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    Summary
    EudraCT Number:2016-003023-30
    Sponsor's Protocol Code Number:401GSDIA01
    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:2018-06-27
    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 number2016-003023-30
    A.3Full title of the trial
    A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease Type Ia (GSDIa)
    Estudio de fase 1/2, abierto, de seguridad y búsqueda de dosis de la transferencia del gen de la glucosa-6-fosfatasa (G6Pasa) mediada por el virus adenoasociado (AAV) de serotipo 8 (AAV8) en adultos con glucogenosis de tipo Ia (GSD-Ia)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An early-phase clinical study of a vector transferring the gene for Glucose-6-Phosphatase (G6Pase) in adults with Glycogen Storage Disease Type Ia.
    Estudio de fase temprana de un vector que transfiere el gen de la glucosa-6-fosfatasa (G6Pasa) ) en adultos con glucogenosis de tipo Ia
    A.4.1Sponsor's protocol code number401GSDIA01
    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 SponsorUltragenyx Pharmaceutical, Inc.
    B.1.3.4CountryUnited States
    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 supportUltragenyx Pharmaceutical, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUltragenyx Pharmaceutical, Inc.
    B.5.2Functional name of contact pointClinical Development
    B.5.3 Address:
    B.5.3.1Street Address840 Memorial Drive
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02139
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34900834223
    B.5.6E-mailRegistroEspanolDeEstudiosClinicos@druginfo.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/3/16/1771
    D.3 Description of the IMP
    D.3.1Product nameDTX401
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot yet assigned.
    D.3.9.2Current sponsor codeDTX401
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number5000000000000
    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 No
    D.3.11.3.2Gene therapy medical product Yes
    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 numberClassified as gene therapy medicinal product, EMA/506999/2016
    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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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
    Glycogen Storage Disease Type Ia (GSDIa).
    glucogenosis de tipo Ia (GSD-Ia)
    E.1.1.1Medical condition in easily understood language
    Inherited disorder causing decrease of glucose.
    desorden hereditario causante de la disminución de glucosa
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10056911
    E.1.2Term Glycogen storage disease type IA
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the safety of single IV doses of DTX401 in adults with GSDIa, including the incidence of dose-limiting toxicities (DLTs).
    Determinar la seguridad de distintas dosis intravenosas (IV) únicas de DTX401 en adultos con GSDIa, incluida la incidencia de toxicidades limitantes de la dosis (TLD).
    E.2.2Secondary objectives of the trial
    To establish a dose of DTX401 that achieves symptom-free euglycemia (glucose >=60 mg/dL
    [>=3.33 mmol/L]) in a setting of a controlled fasting challenge to allow further clinical development.
    Determinar una dosis de DTX401 que logre la euglucemia asintomática (glucosa >= 60 mg/dl [>=3,33 mmol/l]) en el contexto de una prueba de provocación en ayunas controlada para poder continuar con el desarrollo clínico.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Willing and able to provide written informed consent.
    2. Males and females >=18 years of age.
    3. Documented GSDIa with confirmation by molecular testing.
    4. Documented history of >=1 hypoglycemic event with glucose <60 mg/dL (<3.33 mmol/L).
    5. Subject’s GSDIa disease is stable as evidenced by no hospitalization for severe hypoglycemia during the 4-week period preceding the screening visit.
    6. Hematology and coagulation panel results are within the normal range or, if outside the normal range, are deemed not clinically significant in the opinion of the investigator.
    7. No known allergic reaction to any component of DTX401.
    8. Willing and able to comply with study procedures and requirements, including periodic inpatient hospitalization, frequent blood collections, and 24-hour urine collection.
    9. Males and females of childbearing potential must be willing to use effective contraception at the time of administration of DTX401 and for 52 weeks following administration of DTX401 to prevent the potential transmission of the AAV vector.
    1.Disposición y capacidad para otorgar el consentimiento informado por escrito.
    2.Varones y mujeres de 18 años o más.
    3.GSDIa documentada con confirmación por análisis molecular.
    4.Antecedentes documentados de >= 1 episodio hipoglucémico con un valor de glucosa < 60 mg/dl (< 3,33 mmol/l).
    5.GSDIa estable, demostrado por la ausencia de hospitalizaciones por hipoglucemia grave durante el periodo de 4 semanas previo a la visita de selección.
    6.Resultados de las pruebas de hematología y coagulación dentro de la normalidad o, si están fuera del intervalo normal, sin significación clínica a juicio del investigador.
    7.Ausencia de reacciones alérgicas conocidas a cualquiera de los componentes de DTX401.
    8.Disposición y capacidad para cumplir los procedimientos y requisitos del estudio, incluidas las hospitalizaciones periódicas, las extracciones de sangre frecuentes y la obtención de muestras de orina de 24 horas.
    9. Los varones y las mujeres potencialmente fértiles deben aceptar el uso de métodos anticonceptivos eficaces desde el momento de la administración de DTX401 y durante las 52 semanas siguientes para prevenir la posible transmisión del vector del AAV
    E.4Principal exclusion criteria
    1. Screening or Baseline (Day 0) glucose level <60 mg/dL (<3.33 mmol/L); subjects may be rescreened after glucose is controlled and stable, at the discretion of the investigator.
    2. Liver transplant, including hepatocyte cell therapy/transplant.
    3. Presence of liver adenoma >5 cm in size.
    4. Presence of liver adenoma >3 cm and <=5 cm in size that has a documented annual growth rate of >=0.5 cm per year.
    5. Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the following laboratory abnormalities: alanine aminotransferase (ALT) or aspartate aminotransferase > the upper limit of normal (ULN), total bilirubin >1.5 × ULN, or alkaline phosphatase >2.5 × ULN. Liver function tests may be repeated during the screening period at the investigator’s discretion.
    6. Serum creatinine >2.0 mg/dL.
    7. Triglycerides >=1000 mg/dL at the time of the screening visit.
    8. Presence of active, or history of treatment for, hepatitis B virus or hepatitis C virus infection.
    9. History of human immunodeficiency virus infection AND any of the following: CD4+ cell count <350 cells/mm3, change in antiretroviral therapy regimen within 6 months prior to Day 0, or viral load >200 copies/mL, on 2 separate occasions, as measured by polymerase chain reaction.
    10. History of a malignancy for which the subject has received treatment in the past 2 years except for prostate cancer treated with watchful waiting or surgically removed nonmelanoma skin cancer.
    11. Active infection (viral or bacterial).
    12. Anti-AAV8 neutralizing antibody titer >=1:5.
    13. Participation (current or previous) in another gene transfer study.
    14. Participation in another investigational product study within 3 months of Screening.
    15. Has a positive serum pregnancy test at Screening (females of childbearing potential only), a positive urine pregnancy test at Baseline (Day 0; females of childbearing potential only), or is nursing.
    16. Has any other significant medical condition that the investigator feels would be a risk to the subject or would impede the study.
    1.Glucosa < 60 mg/dl (< 3,33 mmol/l) en la selección o en el momento basal (Día 0); los sujetos podrán repetir la selección una vez la glucosa esté controlada y estable, a criterio del investigador.
    2.Trasplante de hígado, incluido el trasplante o tratamiento de hepatocitos.
    3.Presencia de adenoma hepático con un tamaño > 5 cm.
    4.Presencia de adenoma hepático con un tamaño > 3 cm y <= 5 cm, que tenga una tasa de crecimiento anual documentada >= 0,5 cm al año.
    5.Inflamación hepática o cirrosis importantes demostradas mediante estudios de imagen o por alguna de los anomalías analíticas siguientes: alanina aminotransferasa (ALT) o aspartato aminotransferasa > el límite superior de la normalidad (LSN); bilirrubina total > 1,5 veces el LSN o fosfatasa alcalina > 2,5 veces el LSN. Durante el periodo de selección se podrán repetir las pruebas de la función hepática a criterio del investigador.
    6.Creatinina sérica > 2,0 mg/dl.
    7.Triglicéridos >= 1000 mg/dl en el momento de la visita de selección.
    8.Presencia de infección activa o antecedentes de tratamiento para la infección por el virus de la hepatitis B o C.
    9.Antecedentes de infección por el virus de la inmunodeficiencia humana Y cualquiera de los siguientes: Recuento de linfocitos CD4+ < 350 células/mm3, variación de pauta de tratamiento antirretroviral en los 6 meses anteriores al día 0 o viremia > 200 copias/ml documentada en dos ocasiones distintas mediante reacción en cadena de la polimerasa.
    10.Antecedentes de neoplasia maligna para la que el sujeto haya recibido tratamiento en los 2 últimos años, excepto el cáncer de próstata tratado con espera vigilante o el cáncer de piel no melanomatoso extirpado quirúrgicamente.
    11.Infección activa (vírica o bacteriana).
    12.Título de anticuerpos neutralizantes frente al AAV8 >= 1:5.
    13.Participación (actual o previa) en otro estudio de transferencia génica.
    14.Participación en otro estudio con un producto en investigación en los 3 meses anteriores a la selección.
    15.Resultado positivo en una prueba de embarazo en suero practicada en la selección (solo las mujeres potencialmente fértiles) o en una prueba de embarazo en orina practicada en el momento basal (Día 0; solo las mujeres potencialmente fértiles) o paciente en periodo de lactancia.
    16.Cualquier otra condición médica de importancia que, en opinión del investigador suponga un riesgo para el sujeto o impida la realización del estudio
    E.5 End points
    E.5.1Primary end point(s)
    The incidence of AEs, including the incidence of DLTs at each dose level, TEAEs, and SAEs for each dosing cohort, assessed by severity and relationship to study product.
    Incidencia de acontecimientos adversos (AA), incluida la incidencia de TLD en cada nivel de dosis, acontecimientos adversos de aparición durante el tratamiento (AAAT) y acontecimientos adversos graves (AAG) por cohorte de dosis, evaluados según la intensidad y la relación con el fármaco del estudio.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From the time the subject signs the Informed Consent Form (ICF) and up to 30 days after the end of the study / early withdrawal visit.
    Desde la firma del consentimiento informado hasta 30 dias después de la visita de fin de estudio / retirada prematura.
    E.5.2Secondary end point(s)
    The change from baseline in time (in minutes) to first hypoglycemic event (defined as glucose <60 mg/dL [<3.33 mmol/L]) during a controlled fasting challenge at 6, 12, 24, and 52 weeks after IV administration of DTX401.
    Variación con respecto al momento basal del tiempo (en minutos) hasta el primer episodio hipoglucémico (definido como un valor de glucosa < 60 mg/dl [< 3,33 mmol/l]) durante una prueba de provocación en ayunas controlada a las 6, 12, 24 y 52 semanas después de la administración IV de DTX401.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 0 and weeks 6, 12, 24 and 52.
    Día 0 y semanas 6, 12, 24 y 52
    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 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 Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open 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 No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    Canada
    Netherlands
    Spain
    United States
    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.
    UVUP
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days10
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days21
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 12
    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)
    After patients have ended their participation in the trial, they will be treated according to the normal standard of care. After completion of this study, subjects will be offered enrollment in a 4-year extension study to evaluate the long-term (a total of 5 years) safety and efficacy of DTX401.
    Después de que los pacientes hayan terminado su participación en el ensayo, serán tratados de acuerdo con el estándar de atención normal. Después de completar este estudio, se ofrecerá a los sujetos la inscripción en un estudio de extensión de 4 años para evaluar la seguridad y eficacia a largo plazo (un total de 5 años) de DTX401.
    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 Decision2018-11-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-07-27
    P. End of Trial
    P.End of Trial StatusOngoing
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