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:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-11
    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 ConcernedSpain - AEMPS
    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.
    Ensayo clínico de fase I, multicéntrico, abierto y controlado para evaluar la capacidad de ureagénesis en recién nacidos y lactantes hasta los 12 meses con inicio neonatal e infantil de trastornos del ciclo de la urea (TCU) utilizando un marcador de cloruro de amonio 15N en comparación con recién nacidos y lactantes sin TCU.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical trial assessing urea formation capacity in babies up to 12 months old using 15N ammonium chloride tracer
    Ensayo clínico de evaluación de la capacidad de formación de urea en bebés de hasta 12 meses mediante el uso de un marcador de cloruro de amonio 15N
    A.3.2Name or abbreviated title of the trial where available
    15N ammonium chloride ureagenesis validation clinical trial
    Ensayo clínico de validación de la ureagénesis mediante cloruro de amonio 15N
    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 supportUnicyte AG
    B.4.2CountrySwitzerland
    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 number+41 (41) 619 89 49
    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.3.4Pharmaceutical form 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 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 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
    El paciente tiene un diagnóstico confirmado genéticamente de cualquiera de los siguientes trastornos del ciclo de la urea: ASS, CPS1, ASL, OTC
    Los pacientes sin TCU pueden tener otras enfermedades estables que no interfieran con el ensayo clínico según el criterio del investigador
    E.1.1.1Medical condition in easily understood language
    Urea formation disorders
    Trastornos de la formación de 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 in neonates and infants with a confirmed diagnosis of UCD over a period of up to 36 weeks using a 15NH4Cl diagnostic tracer.
    Evaluar la variabilidad intrasujeto de la ureagénesis en distintos momentos en neonatos y lactantes con diagnóstico confirmado de TCU durante un período de hasta 36 semanas utilizando un marcador diagnóstico, 15NH4Cl.
    E.2.2Secondary objectives of the trial
    Key Secondary Objective:
    •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.
    Objetivos secundarios:
    •Evaluar la ureagénesis y su variabilidad en neonatos y lactantes sin TCU durante un período de 12 semanas utilizando un marcador diagnóstico, 15NH4Cl.
    •Comparar la variabilidad intersujeto y el grado de alteración de la ureagénesis en sujetos con TCU con la ureagénesis en neonatos y lactantes sin TCU utilizando un marcador diagnóstico, 15NH4Cl.
    •Correlacionar la ureagénesis, en sujetos con y sin TCU, según lo determinado por el ensayo con el marcador 15NH4Cl, con las concentraciones sanguíneas de amoniaco, citrulina y glutamina.
    Objetivos de seguridad:
    •Evaluar la seguridad y la tolerabilidad del marcador diagnóstico 15NH4Cl en neonatos y lactantes con y sin TCU.
    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 not interfere with the clinical trial according to the investigator judgement;
    For all subjects (with and without UCD)
    4. Male and female subjects aged 0 to 12 months, inclusive;
    5. Subject with 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 subject.
    Un sujeto debe cumplir TODOS los siguientes criterios en la selección para ser elegible para la participación en este ensayo, con la excepción de los criterios 1 y 2, que no son aplicables para los sujetos sin TCU y el criterio 3 que solo se aplica a los sujetos sin TCU:
    Solo para los sujetos con TCU
    1.El sujeto tiene un diagnóstico confirmado genéticamente de cualquiera de los siguientes trastornos del ciclo de urea: deficiencia de ASS, deficiencia de CPS1, deficiencia de ASL, deficiencia de OTC. Nota: Todos los sujetos deben tener información disponible sobre el genotipado; no obstante, si no se dispone de un diagnóstico genético exacto, el diagnóstico del subtipo de TCU puede confirmarse mediante los parámetros bioquímicos bien aceptados
    2.El sujeto presenta inicio neonatal o infantil de los signos y síntomas del TCU en los 12 primeros meses de vida, o los sujetos tienen antecedentes familiares de TCU y son asintomáticos después del nacimiento debido a un régimen terapéutico iniciado inmediatamente después del nacimiento;
    Solo para los sujetos sin TCU
    3.Los sujetos sin TCU pueden presentar otra enfermedad estable que no interfiera con el ensayo clínico según el criterio del investigador;
    Para todos los sujetos (con y sin TCU)
    4.Sujetos de ambos sexos de 0 a 12 meses de edad, inclusive;
    5.Sujeto con un peso corporal dentro del percentil 5-95 de la edad correspondiente según el Patrón Internacional de Crecimiento Infantil de la OMS de 2006
    6.El sujeto presenta afecciones clínicas estables (cualquier enfermedad aguda debe estabilizarse/tratarse antes de la inclusión).
    7.Los padres/representantes legales aceptan que el sujeto no participe en ningún ensayo clínico de intervención con un medicamento en fase de investigación del que se sospeche una interacción con el ciclo de urea o el marcador diagnóstico 15NH4Cl mientras dure el ensayo hasta la llamada telefónica final de seguimiento.
    8.Capacidad y disposición de los padres/representantes legales para cumplir los requisitos del protocolo, incluida la capacidad de llevar al sujeto a las visitas programadas del ensayo.
    9.Consentimiento informado por escrito por parte de los padres/representantes legales del sujeto.
    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;
    4. Subject as 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 as 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 as acute liver failure, clinical or radiological evidence of liver fibrosis or cirrhosis, or presents a hepatic or extrahepatic malignancy.
    Un sujeto no debe cumplir NINGUNO de los siguientes criterios en la selección para ser elegible para la participación en este ensayo, con la excepción del criterio 1, que no es aplicable para los sujetos con TCU:
    Solo para los sujetos con TCU
    1.El sujeto tiene presuntamente un TCU de cualquier subtipo. Nota: No se incluirá en este ensayo a sujetos en los que se sospeche la presencia de un TCU de cualquier subtipo, pero para los que no se disponga de información confirmatoria de genotipado ni de un patrón diagnóstico bioquímico típico de cualquier defecto en los genes afectados en caso de TCU
    Para todos los sujetos (con y sin TCU)
    2.El sujeto es un recién nacido prematuro (no ha completado 37 semanas de gestación).
    3.El sujeto está en un período de descenso considerable del peso posnatal según el criterio del investigador.
    4.El sujeto ha recibido cualquier compuesto en fase de investigación en los 30 días (o 5 semividas, lo que sea más largo) previos a la primera dosis del marcador diagnóstico que, según el criterio del investigador, podría interferir en el ensayo clínico.
    5.El sujeto presenta cualquier otro trastorno agudo grave, o genético o que acorte la vida que haría que la realización o el seguimiento del ensayo contraviniese las normas éticas o médicas.
    6.El sujeto presenta insuficiencia hepática aguda, signos clínicos o radiológicos de fibrosis o cirrosis hepática, o una neoplasia maligna hepática o extrahepática.
    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.
    •Variabilidad intrasujeto entre distintos momentos del área de enriquecimiento de la urea [15N] bajo la curva de la concentración plasmática en el tiempo a lo largo de 2 horas (ABC0-2) en sujetos dentro de cada una de las 4 deficiencias enzimáticas diferentes y con TCU de inicio neonatal o bien infantil según lo determinado con el marcador diagnóstico 15NH4Cl durante un período de 36 semanas.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Over a 36-week period
    Durante un periodo de 36 semanas
    E.5.2Secondary end point(s)
    Key Secondary Endpoints:
    •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 concentration-time 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.
    Criterios secundarios de valoración:
    •El número de sujetos con TCU que demuestran una enfermedad estable en las Semanas 0, 12, 24 y 36.
    •Las concentraciones sanguíneas de amoníaco, glutamina y citrulina hasta 2 horas después
    o hasta 4 administraciones del marcador diagnóstico 15NH4Cl a sujetos con TCU (semanas 0, 12, 24 y 36).
    o hasta 2 administraciones del marcador diagnóstico 15NH4Cl a sujetos sin TCU (semanas 0 y 12).
    •Número de episodios y crisis de hiperamonemia experimentados desde la visita previa del ensayo en las semanas 12, 24 y 36 (solo sujetos con TCU).
    •Número de sujetos que siguen una dieta estable en proteínas, sin ajustes no programados desde la visita anterior del ensayo, en las semanas 12, 24 y 36 (solo sujetos con TCU).
    Criterios secundarios de valoración:
    •Variabilidad intrasujeto entre distintos momentos del área de enriquecimiento de la urea [15N] bajo la curva de la concentración plasmática en el tiempo a lo largo de 2 horas (ABC0-2) después de
    o hasta 4 administraciones del marcador diagnóstico 15NH4Cl durante un período de 36 semanas a sujetos con TCU de inicio infantil.
    o hasta 2 administraciones del marcador diagnóstico 15NH4Cl durante un periodo de 12 semanas a sujetos sin TCU.
    •Varianza intersujeto del área de enriquecimiento de la urea [15N] bajo la curva de la concentración plasmática en el tiempo durante 2 horas (ABC0-2) después de cada administración del marcador diagnóstico 15NH4Cl
    o a sujetos con TCU de inicio neonatal (semanas 0, 12, 24 y 36).
    o a sujetos con TCU de inicio infantil (semanas 0, 12, 24 y 36).
    o a sujetos sin TCU (semanas 0 y 12).
    •Variabilidad intersujeto entre distintos momentos del área de enriquecimiento de la urea [15N] bajo la curva de la concentración plasmática en el tiempo a lo largo de 2 horas (ABC0-2) después de
    o hasta 4 administraciones del marcador diagnóstico 15NH4Cl durante un período de 36 semanas a sujetos con TCU de inicio neonatal.
    o hasta 4 administraciones del marcador diagnóstico 15NH4Cl durante un período de 36 semanas a sujetos con TCU de inicio infantil.
    o hasta 2 administraciones del marcador diagnóstico 15NH4Cl durante un periodo de 12 semanas a sujetos sin TCU.
    •Varianza entre sujetos del área bajo la curva de la concentración sanguínea en el tiempo durante 2 horas (ABC0-2) para amoníaco, citrulina y glutamina después de
    o hasta 4 administraciones del marcador diagnóstico 15NH4Cl en las semanas 0, 12, 24 y 36 a sujetos con TCU.
    o hasta 2 administraciones del marcador diagnóstico 15NH4Cl en las semanas 0 y 12 a sujetos sin TCU.
    Criterios de valoración de la seguridad:
    •Acontecimientos adversos (AA); hallazgos en las exploraciones de desarrollo físico, neurológico e infantil; constantes vitales; análisis clínicos; parámetros bioquímicos; niveles plasmáticos de aminoácidos; frecuencia e intensidad de los episodios y crisis de hiperamonemia; y cambios en el contenido de proteínas en la dieta proteica y la medicación concomitante.
    E.5.2.1Timepoint(s) of evaluation of this end point
    According to the protocol
    Según el protocolo
    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)
    Evaluación de la ureagénesis mediante cloruro de amonio marcado 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
    Los sujetos sin diagnóstico o sospecha de TCU se inscribirán en este ensayo como sujetos de control
    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.8.5The 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 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
    Israel
    Saudi Arabia
    Turkey
    Austria
    Belgium
    France
    Germany
    Italy
    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
    Última visita ultimo paciente (UVUP)
    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.1.1Number 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) 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
    recién nacidos, bebés hasta 12 meses
    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.
    No se proporcionará ningún marcador de diagnóstico después de la finalización del ensayo.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-12-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-26
    P. End of Trial
    P.End of Trial StatusOngoing
    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-Wed Apr 24 09:32:19 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA