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    The EU Clinical Trials Register currently displays   35419   clinical trials with a EudraCT protocol, of which   5814   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2018-000156-18
    Sponsor's Protocol Code Number:301OTC02
    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:2018-05-14
    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 number2018-000156-18
    A.3Full title of the trial
    A Long-Term Follow-up Study to Evaluate the Safety and Efficacy 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 seguimiento a largo plazo para evaluar la seguridad y la eficacia 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
    A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term
    Un estudio clinico para conocer los efectos a largo plazo 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 number301OTC02
    A.5.4Other Identifiers
    Name:IND NumberNumber:17190
    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/3/16/1623
    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 20.0
    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 long-term safety of DTX301 following a single IV dose in adults with late-onset OTC deficiency.
    Determinar la seguridad a largo plazo de DTX301 después de una sola dosis intravenosa (IV) en adultos con deficiencia de OTC de comienzo tardío.
    E.2.2Secondary objectives of the trial
    To evaluate the long-term efficacy of DTX301 on AUC0-24 for plasma ammonia following a single IV dose in adults with late-onset OTC deficiency.

    To evaluate the long-term effects of DTX301 on the rate of ureagenesis in adults with late-onset OTC deficiency.
    Evaluar la eficacia a largo plazo de DTX301 en AUC0-24 para el amoníaco plasmático después de una sola dosis IV en adultos con deficiencia de OTC de comienzo tardío.

    Evaluar los efectos a largo plazo de DTX301 en la tasa de ureagénesis en adultos con deficiencia de OTC de comienzo tardío.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Completed the Week 52 visit in Study 301OTC01. Note that the Day 0 visit of Study 301OTC02 may coincide with the Week 52 visit of Study 301OTC01.
    2. Willing and able to provide written informed consent.
    3. Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.
    1. Haber completado la visita de la semana 52 en el estudio 301OTC01. Hay que advertir de que la visita del día 0 del estudio 301OTC02 podría coincidir con la visita de la semana 52 del estudio 301OTC01.
    2. Disposición y capacidad para otorgar el consentimiento informado por escrito.
    3. Disposición, capacidad y compromiso para cumplir las visitas programadas al centro del estudio y los procedimientos y requisitos del estudio.
    E.4Principal exclusion criteria
    1. Planned or current participation in another interventional clinical study that may confound the efficacy or safety evaluation of DTX301 during the duration of this study.
    2. Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study.
    1. Participación prevista o actual en cualquier otro estudio clínico intervencionista que pueda confundir la evaluación de la eficacia o la seguridad de DTX301 durante este estudio.
    2. Cualquier enfermedad clínicamente importante que, en opinión del investigador, suponga un riesgo para la seguridad del sujeto o impida el estudio.
    E.5 End points
    E.5.1Primary end point(s)
    The incidence of AEs and SAEs for each dosing cohort assessed by severity and relationship to study product.
    Incidencia de AA y AAG por cada 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 la firma del Consentimiento Informado por el sujeto hasta el final del estudio/visita de retirada prematura.
    E.5.2Secondary end point(s)
    The change from baseline (Day 0 of Study 301OTC01) in AUC0-24 for plasma ammonia over time to 260 weeks following IV administration of DTX301.

    The change from baseline (average of Screening and Day 1 results of Study 301OTC01) 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 260 weeks following IV administration of DTX301.
    Variación con respecto al momento basal (día 0 del estudio 301OTC01) del AUC0-24 para el amoníaco plasmático a lo largo del tiempo hasta 260 semanas después de la administración IV de DTX301.

    Variación con respecto al momento basal (promedio de los resultados en la selección y el día 1 del estudio 301OTC01) de la tasa de ureagénesis (medida mediante la generación de [13C]urea durante 4 horas) según lo determinado por la
    espectrometría de masas-cromatografía de gases a lo largo del tiempo hasta 260 semanas después de la administración IV de DTX301.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Plasma ammonia: Day 0, Week 52, Week 104, Week 156, Week 208

    Ureagenesis: Day 0, Week 26, Week 52, Week 78, Week 104, Week 130, Week 156, Week 182, Week 208
    Amoniaco plásmatico: Dia 0, Semana 52, Semana 104, Semana 156, Semana 208

    Ureagénesis: Dia 0, Semana 26, Semana 52, Semana 78, Semana 104, Semana 130, Semana 156, Semana 182, Semana 208.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Long-term follow-up study
    Estudio de seguimiento a largo plazo
    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 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
    Spain
    United Kingdom
    United States
    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
    Ultima visita del ultimo sujeto
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months3
    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: 8
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 9
    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.
    Una vez que los pacientes terminen su participación en el ensayo, recibirán tratamiento de acuerdo con la práctica clínica habitual.
    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 Decision2018-06-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-14
    P. End of Trial
    P.End of Trial StatusOngoing
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