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    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7259   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).

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    Summary
    EudraCT Number:2015-000104-26
    Sponsor's Protocol Code Number:UX003-CL203
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-02-03
    Trial results View 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 number2015-000104-26
    A.3Full title of the trial
    An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 Years Old
    Estudio abierto de terapia enzimática sustitutiva con la rhGUS UX003 en pacientes con MPS VII menores de 5 años
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A safety, tolerability and efficacy study in MPS 7 patients less than 5 years of age receiving enzyme (UX003) replacement by intravenous injection
    Un estudio de seguridad, tolerabilidad y eficacia en pacientes menores de 5 años con MPS VII tratados con una enzima (UX003) sustitutiva por vía intravenosa
    A.4.1Sponsor's protocol code numberUX003-CL203
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/151/2014
    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 Operation
    B.5.3 Address:
    B.5.3.1Street Address60 Leveroni Court
    B.5.3.2Town/ cityNovato, CA
    B.5.3.3Post code94949
    B.5.3.4CountryUnited States
    B.5.4Telephone number14154838148
    B.5.6E-mailrhostutler@ultragenyx.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/3/12/973
    D.3 Description of the IMP
    D.3.1Product nameRecombinant human beta-glucuronidase
    D.3.2Product code UX003
    D.3.4Pharmaceutical form 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 available
    D.3.9.1CAS number 1638194-78-1
    D.3.9.2Current sponsor codeUX003
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS
    D.3.9.4EV Substance CodeSUB128266
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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) Yes
    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 Yes
    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 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
    Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome)
    Mucopolisacaridosis de tipo VII (MPS VII, síndrome de Sly)
    E.1.1.1Medical condition in easily understood language
    Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome)
    Mucopolisacaridosis de tipo VII (MPS VII, síndrome de Sly)
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate the effect of UX003 treatment in pediatric MPS 7 subjects less than 5 years of age on:
    -Safety and tolerability
    -Efficacy as determined by the reduction of uGAG excretion
    El objetivo principal consiste en evaluar el efecto del tratamiento con UX003 en pacientes pediátricos con MPS VII menores de 5 años de edad en:
    -Seguridad y tolerabilidad
    -La eficacia según lo determinado por la reducción de la excreción de GAG (GAGu)
    E.2.2Secondary objectives of the trial
    The secondary objective is to evaluate the effect of UX003 on growth velocity and hepatosplenomegaly.
    El objetivo secundario es evaluar el efecto de UX003 en la velocidad de crecimiento y en la hepatoesplenomegalia.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay, or genetic testing
    -Under 5 years of age at the time of informed consent
    -Written informed consent of Legally Authorized Representative after the nature of the study has been explained, and prior to any research-related procedures
    -Diagnóstico confirmado de MPS VII sobre la base de la determinación enzimática de glucuronidasa en leucocitos o fibroblastos o del análisis genético.
    -Menores de 5 años de edad en el momento del consentimiento informado.
    -Consentimiento informado por escrito del representante legalmente autorizado después de haberse explicado la naturaleza del estudio y antes de cualquier procedimiento relacionado con el estudio.
    E.4Principal exclusion criteria
    -Undergone a successful bone marrow or stem cell transplant or has evidence of any degree of detectable chimaerism with donor cells
    -Any known hypersensitivity to rhGUS or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
    -Use of any investigational product (drug or device or combination) other than UX003 within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments at any time during the study
    -Has a condition of such severity and acuity, in the opinion of the Investigator, which may not allow safe study participation. For patients with hydrops fetalis, the ongoing interventions to manage fluid balance can be continued; if the addition of ERT is considered a fluid-overload risk, the individual should be excluded.
    -Has a concurrent disease or condition that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or affect safety. Since hydropic patients have a high rate of mortality, the risk of death prior to 1 year of age should not be considered sufficient to exclude the patient from the study for compliance.
    -Se ha sometido a un trasplante exitoso de médula ósea o células madre, o tiene signos de algún grado de quimerismo detectable con células del donante.
    -Cualquier hipersensibilidad conocida a la rhGUS o sus excipientes que, en opinión del investigador, aumenta el riesgo del paciente de sufrir efectos adversos.
    -Uso de un producto en investigación (medicamento o dispositivo, o combinación) distinto de UX003 dentro de los 30 días anteriores a la selección o necesidad de un fármaco en investigación antes de finalizar todas las evaluaciones programadas del estudio en cualquier momento durante su transcurso.
    -Tiene una afección cuya gravedad y carácter agudo podría no permitir, en opinión del investigador, la participación segura en el estudio. En los pacientes con hidropesía fetal pueden continuar las intervenciones en curso para controlar el balance hídrico. El paciente se excluirá del estudio si se considera que añadir el TSE representa un riesgo de sobrecarga de líquidos.
    -Tiene una enfermedad o un trastorno concurrente que, en opinión del investigador, pone al paciente en un alto riesgo de no cumplir adecuadamente con el tratamiento, de no completar el estudio o que podría interferir en la participación en el estudio o comprometer la seguridad. Como la tasa de mortalidad es alta en los pacientes con hidropesía, el riesgo de muerte antes de 1 año de edad no debe considerarse motivo suficiente para excluir al paciente del estudio por problemas de cumplimiento
    E.5 End points
    E.5.1Primary end point(s)
    Urinary GAG Excretion: the mean percent change in uGAG excretion from Week 0 to Week 48.
    Excreción de GAG urinarios: cambio en el porcentaje de excreción media de GAGu entre la semana 0 y la semana 48.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Urinary GAG Excretion: Urine samples will be collected every other visit up to Week 12, then
    every 6 weeks to Week 48, then every 12 weeks to Week 240. And 2 samples will be collected during the baseline week.
    Excreción de GAG urinarios: Las muestras de orina se recogerán cada 2 visitas hasta visita de semana 12, después cada 6 semanas hasta visita semana 48, después cada 12 semanas hasta visita de semana 240. Y se recogerán 2 muestras en la visita basal.
    E.5.2Secondary end point(s)
    Growth and hepatosplenomegaly will be evaluated to compare pretreatment with post treatment effects
    El crecimiento y la hepatoesplenomegalia se evaluarán para comparar los efectos pre-tratamiento
    con los efectos post-tratamiento.
    E.5.2.1Timepoint(s) of evaluation of this end point
    -For growth: anthropometrics will be measured every 12 weeks from baseline to Week 240,
    -For liver/spleen size: will be measured at baseline, Week 12, 24, 48, then every 48 weeks to Week 240
    -Para crecimiento: las medidas antropométricas se realizarán cada 12 semanas desde visita basal hasta visita de semana 240.
    -Para el tamaño de hígado/bazo: Se tomarán medidas en visita basal, semana 12, 24, 48 y después cada 48 semanas hasta la visita de semana 240.
    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 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) No
    E.7.1.1First administration to humans No
    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.8.2.4Number 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 EEA2
    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
    Portugal
    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
    Última visita del último paciente
    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 months0
    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 months6
    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 Yes
    F.1.1Number of subjects for this age range: 15
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 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
    Young pediatric patients (less than 5 years old).
    Pacientes pediátricos (menores de 5 años).
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 15
    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)
    Subjects successfully completing the first 48 weeks may continue treatment for up to 240 weeks (5 years) or until one of the following occurs: the patient withdraws consent, the patient is discontinued from the study at the discretion of the Investigator or Ultragenyx, the study is terminated, or UX003 becomes commercially available. However, patients are not obligated to participate in the long-term extension phase of this study.
    Los pacientes que completen con éxito las primeras 48 semanas pueden continuar el tratamiento hasta 240 semanas (5 años) o hasta que una se las siguientes situaciones ocurra: el paciente es discontinuado del estudio a juicio del Investigador o de Ultragenyx, el estudio sea terminado, o UX003 está comercialmente disponible. No obstante, no es obligatorio que los pacientes participen en la fase de extensión de este estudio.
    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 Decision2016-04-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-02-23
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2019-03-26
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