare l'ureogenesi e la sua variabilità nei neonati e negli infanti senzaUCD nell'arco di un periodo di 12settimane con l'ausilio di un tracciante per uso diagnostico a base di 15NH4Cl
    Valutare la stabilità della malattia nei neonati e negli infanti con diagnosi confermata di UCD nell'arco di un periodo massimo di 36 settimane
    Valutare l'ureogenesi e la sua variabilità nei neonati e negli infanti senza UCD nell'arco di un periodo di 12 settimane con l'ausilio di un tracciante per uso diagnostico a base di 15NH4Cl
    Confrontare la variabilità inter-individuale e l'estensione della compromissione dell'ureogenesi nei soggetti con UCD rispetto alla capacità di ureogenesi nei neonati e negli infanti senza UCD con l'ausilio di un tracciante per uso diagnostico a base di 15NH4Cl
    Correlare l'ureogenesi, nei soggetti con e senza UCD, come determinato dal test con il tracciante per uso diagnostico a base di 15NH4Cl, con le concentrazioni ematiche di ammoniaca, citrullina e glutammina
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A subject must meet ALL of the following criteria at Screening to be
    eligible for this trial, with the exception of criteria 1 and 2 which do not
    apply to subjects without UCD and criterium 3 that only applies for
    subjects without UCD

    Only for subjects with UCD
    1. Subject has a genetically confirmed diagnosis of any of the following
    urea cycle disorders: ASS, CPS1, ASL, OTC. Note: All subjects should
    have genotyping information available, however if an exact genetic
    diagnosis is not available, diagnosis of the UCD sub-type may be
    confirmed by well accepted biochemical parameters
    2. Subject has neonatal or infantile onset of UCD signs and symptoms
    within the first 12 months of life; or subjects who have a family history
    of UCD and are asymptomatic after birth due to a therapeutic regimen
    started directly after birth;
    Only for subjects without UCD
    3. Subjects without UCD can have other stable illness that does not
    interfere with the clinical trial according to the investigator judgement;
    For all subjects (with and without UCD)
    4. Male and female subjects aged up to 12 months, inclusive;
    5. Subject has a body weight within the 5-95 percentile of the
    corresponding age according to the WHO Child Growth Standards 2006;
    6. Subject has stable clinical conditions (any acute condition needs to
    be stabilised/treated before inclusion);
    7. The parent(s) / legal representative(s) agrees that the subject will
    not participate in any interventional clinical trial with an investigational
    drug suspected of having an interaction with the urea cycle or 15NH4Cl
    diagnostic tracer for the duration of the trial until the final follow-up
    telephone call;
    8. Ability and willingness of the parent(s) / legal representative(s) to
    comply with the protocol requirements, including ability to bring the
    subject to the scheduled trial visits;
    9. Written informed consent by the parent(s) / legal representative(s)
    of the subj
    Per essere idonei a questo studio, un soggetto deve soddisfare TUTTI i seguenti criteri all'atto della selezione (screening), fatta eccezione per i criteri 1 e 2 che non si applicano ai soggetti senza UCD e al criterio 3 che si applica solo ai soggetti senza UCD:
    Solo per i soggetti con UCD
    1. Il soggetto ha diagnosi geneticamente confermata di uno qualunque dei seguenti disturbi del ciclo dell'urea: ASS, CPS1, ASL, OTC. Nota: Tutti i soggetti devono disporre dei dati sulla genotipizzazione; tuttavia, se non è disponibile una diagnosi genetica esatta, la diagnosi del sottotipo di UCD può essere confermata sulla base dei parametri biochimici accettati descritti alla Sezione 10.3.11;
    2. Il soggetto ha segni e sintomi di UCD nei primi 12 mesi di vita; oppure il soggetto ha storia familiare di UCD ed è asintomatico dopo la nascita per via di un regime terapeutico avviato direttamente dopo la nascita;
    Solo per i soggetti senza UCD
    3. I soggetti senza UCD possono avere un'altra malattia stabile che, secondo il parere dello sperimentatore, non interferisce con la sperimentazione clinica;
    Per tutti i soggetti (con e senza UCD)
