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    Summary
    EudraCT Number:2021-000903-19
    Sponsor's Protocol Code Number:UX053-CL101
    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:2021-07-20
    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 number2021-000903-19
    A.3Full title of the trial
    A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label) and Repeat Doses (Part 2: Randomized, Double-blind, Placebo-controlled) of UX053 in Patients with GSD III
    Estudio de fase I/II, primero en el ser humano, de dos partes, para evaluar la seguridad, la tolerabilidad y la farmacocinética de dosis únicas ascendentes (Parte 1: sin enmascaramiento) y dosis repetidas (Parte 2: aleatorizada, con doble enmascaramiento y controlada con placebo) de UX053 en pacientes con glucogenosis tipo III
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A first-in-human study of UX053 in Patients with Glycogen Storage Disease type III (GSD III)
    Estudio, primero en el ser humano, de UX053 en pacientes con glucogenosis tipo III
    A.4.1Sponsor's protocol code numberUX053-CL101
    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 pointUX053-CL101 Clinical Operations
    B.5.3 Address:
    B.5.3.1Street Address60 Leveroni Court
    B.5.3.2Town/ cityNovato
    B.5.3.3Post codeCA 94949
    B.5.3.4CountryUnited States
    B.5.6E-mailUX053-CL101@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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namemRNA encoding the human glycogen debranching enzyme formulated in a lipid nanoparticle delivery
    D.3.2Product code UX053
    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 yet available
    D.3.9.2Current sponsor codeUX053 mRNA
    D.3.9.3Other descriptive namemRNA encoding the human glycogen debranching enzyme
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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 numberEMA/CAT/615211/2020 Gene therapy medicinal product
    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 No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    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 III (GSD III)
    glucogenosis tipo III
    E.1.1.1Medical condition in easily understood language
    A disease where the body cannot break down glycogen (stored form of glucose). It affects the liver, heart muscle, and skeletal muscle. It is also known as Cori disease or Forbes disease.
    Una enfermedad que hace que el cuerpo no pueda descomponer el glucógeno (forma de glucosa almacenada). Afecta al hígado, miocardio y músculo esquelético. También se conoce como glucogenosis tipo III.
    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 10053250
    E.1.2Term Glycogen storage disease type III
    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
    Evaluate the safety of UX053 in adults with GSD III
    Evaluar la seguridad de UX053 en adultos con GSD III
    E.2.2Secondary objectives of the trial
    Characterize the PK of UX053 in adults with GSD III
    Caracterizar la FC de UX053 en adultos con GSD III
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Confirmed diagnosis of Glycogen Storage Disease type III (GSD III) (all subtypes) based on pathogenic mutations in the amylo-α-1,6-glucosidase 4-alpha-glucanotransferase (AGL) gene on both alleles or glycogen debranching enzyme (GDE) deficiency based on biopsy of liver, muscle, or fibroblasts
    2. History of any of the following:
    a. Severe hypoglycemia, defined as neuroglycopenia (eg, altered mental status, seizure, dizziness, slurred speech, blurry vision, abnormal behavior, perioral paresthesia, requiring intervention by a caregiver) or blood glucose < 54 mg/dL (3 mmol/L) within the last year
    b. ≥ 2 incidents of symptomatic hypoglycemia (defined as blood glucose < 70 mg/dL [3.9 mmol/L] if measured at the time of symptoms) within the last year, despite nutrition management
    c. Ongoing liver injury, defined as alanine aminotransferase (ALT) > 2.5x the upper limit of normal (ULN) within the last year
    3. ALT ≤ 5x ULN during the 3 months prior to the Baseline Visit
    4. Males or females ≥ 18 years of age
    5. After nutrition optimization (if necessary) for subjects in the repeat dose (RD) Period, during the beginning of the Screening Period, willing and able to maintain nutritional intake consistent with the nutrition guidelines based on expert recommendations for the remainder of the study
    6. Willing and able to provide access to medical records surrounding medical treatment that occurred prior to enrollment
    7. Willing and able to provide written informed consent, or in the case of adult subjects with cognitive limitation, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained and prior to any test procedures or assessments
    8. Females of childbearing potential must have a negative pregnancy test at Screening (ISV) and be willing to have additional pregnancy tests during the study. Subjects of childbearing potential or fertile males who are sexually active with partners of child-bearing potential must consent to use a highly effective contraceptive method, as described in Appendix 4 of the Protocol, from the Period following the signing of the informed consent through 30 days after last dose of study drug
    1.Diagnóstico confirmado de GSD III (todos los subtipos), basado en mutaciones patogénicas en el gen amilo-α-1,6-glucosidasa 4-alfa-glucanotransferasa (AGL) en ambos alelos o deficiencia de la enzima desramificadora del glucógeno (EDG) en biopsias hepáticas o musculares o en fibroblastos
    2.Antecedentes de cualquiera de los trastornos siguientes:
    a.Hipoglucemia grave, definida como neuroglucopenia (p. ej., alteraciones del estado mental, convulsiones, mareo, habla farfullante, visión borrosa, alteraciones de la conducta o parestesias peribucales que requieran la intervención de un cuidador) o glucemia < 54 mg/dl (3 mmol/l) dentro del último año
    b.>=2 incidentes de hipoglucemia sintomática (definida como glucemia <70 mg/dl [3,9 mmol/l] medida en el momento en que se producen los síntomas) dentro del último año, a pesar de la gestión de la nutrición
    c.Lesión hepática en curso, definido como alanina aminotransferasa (ALT) >2,5 veces el límite superior de la normalidad (LSN) dentro del último año
    3.ALT <=5 veces el LSN durante los 3 meses anteriores a la visita basal
    4.Hombres y mujeres >=18 años de edad
    5.Después de la optimización de la nutrición (si fuese necesaria) en los pacientes del periodo con DR, durante el comienzo del periodo de selección, estar de acuerdo y poder mantener una ingesta nutricional conforme a las pautas nutricionales basadas en las recomendaciones de los expertos durante el resto del estudio
    6.Estar de acuerdo y poder conceder acceso a la parte de la historia clínica relacionada con el tratamiento médico recibido antes de la inclusión
    7.Estar de acuerdo y poder otorgar el consentimiento informado por escrito o, en el caso de pacientes adultos con restricciones cognitivas, proporcionar el asentimiento por escrito (si se requiere) y el consentimiento informado por escrito de un representante legal, después de haber explicado la naturaleza del estudio y antes de realizar cualquier evaluación o procedimiento
    8.Las mujeres que puedan quedar embarazadas deben haber obtenido un resultado negativo en la prueba de embarazo de la selección (VSI) y estar dispuestas a someterse a más pruebas de embarazo durante el estudio. Las pacientes que puedan quedar embarazadas o los hombres fértiles que mantienen relaciones sexuales con parejas que puedan quedar embarazadas deben acceder a utilizar un método anticonceptivo de gran eficacia, según se describe en el apéndice 4 del protocolo, desde el periodo que sigue a la firma del consentimiento informado y hasta 30 días después de la última administración del fármaco del estudio
    E.4Principal exclusion criteria
    1. History of liver transplant, including hepatocyte cell therapy/transplant, or active listing for liver transplant
    2. History of cirrhosis, or presence of any of the following:
    a. Total bilirubin ≥ 1.3 mg/dL and international normalized ratio (INR) ≥ 1.3
    b. Evidence of portal hypertension, including, but not limited to the following symptoms splenomegaly, ascites, thrombocytopenia, esophageal varices, or history of hepatic encephalopathy
    c. Model for End Stage Liver Disease (MELD) score > 12
    3. Current Hepatitis B or C infection or history of chronic Hepatitis B or C infection
    4. Severe renal impairment defined as a glomerular filtration rate (GFR) ≤ 29 mL/min
    5. Any prior history of hepatocellular carcinoma or presence of liver adenoma > 5 cm at the longest diameter or > 3 cm and ≤ 5 cm in size that has an annual growth rate of ≥ 0.5 cm per year
    6. Current or history of malignancies in the 3 years prior to the Screening Visit (ISV for RD)
    7. Hospitalizations related to GSD III disease between the Screening (ISV for RD) and Baseline Visit
    8. Known history of human immunodeficiency virus infection
    9. Presence or history of any hypersensitivity reactions requiring medical evaluation and management (including injection/infusion associated reactions, such as lymphadenopathy) to UX053, its excipients, or any drug products that contain polysorbate or PEG. This may include mRNA-based vaccines that contain PEG or polysorbate
    10. Significant cardiac disease, including heart failure with New York Heart Association (NYHA) Function Capacity III or IV or Objective Assessment C or D, unstable angina, or ejection fraction (EF) < 35%, or uncontrolled arrhythmia or resistant hypertension. Asymptomatic cardiomyopathy and left ventricular hypertrophy (LVH) are allowed
    11. Presence or history of any co-morbid condition or abnormal labs that, in the view of the Investigator, places the subject’s safety at risk; places the subject at high risk of poor treatment compliance or not completing the study; or would significantly affect the interpretation of study results
    12. Poorly controlled diabetes, defined as the presence of any of the following:
    a. Hemoglobin A1C > 8%
    b. History of diabetic nephropathy, neuropathy, or retinopathy
    c. History of diabetic ketoacidosis during the past year
    13. Poorly controlled hypothyroidism, based on the judgement of the Investigator or Ultragenyx, whichever is most conservative
    14. History of chronic coagulopathy, thrombophilia, or disorder of complement activation
    15. Use of concomitant medications that alter PT/INR, including warfarin and direct oral anticoagulants (eg, rivaroxaban, apixaban, and edoxaban). Patients who receive medications that affect platelet function, such as aspirin or clopidogrel, are allowed, unless they have comorbidities that in the judgment of the Investigator place them at undue risk to participate in the study.
    16. Current treatment with long-term immunosuppressive medications. This includes subjects with autoimmune conditions managed with immunosuppressive medications and solid organ transplant recipients.
    17. Active tuberculosis requiring treatment in the past 3 years
    18. Symptomatic COVID-19 infection
    19. History of active alcohol and/or drug abuse that in the Investigator’s assessment would impair the subject’s ability to comply with the protocol
    20. Receipt of only 1 of 2 planned doses of a COVID-19 vaccine. Subjects who have not received a COVID-19 vaccine, and those who have completed COVID-19 vaccination are eligible.
    21. Planned surgery, including dental surgeries, during the SAD Period for subjects in Part 1 or prior to Week 10 of the RD Period for subjects in Part 2
    22. Pregnant or breastfeeding at the Screening Visit (ISV for RD) or planning to become pregnant (self or partner) at any time during the study
    23. Females of childbearing potential with hepatocellular adenoma who are unwilling to use nonhormonal contraception
    24. Use of any IP or investigational medical device within 30 days or for IP within 5 half lives, whichever is longer, prior to the Screening Visit (ISV for RD), or requirement for any investigational agent prior to completion of all scheduled study assessments
    1.Antecedentes de trasplante hepático, incluido el trasplante o tratamiento celular con hepatocitos, o encontrarse en lista de espera para trasplante hepático
    2.Antecedentes de cirrosis o presencia de cualquiera de las circunstancias siguientes:
    a.Bilirrubina total >=1,3 mg/dl e índice INR >=1,3
    b.Signos de hipertensión portal, incluidos, entre otros, los siguientes síntomas: esplenomegalia, ascitis, trombocitopenia, varices esofágicas o antecedentes de encefalopatía hepática
    c.Puntuación >12 en Modelo de hepatopatía en estadio terminal (MELD)
    3.Infección actual por virus de hepatitis B o C o antecedentes de hepatitis B o C crónica
    4.Insuficiencia renal grave, definida como tasa filtración glomerular (FG) <= 29 ml/min
    5.Cualquier antecedente de carcinoma hepatocelular o presencia de adenoma hepático con un diámetro mayor >5 cm o un tamaño >3 cm y <=5 cm con un ritmo de crecimiento anual >= 0,5 cm
    6.Neoplasias malignas actuales o antecedentes de neoplasias malignas en 3 años anteriores a visita selección (VSI del periodo con DR)
    7.Hospitalizaciones relacionadas con la GSD III entre selección (VSI del periodo con DR) y visita basal
    8.Antecedentes conocidos de infección por el virus inmunodeficiencia humana (VIH)
    9.Presencia o antecedentes de cualquier reacción de hipersensibilidad que requiera evaluación y tratamiento médicos (incluidas reacciones asociadas a la inyección/infusión, como aparición de linfadenopatías) frente a UX053, sus excipientes o cualquier producto farmacéutico que contenga polisorbato o PEG, lo que incluye las vacunas basadas en ARNm que contienen PEG o polisorbato
    10.Cardiopatía significativa, incluida insuficiencia cardiaca con capacidad funcional de III o IV según Asociación Cardiológica de Nueva York (New York Heart Association, NYHA) o una evaluación objetiva C o D, angina inestable o fracción de eyección (FE) < 35 %, o arritmia no controlada o hipertensión resistente. Se permiten miocardiopatía asintomática e hipertrofia ventricular izquierda (HVI)
    11.Presencia o antecedentes de cualquier enfermedad concomitante o resultados analíticos anormales que, a juicio del investigador, pondrían en peligro la seguridad del paciente, presentan riesgo alto de que paciente no cumpla tratamiento debidamente o no finalice estudio o influirían de forma considerable en la interpretación de resultados del estudio
    12.Diabetes insuficientemente controlada, definida como presencia de cualquiera de las circunstancias siguientes:
    a.Hemoglobina A1C > 8 %
    b.Antecedentes retinopatía, neuropatía o nefropatía diabéticas
    c.Antecedentes de cetoacidosis diabética en último año
    13.Hipotiroidismo insuficientemente controlado, según juicio del investigador o Ultragenyx, el que sea más conservador
    14.Antecedentes coagulopatía crónica, trombofilia o trastorno de activación del complemento
    15.Uso medicamentos concomitantes que alteran TP/INR, incluidos warfarina y anticoagulantes orales directos (como rivaroxaban, apixaban y edoxaban). Se permite la inclusión de pacientes que reciben medicamentos que afectan a función plaquetaria, como ácido acetilsalicílico o clopidogrel, a menos que presenten enfermedades concomitantes que, a juicio investigador, pondrían a dichos participantes en una situación de riesgo indebido al participar en estudio.
    16.Tratamiento actual con medicamentos inmunodepresores a largo plazo. Esto incluye a pacientes con afecciones autoinmunitarias tratadas con inmunodepresores y a receptores de trasplantes de órganos sólidos.
    17.Tuberculosis activa que requirió tratamiento en últimos 3 años
    18.Enfermedad por coronavirus de 2019 (COVID-19) sintomática
    19.Alcoholismo o drogadicción previa o activa que, en opinión investigador, pueda interferir con capacidad del paciente para cumplir con protocolo
    20.Haber recibido solo 1 de las 2 dosis previstas de la vacuna contra la enfermedad causada por el coronavirus de 2019 (COVID-19). Pueden participar los pacientes que no hayan recibido la vacuna contra la COVID-19 y los que hayan completado la vacunación contra la COVID-19.
    21.Intervención quirúrgica programada, incluidas las dentales, durante el periodo con DUA para los pacientes de la parte 1 o antes de la semana 10 del periodo con DR para los pacientes de la parte 2
    22.Mujeres embarazadas o en periodo de lactancia en la visita de selección (VSI del periodo con DR) o tener previsto quedarse embarazada (la participante o la pareja del participante) en cualquier momento durante el estudio
    23.Mujeres que puedan quedar embarazadas y presenten adenoma hepatocelular que no estén dispuestas a utilizar métodos anticonceptivos no hormonales
    24. Uso de cualquier PEI o producto sanitario experimental dentro de los 30 días, o 5 semividas (en el caso del PEI), lo que sea más tiempo, previos a la visita de selección (VSI del periodo con DR), o necesidad de utilizar cualquier medicamento experimental antes de completar todas las evaluaciones del estudio programadas
    E.5 End points
    E.5.1Primary end point(s)
    The incidence and severity of TEAEs, serious TEAE and related TEAEs in the SAD and RD Periods
    Incidencia e intensidad de los AAST, los AAST graves y los AAST relacionados durante los periodos con DUA y DR
    E.5.1.1Timepoint(s) of evaluation of this end point
    SAD Period: Adverse Events monitored from Screening through to D90. In the event of an ET, all efforts will be made to monitor the subject through the end of the study. At minimum, a safety follow-up phone call will occur within the 4 weeks following the subject’s last treatment.

