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    Summary
    EudraCT Number:2016-001057-40
    Sponsor's Protocol Code Number:301OTC01
    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:2016-12-27
    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 number2016-001057-40
    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 Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency.
    Estudio de fase 1/2, abierto, de seguridad y determinación de dosis de la transferencia del gen de la ornitina transcarbamilasa (OTC) humana mediada por el virus adenoasociado (AAV) de serotipo 8 (AAV8) en adultos con deficiencia de OTC de comienzo tardío.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An early-phase clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency.
    Estudio clínico de fase temprana de un virus que transfiere el gen de la ornitina transcarbamilasa (OTC) humana en adultos con deficiencia de OTC de comienzo tardío.
    A.4.1Sponsor's protocol code number301OTC01
    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 SponsorDimension Therapeutics, 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 supportDimension Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDimension Therapeutics, 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/3/16/1623
    D.3 Description of the IMP
    D.3.1Product nameDTX301
    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.3.9.3Other descriptive nameDTX301
    D.3.9.4EV Substance CodeSUB184280
    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/CAT/803478/2015
    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
    Ornithine transcarbamylase deficiency
    Deficiencia de ornitina transcarbamilasa
    E.1.1.1Medical condition in easily understood language
    Inherited disorder causing accumulation of ammonia
    Transtorno hereditario que da lugar a la acumulación de amoniaco
    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 19.0
    E.1.2Level LLT
    E.1.2Classification code 10071107
    E.1.2Term Ornithine transcarbamylase deficiency
    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 intravenous (IV) doses of DTX301 in adults with late-onset OTC deficiency.
    Determinar la seguridad de distintas dosis intravenosas (IV) únicas de DTX301 en adultos con deficiencia de OTC de comienzo tardío.
    E.2.2Secondary objectives of the trial
    To establish a dose of DTX301 that has a clinically meaningful increase in the rate of ureagenesis to allow further clinical development.
    To evaluate the efficacy of single IV doses of DTX301 in adults with late-onset OTC deficiency, in the setting of tapering or discontinuing ammonia scavenger medications.
    Establecer una dosis de DTX301 que produzca un aumento clínicamente significativo de la tasa de ureagénesis y que permita continuar el desarrollo clínico.
    Evaluar la eficacia de una dosis IV única de DTX301 en adultos con deficiencia de OTC de comienzo tardío en el contexto de la reducción gradual o suspensión de los medicamentos quelantes de amonio.
    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 with documented diagnosis of late-onset (defined as first manifestation of signs and symptoms at ≥1 month of age) OTC deficiency, confirmed via enzymatic, biochemical, or molecular testing. This may include identification of a pathogenic mutation, pedigree analysis, liver OTC activity that is <20% of normal activity, or elevated urinary orotate (>20 μmol/mmol creatinine) after an allopurinol challenge test.
    3. Documented history of ≥1 symptomatic hyperammonemia event with ammonia ≥100 μmol/L.
    4. Subject’s OTC deficiency is stable as evidenced by either a) no clinical symptoms of hyperammonemia OR b) an ammonia level <100 μmol/L within the 4 week period preceding the Screening visit.
    5. Subject’s ammonia level on Day 1 (predose) is <100 μmol/L and is within the range of historical ammonia levels obtained when the subject was clinically stable. If the Day 1 (predose) ammonia is >100 μmol/L and the subject is clinically stable, the ammonia level may be repeated and DTX301 administered if the investigator determines that the repeat
    ammonia is within the subject’s normal range. NOTE: If the subject is deemed clinically unstable, dosing will be held and the subject can be rescreened once the subject is determined to be clinically stable.
    6. On stable dose of ammonia scavenger therapy for ≥4 weeks.
    7. Willing to taper or discontinue ammonia scavengers during the study if deemed safe by the investigator.
    8. No known allergic reaction to any component of DTX301.
    9. Willing and able to comply with study procedures and requirements, including periodic inpatient hospitalizations, frequent blood draws, and urine collections over a 24-hour period.
    10. Hematology and coagulation panel results are within the normal range or, if outside the normal range, deemed not clinically significant in the opinion of the investigator.
    11. Males and all females of childbearing potential must be willing to use effective contraception at the time of administration of gene transfer and for the 52 weeks following administration of DTX301 to prevent the potential transmission of the AAV vector. For male subjects, appropriate contraceptive methods include the use of a condom with spermicide. For female subjects, appropriate contraceptive methods include the use of a condom with spermicide plus at least 1 of the following:
    a. Oral contraceptives, other hormonal contraceptives (vaginal products, skin patches, or implanted or injectable products), or mechanical products such as an intrauterine device;
    b. Use of a diaphragm or cervical/vault cap;
    c. Previous female sterilization (surgical bilateral oophorectomy [with or without hysterectomy] or tubal ligation) at least 6 weeks prior to DTX301 administration. In case of an oophorectomy alone, the reproductive status of the subject must have been confirmed by follow-up hormone level assessment.
    NOTE: Abstinence is an acceptable form of birth control; however, appropriate contraception must be used if the subject becomes sexually active. A condom with spermicide is required to be used by all sexually active vasectomized males in the study in order to prevent potential transmission of the vector via seminal fluid.
    NOTE: Females of childbearing potential are defined as all females physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception for the duration of the study. Females are considered post-menopausal and not of childbearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (eg, age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least 6 weeks prior to enrollment. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of childbearing potential.
    1. Disposición y capacidad para otorgar el consentimiento informado por escrito.
    2. Varones y mujeres mayores de 18 años de edad con diagnóstico documentado de deficiencia de OTC de comienzo tardío (definido como primera manifestación de signos y síntomas a una edad >=1 mes), confirmado mediante análisis enzimático, bioquímico o molecular. Esto puede incluir la identificación de una mutación patógena, análisis genealógico, actividad de la OTC hepática <20 % de la actividad normal, o concentración urinaria de orotato elevada (>20 µmol/mmol de creatinina) después de una prueba de exposición a alopurinol.
    3. Historia clínica documentada de más de 1 episodio sintomático de hiperamonemia con amonio >= 100 µmol/l.
    4. Demostración de que la deficiencia de OTC del sujeto se halla estabilizada basada en: a) ausencia de síntomas clínicos de la hiperamonemia, O b) concentración de amonio <100 µmol/l dentro del periodo de 4 semanas que precede a la visita de selección.
    5. La concentración de amonio del sujeto determinada el día 1 (antes de la dosis) es <100 µmol/l y se sitúa dentro del intervalo de los niveles históricos de amonio medidos estando el sujeto clínicamente estabilizado. Si la concentración de amonio determinada el día 1 (antes de la dosis) es >100 µmol/l y el sujeto se encuentra clínicamente estabilizado, se podrá repetir el análisis del nivel de amonio y administrar DTX301 si el investigador determina que el valor repetido de amonio está dentro del intervalo de normalidad del sujeto. NOTA: Si al sujeto se le considera clínicamente inestable, se aplazará la administración y se podrá repetir el análisis cuando se determine que está clínicamente estabilizado.
    6. El sujeto ha estado recibiendo una dosis estable de fármacos quelantes de amonio durante >=4 semanas.
    7. Disposición para reducir gradualmente o suspender los quelantes de amonio durante el estudio si el investigador lo considera seguro.
    8. Ausencia de reacciones alérgicas conocidas a algún componente del DTX301.
    9. Disposición y capacidad para cumplir los procedimientos y requisitos del estudio, incluso las hospitalizaciones periódicas, extracciones frecuentes de sangre y tomas de muestras de orina de 24 horas.
    10. Resultados de las pruebas de hematología/coagulación dentro de la normalidad o, si están fuera del intervalo normal, carentes de significación clínica a juicio del investigador.
    11. Los varones y las mujeres potencialmente fértiles deben aceptar el uso de métodos anticonceptivos eficaces en el momento de la administración de la transferencia génica y durante las 52 semanas siguientes a la administración de DTX301 para prevenir la posible transmisión del vector del AAV. Para los varones se considera un método anticonceptivo adecuado el uso de preservativo con espermicida. Para las mujeres, se consideran métodos anticonceptivos adecuados el uso de preservativo con espermicida Y al menos uno de los siguientes:
    a. Anticonceptivos orales, otros anticonceptivos hormonales (productos vaginales, parches cutáneos, implantes o productos inyectables) o métodos de barrera como el dispositivo intrauterino;
    b. Uso de diafragma o capuchón cervical;
    c. Esterilización previa en las mujeres (ovariectomía quirúrgica bilateral, con o sin histerectomía, o ligadura de trompas) al menos 6 semanas antes de la administración de DTX301. En caso de ovariectomía sola, únicamente cuando se haya confirmado el estado reproductivo de la mujer mediante la determinación de las concentraciones hormonales.
    NOTA: La abstinencia se considera una forma aceptable de anticoncepción; sin embargo, si el sujeto tiene relaciones sexuales deberá utilizar un método anticonceptivo adecuado. Todos los varones vasectomizados que tengan relaciones sexuales durante el estudio deberán usar preservativo con espermicida para prevenir la posible transmisión del vector a través del líquido seminal.
    NOTA: Se consideran mujeres potencialmente fértiles aquellas que tienen la capacidad fisiológica de quedarse embarazadas, salvo que utilicen métodos anticonceptivos altamente eficaces durante todo el periodo de estudio. Se considera que las mujeres están en la menopausia y no en edad fértil si han tenido 12 meses de amenorrea natural (espontánea) con un perfil clínico apropiado (por ejemplo, edad adecuada, antecedentes de síntomas vasomotores) o se han sometido a ovariectomía quirúrgica bilateral (con o sin histerectomía) o a ligadura de trompas al menos seis semanas antes de la inclusión. Si sólo se ha practicado ovariectomía, se considerará que son fértiles hasta que se confirme su capacidad reproductiva mediante un seguimiento de la concentración hormonal.
    E.4Principal exclusion criteria
    1. Screening or Baseline (Day 0) ammonia level ≥100 μmol/L or signs and symptoms indicative of hyperammonemia during the 4-week period preceding Day 0; subjects may be rescreened once after their ammonia is controlled and stable for at least 28 days, at the discretion of the investigator.
    2. Liver transplant, including hepatocyte cell therapy/transplant.
    3. History of liver disease as evidenced by any of the following: portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy, or a liver biopsy with evidence of stage 3 fibrosis.
    4. Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the following laboratory abnormalities: alanine aminotransferase or aspartate aminotransferase >2.0 × upper limit of normal (ULN), total bilirubin >1.5 × ULN, alkaline phosphatase >2.5 × ULN.
    5. Serum creatinine >2.0 mg/dL.
    6. Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection, documented by current use of antiviral therapy for HBV or HCV or by hepatitis B surface antigen (HBsAg) or HCV RNA positivity.
    NOTE: Subjects with a history of HCV infection must have documentation of 2 negative viral assays by polymerase chain reaction (PCR), collected at least 6 months apart, to be considered negative for HCV. Subjects with a history of HCV infection who test positive for HCV RNA at Screening can be rescreened once, after they have been treated and have documentation of at least 2 negative samples collected at least 6 months apart.
    7. History of human immunodeficiency virus (HIV) 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 plasma viral load >200 copies/mL, documented on 2 separate occasions, as measured by PCR.
    8. Active infection (viral or bacterial).
    9. Anti-AAV8 neutralizing antibody titer >1:5.
    10. Participation (current or previous) in another gene transfer study.
    11. Participation in another investigational medicine study within 3 months of Screening.
    12. 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 non-melanoma skin cancer.
    13. 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.
    14. Has any other significant medical condition that the investigator feels would be a risk to the subject or would impede the study.
    1. Concentración de amonio en la selección o en el momento basal (día 0) >=100 µmol/l, o presentar signos y síntomas de hiperamonemia durante el periodo de 4 semanas que precede al día 0; se podrá repetir una vez el análisis si el nivel de amonio se mantiene controlado y estable durante al menos 28 días, a criterio del investigador.
    2. Trasplante de hígado, incluido el trasplante o tratamiento de hepatocitos.
    3. Antecedentes de hepatopatía, revelada por alguno de los siguientes: hipertensión portal, ascitis, esplenomegalia, varices esofáticas, encefalopatía hepática o una biopsia de hígado con indicios de fibrosis en estadio 3.
    4. Inflamación hepática importante o cirrosis demostradas mediante estudios de imagen o por alguna de los anomalías analíticas siguientes: alanina aminotransferasa o aspartato aminotransfertasa >2,0 veces el límite superior de la normalidad (LSN); bilirrubina total >1,5 veces el LSN, o fosfatasa alcalina >2,5 veces el LSN.
    5. Creatinina sérica >2,0 mg/dl.
    6. Infección activa por el virus de la hepatitis B (VHB) o por el virus de la hepatitis C (VHC), documentada por el uso actual de tratamiento antiviral contra el VHB/VHC o por la positividad del antígeno de superficie del virus de la hepatitis B (HBsAg) o del ARN del VHC. NOTA: Los sujetos con antecedentes de infección por el VHC deberán tener documentados 2 resultados virales negativos en la prueba de la reacción en cadena de la polimerasa (RCP), obtenidos al menos con 6 meses de separación, para que se les considere negativos en términos de VHC. Los sujetos con antecedentes de infección por el VHC que den resultado positivo de ARN del VHC en la selección podrán repetir una vez la selección después de recibir tratamiento y tener documentados como mínimo 2 resultados negativos obtenidos al menos con 6 meses de separación.
    7. Antecedentes de infección por el virus de la inmunodeficiencia humana (VIH) Y cualquiera de los siguientes: Recuento de linfocitos CD4+ <350 células/mm3, cambio de régimen de tratamiento antiviral en los 6 meses que preceden al día 0, o viremia >200 copias/ml documentada en 2 ocasiones separadas mediante RCP.
    8. Infección activa (viral o bacteriana).
    9. Título de anticuerpos neutralizantes anti-AAV8 >1:5.
    10. Participación (actual o previa) en otro estudio de transferencia génica.
    11. Participación en otro estudio de investigación de medicamentos en los 3 meses que preceden a la selección.
    12. Antecedentes de neoplasia maligna para la que el sujeto ha recibido tratamiento en los 2 últimos años, excepto el cáncer de próstata tratado con conducta expectante o el cáncer de piel no melanoma extirpado quirúrgicamente.
    13. Tener 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 estar en periodo de lactancia.
    14. Cualquier otra enfermedad de importancia que el investigador considere que supone un riesgo para el sujeto o impide la realización del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    The incidence of adverse events (AEs), treatment-emergent adverse events (TEAEs), and serious adverse events (SAEs) for each dosing cohort, assessed by severity and relationship to study product.
    Incidencia de acontecimientos adversos (AA), 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 through the end of study/early withdrawal visit.
    Desde el momento en que el sujeto firma el documento de Consentimiento Informado hasta el fin del estudio/visita de retirada prematura.
    E.5.2Secondary end point(s)
    - The change from baseline (baseline is defined as the average of Screening and Day 1 results) in the rate of ureagenesis (as measured by the generation of [13C]urea over 4 hours) as determined by gas chromatography mass spectrometry over time to 52 weeks after the IV administration of DTX301.
    - The change from baseline (Day 0) in area under the curve from time zero to 24 hours (AUC0-24) for serum ammonia over time to 52 weeks after IV administration of DTX301 in the setting of tapering or discontinuing ammonia scavenger medications.
    - Variación respecto al valor basal (que se define como el promedio de los resultados obtenidos en la selección y el día 1) de la tasa de ureagénesis (que se mide por la formación de [13C]urea durante 4 horas), determinada mediante cromatografía de gases y espectrometría de masas a lo largo del tiempo, hasta 52 semanas después de la administración IV de DTX301.
    - Variación respecto al valor basal (día 0) del área bajo la curva de 0 a 24 horas (AUC0 24) del amonio sérico a lo largo del tiempo, hasta 52 semanas después de la administración IV de DTX301 en el contexto de la reducción gradual o suspensión de los medicamentos quelantes de amonio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Ureagenesis: at Screening, Day 1 prior to DTX301 infusion, Week 6, Week 12, Week 20, Week 24, Week 52

    Serum ammonia: Day 0, Week 6, Week 12, Week 24, Week 52
    Ureagénesis: Selección, Dia 1 antes de la infusion de DTX301, Semana 6, Semana 12, Semana 20, Semana 24, Semana 52

    Amonio sérico: Dia 0, Semana 6, Semana 12, Semana 24, Semana 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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    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
    France
    Spain
    United Kingdom
    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
    Ultima visita del ultimo sujeto
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    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 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: 11
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    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 asked to enroll in a 4-year extension study to evaluate the long-term (a total of 5 years) safety and efficacy of DTX301.
    Una vez que los pacientes terminen su participación en el estudio, recibirán tratamiento de acuerdo con la práctica clínica habitual. Una vez finalizado este estudio, se pedirá a los sujetos que participen en un estudio de extensión de 4 años para evaluar la seguridad y eficacia a largo plazo (5 años en total) de DTX301.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-03-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-02-28
    P. End of Trial
    P.End of Trial StatusOngoing
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