    4. Soggetti di sesso maschile e femminile di età fino ai 12 mesi (inclusi);
    5. Il soggetto ha un peso corporeo entro il 5-95 percentile dell'età corrispondente, secondo gli standard di crescita dell'OMS per il 2006 (Child Growth Standards 2006; Appendice 2);
    6. Il soggetto presenta condizioni cliniche stabili (qualunque condizione acuta deve essere stabilizzata/trattata prima dell'ammissione allo studio);
    7. Il genitore/i genitori/tutore legale convengono che il soggetto non parteciperà ad alcuna sperimentazione clinica interventistica con un farmaco sperimentale che mostra un'interazione sospetta con il ciclo dell'urea o con il tracciante per uso diagnostico a base di 15NH4Cl per la durata dello studio fino all'ultima telefonata di follow-up;
    8. Capacità e disponibilità del genitore/dei genitori o del tutore legale di conformarsi ai requisiti del protocollo, inclusa la capacità di accompagnare il soggetto alle visite programmate previste dallo studio;
    9. Consenso informato scritto da parte del genitore/dei genitori o del tutore legale del soggetto
    E.4Principal exclusion criteria
    A subject must meet NONE of the following criteria at Screening to be
    eligible for this trial, with the exception of criterion 1 which does not
    apply to subjects with UCD:
    Only for subjects with UCD
    1. Subject has any suspected UCD of any sub-type. Note: subjects
    suspected of having a UCD of any sub-type, but without either
    confirmatory genotyping information or a typical biochemical diagnostic
    pattern for any UCD gene defect, will not be enrolled in this trial.
    For all subjects (with and without UCD)
    2. Subject is a premature neonate (up to 37 gestation weeks not
    completed);
    3. Subject is in a period of significant post-natal weight drop based on
    the judgement of the investigator ;
    4. Subject has received any investigational compound within 30 days
    (or 5 half-lives, whichever is longer) prior to first dose of diagnostic
    tracer and according to the investigator judgement could interfere with
    the clinical trial;
    5. Subject has any other acute severe / other genetic / life limiting
    disorder that would interfere with ethical and/or medical standards in
    the conduct or follow up of the trial.
    6. Subject has acute liver failure, clinical or radiological evidence of
    liver fibrosis or cirrhosis, or presents a hepatic or extrahepatic
    malignancy
    Per essere idoneo a partecipare allo studio, un soggetto NON deve soddisfare alcuno dei seguenti criteri, fatta eccezione per il criterio 1 che non si applica ai soggetti con UCD:
    Solo per i soggetti con UCD
    1. Il soggetto ha una sospetta UCD di qualunque sottotipo. Nota: I soggetti che sospettano di avere un UCD di qualunque sottotipo, ma non confermato con metodo di genotipizzazione e privo di pattern diagnostico biochimico tipo per qualunque difetto genetico dell'UCD (Sezione 10.3.11), non saranno ammessi a questo studio.
    Per tutti i soggetti (con e senza UCD)
    2. Il soggetto è un neonato pretermine (fino a 37 settimane di gestazione non completate);
    3. Il soggetto, a giudizio dello sperimentatore, è in un periodo di significativo calo ponderale post-natale;
    4. Il soggetto ha ricevuto un composto sperimentale entro 30 giorni (o 5 emivite, a seconda di quale periodo sia più lungo) precedentemente alla prima dose del tracciante per uso diagnostico e, a giudizio dello sperimentatore, ciò potrebbe interferire con lo studio clinico;
    5. Il soggetto ha qualunque altro disturbo grave acuto/genetico/potenzialmente letale che potrebbe interferire con gli standard etici e/o medici della conduzione o del follow-up dello studio;
    6. Il soggetto soffre di insufficienza epatica acuta, ha evidenza clinica o radiologica di fibrosi o cirrosi epatica o presenta una neoplasia epatica o extraepatica.
    E.5 End points
    E.5.1Primary end point(s)
    The intra-subject inter-occasion variability of the [15N] urea
    enrichment area under the plasma concentration-time curve over 2
    hours (AUC0-2) following up to 4 15NH4Cl diagnostic tracer
    administrations over a 36-week period to subjects with neonatal-onset
    UCD.
    La variabilità intra-individuale e inter-occasione dell'area di arricchimento dell'urea [15N] sotto la curva concentrazione plasmatica-tempo nell'arco di 2 ore (AUC0-2) dopo un massimo di 4 somministrazioni di tracciante per uso diagnostico a base di 15NH4Cl in un periodo di 36 settimane ai soggetti con UCD a esordio neonatale
    E.5.1.1Timepoint(s) of evaluation of this end point
    Over a 36-week period
    In un periodo di 36 settimane
    E.5.2Secondary end point(s)
    The intra-subject inter-occasion variability of the [15N] urea
    enrichment area under the plasma concentration-time curve over 2
    hours (AUC0-2) following up to 4 15NH4Cl diagnostic tracer
    administrations over a 36-week period to subjects with infantile-onset
    UCD.
    •The number of subjects with UCD demonstrating stable disease at
    Weeks 0, 12, 24 and 36.
    •The blood concentrations of ammonia, glutamine and citrulline up to 2
    hours following
    o up to 4 15NH4Cl diagnostic tracer administrations to subjects with
    UCD (Weeks 0, 12, 24 and 36).
    o up to 2 15NH4Cl diagnostic tracer administrations to subjects without
    UCD (Weeks 0 and 12).
    •The number of hyperammonaemia events and crises experienced since
    the previous trial visit at Weeks 12, 24 and 36 (subjects with UCD only).
    •The number of subjects following a stable protein diet, with no
    unplanned adjustments since the previous trial visit, at Weeks 12, 24
    and 36 (subjects with UCD only).
    Secondary Endpoints:
    •The intra-subject inter-occasion variability of the [15N] urea
    enrichment area under the plasma concentration-time curve over 2
    hours (AUC0-2) following
    o up to 4 15NH4Cl diagnostic tracer administrations over a 36-week
    period to subjects with infantile-onset UCD.
    o up to 2 15NH4Cl diagnostic tracer administrations over a 12-week
    period to subjects without UCD.
    •The inter-subject variance of the [15N] urea enrichment area under
    the plasma concentration-time curve over 2 hours (AUC0-2) following
    each 15NH4Cl diagnostic tracer administration
    o to subjects with neonatal-onset UCD (Weeks 0, 12, 24 and 36).
    o to subjects with infantile-onset UCD (Weeks 0, 12, 24 and 36).
    o to subjects without UCD (Weeks 0 and 12).
    •The inter-subject inter-occasion variability of the [15N] urea
    enrichment area under the plasma concentration-time curve over 2
    hours (AUC0-2) following
    o up to 4 15NH4Cl diagnostic tracer administrations over a 36-week
    period to subjects with neonatal-onset UCD.
    o up to 4 15NH4Cl diagnostic tracer administrations over a 36-week
    period to subjects with infantile-onset UCD.
    o up to 2 15NH4Cl diagnostic tracer administrations over a 12-week
    period to subjects without UCD.
    The inter-subject variance of the area under the blood concentrationtime curve over 2 hours (AUC0-2) for ammonia, citrulline and glutamine
    following
    o up to 4 15NH4Cl diagnostic tracer administrations over a 36-week
    period to subjects with UCD.
    o up to 2 15NH4Cl diagnostic tracer administrations over a 12-week
    period to subjects without UCD
    Safety Endpoints:
    •Adverse events (AEs); physical, neurological and child development
    examination findings; vital signs; clinical laboratory tests; biochemical
    parameters; plasma amino acid levels; frequency and severity of
    hyperammonaemia events and crises; and compliance to protein intake
    and concomitant medication.
    La variabilità intra-individuale e inter-occasione dell'area di arricchimento dell'urea [15N] sotto la curva concentrazione plasmatica-tempo nell'arco di 2 ore (AUC0-2) dopo un massimo di 4 somministrazioni di tracciante per uso diagnostico a base di 15NH4Cl in un periodo di 36 settimane ai soggetti con UCD a esordio infantile.
    Il numero di soggetti con UCD che evidenziano malattia stabile alle settimane 0, 12, 24 e 36.
    Le concentrazioni ematiche di ammoniaca, glutammina e citrullina fino a 2 ore dopo
    o un massimo di 4 somministrazioni di tracciante per uso diagnostico a base di 15NH4Cl ai soggetti con UCD (settimane 0, 12, 24 e 36)
    o un massimo di 2 somministrazioni di tracciante per uso diagnostico a base di 15NH4Cl ai soggetti senza UCD (settimane 0 e 12).
    Il numero di eventi di iperammoniemia e di crisi verificatisi dopo l'ultima visita programmata prevista dallo studio alle settimane 12, 24 e 36 (solo per i soggetti con UCD).