    RD Period: Adverse Events monitored from Screening through to W48. In the event of an ET, all efforts will be made to monitor the subject through the end of the study. At minimum, a safety follow-up phone call will occur within the 4 weeks following the subject’s last treatment.
    Periodo de DUA: supervisión de acontecimientos adversos desde la selección hasta el D90. De darse una FA, se hará todo lo posible por supervisar al paciente hasta el final del estudio. Como mínimo, se hará una llamada de seguimiento de la seguridad en las 4 semanas posteriores al último tratamiento del paciente.

    Periodo de DR: supervisión de los acontecimientos adversos desde la selección hasta la S48. De darse una FA, se hará todo lo posible por supervisar al paciente hasta el final del estudio. Como mínimo, se hará una llamada de seguimiento de la seguridad en las 4 semanas posteriores al último tratamiento del paciente.
    E.5.2Secondary end point(s)
    PK parameters of AGL (amylo-α-1,6-glucosidase 4-alpha-glucanotransferase) mRNA and ATX95, including Tmax, Cmax, AUClast, AUCinf, AUCtau (RD Period only), RAUC (RD Period only), Tlast, T1/2, CL, Vss (SAD Period only)
    Parámetros FC del ARNm del AGL y ATX95, incluidos el Tmáx, Cmáx, AUCúlt, AUCinf, AUCtau (solo en el periodo con DR), RAUC (solo en el periodo con DR), Túlt, T1/2, Acl., Vdeq (solo en el periodo con DUA)
    E.5.2.1Timepoint(s) of evaluation of this end point
    SAD Period: AGL mRNA and ATX95 D0, D1, D4, D7, D14, D21 and D28
    RD Period: AGL mRNA and ATX95 W0, W1, W2, W8, W9, W10 and W12
    Periodo de DUA: ARNm de la AGL y ATX95 en D0, D1, D4, D7, D14, D21 y D28.

    Periodo DR: ARNm de la AGL y ATX95 en S0, S1, S2, S8, S9, S10 y S12.
    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 No
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Parte 1: sin enmascaramiento. Parte 2: aleatorizada, doble enmascaramiento y controlada con placebo
    Part 1 Open-label. Part 2 Randomised, Double-blind, Placebo-controlled
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA6
    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
    Germany
    Italy
    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
    The end of the study is defined as the date of the last protocol-specified visit (including safety follow-up telephone calls in the event of early termination [ET]) for the last subject in the study.
    El final del estudio se define como la fecha de la última visita especificada en el protocolo (incluidas las llamadas de seguimiento de la seguridad en caso de finalización anticipada —FA) del último paciente del estudio.
    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: 27
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    In the case of adult subjects with cognitive limitation, provide written assent (if required) and written informed consent by a legally authorized representative
    En el caso de pacientes adultos con limitaciones cognitivas, se proporcionará el asentimiento por escrito (de ser necesario) y un representante legal proporcionará el consentimiento informado por escrito.
    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 19
    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)
    Once chronic toxicity data are available, all subjects, including those previously treated with placebo, will be allowed to receive long-term open-label treatment with UX053.
    Una vez disponibles los datos de toxicidad crónica, todos los pacientes, incluidos los tratados previamente con placebo, podrán recibir tratamiento a largo plazo sin enmascaramiento con UX053.
    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 Decision2021-11-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-18
    P. End of Trial
    P.End of Trial StatusOngoing
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