    Il numero di soggetti sottoposti a dieta ipoproteica stabile, che non hanno richiesto aggiustamenti imprevisti dopo l'ultima visita programmata prevista dallo studio, alle settimane 12, 24 e 36 (solo per i soggetti con UCD)
    Endpoint secondari:
    La variabilità intra-individuale e inter-occasione dell'area di arricchimento dell'urea [15N] sotto la curva della concentrazione plasmatica-tempo nell'arco di 2 ore (AUC0-2) dopo
    o un massimo di 4 somministrazioni di tracciante per uso diagnostico a base di 15NH4Cl in un periodo di 36 settimane ai soggetti con UCD a esordio infantile.
    o un massimo di 2 somministrazioni di tracciante per uso diagnostico a base di 15NH4Cl in un periodo di 12 settimane ai soggetti senza UCD.
    La varianza inter-individuale dell'area di arricchimento dell'urea [15N] sotto la curva concentrazione plasmatica-tempo nell'arco di 2 ore (AUC0-2) dopo ogni somministrazione di tracciante per uso diagnostico a base di 15NH4Cl
    o ai soggetti con UCD a esordio neonatale (settimane 0, 12, 24 e 36).
    o ai soggetti con UCD a esordio infantile (settimane 0, 12, 24 e 36).
    o ai soggetti senza UCD (settimane 0 e 12).
    La variabilità intra-individuale e inter-occasione dell'area di arricchimento dell'urea [ 15N] sotto la curva concentrazione plasmatica-tempo nell'arco di 2 ore (AUC0-2) dopo
    o un massimo di 4 somministrazioni di tracciante per uso diagnostico a base di 15NH4Cl in un periodo di 36 settimane ai soggetti con UCD a esordio neonatale.
    o un massimo di 4 somministrazioni di tracciante per uso diagnostico a base di 15NH4Cl in un periodo di 36 settimane ai soggetti con UCD a esordio infantile.
    o un massimo di 2 somministrazioni di tracciante per uso diagnostico a base di 15NH4Cl in un periodo di 12 settimane ai soggetti senza UCD.
    La varianza inter-individuale dell'area sotto la curva concentrazione ematica-tempo nell'arco di 2 ore (AUC0-2) per la concentrazione di ammoniaca, citrullina e glutammina dopo
    o un massimo di 4 somministrazioni di tracciante per uso diagnostico a base di 15NH4Cl alle settimane 0, 12, 24 e 36 ai soggetti con UCD.
    o un massimo di 2 somministrazioni di tracciante per uso diagnostico a base di 15NH4CI alle settimane 0 e 12 ai soggetti senza UCD
    Endpoint di sicurezza:
    • Eventi avversi; risultati degli esami obiettivi e neurologici, come anche della valutazione dello sviluppo del bambino; segni vitali; test clinici di laboratorio; parametri biochimici; livelli plasmatici degli amminoacidi; frequenza e gravità degli eventi di iperammoniemia e crisi; e modificazioni della dieta ipoproteica e delle terapie concomitanti.
    E.5.2.1Timepoint(s) of evaluation of this end point
    According to the protocol
    In accordo al protocollo
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Ureagenesis assessment using 15N labelled ammonium chloride (15NH4Cl)
    Valutazione dell'ureagenesi utilizzando cloruro di ammonio marcato con 15N (15NH4Cl)
    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 Yes
    E.8.1.1Randomised No
    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 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 Yes
    E.8.2.3.1Comparator description
    I soggetti senza diagnosi o sospetto di UCD saranno arruolati in questo studio come soggetti di cont
    Subjects without a diagnosis or suspicion of UCD will be enrolled in this trial as control subjects
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    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 EEA15
    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
    Israel
    Saudi Arabia
    Turkey
    Austria
    Belgium
    France
    Germany
    Netherlands
    Poland
    Portugal
    Spain
    Switzerland
    United Kingdom
    Czechia
    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 years2
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days15
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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: 2
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 28
    F.1.1.5Children (2-11years) No
    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 Yes
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    newborns, infants up to 12 months
    neonati, bambini fino a 12 mesi
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    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)
    No diagnostic tracer will be provided after the end of the trial.
    Nessun tracciante diagnostico sarà fornito dopo la fine della sperimentazione.
    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 Decision2022-03-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-04-04
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2023-02-13
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Thu Apr 25 07:17:16 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA