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    Summary
    EudraCT Number:2018-000755-40
    Sponsor's Protocol Code Number:ATB200-03
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-01-18
    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 number2018-000755-40
    A.3Full title of the trial
    A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo
    Estudio de fase III, randomizado y doble ciego para evaluar la eficacia y la seguridad de ATB200 administrado por vía intravenosa combinado con AT2221 administrado por vía oral en pacientes adultos con enfermedad de Pompe de comienzo tardío en comparación con alglucosidasa alfa/placebo
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared with
    Alglucosidase Alfa/Placebo.
    Un estudio para investigar la eficacia y la seguridad de ATB200 intravenoso (IV) co administrado con AT2221 oral en pacientes adultos con enfermedad de Pompe comparado con alglucosidasa alfa/placebo
    A.4.1Sponsor's protocol code numberATB200-03
    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 SponsorAmicus 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 supportAmicus Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmicus Therapeutics, Inc.
    B.5.2Functional name of contact pointPatient advocacy
    B.5.3 Address:
    B.5.3.1Street Address1 Cedar Brook Drive
    B.5.3.2Town/ cityCranbury
    B.5.3.3Post codeNJ 08512
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016096622000
    B.5.5Fax number0016096625010
    B.5.6E-mailclinicaltrials@amicusrx.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/3/18/2000
    D.3 Description of the IMP
    D.3.1Product nameATB200
    D.3.2Product code ATB200
    D.3.4Pharmaceutical form Powder for concentrate for solution for injection/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 INNN/A
    D.3.9.1CAS number 420784-05-0
    D.3.9.2Current sponsor codeATB200
    D.3.9.4EV Substance CodeSUB21275
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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.IMP: 2
    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/3/18/2129
    D.3 Description of the IMP
    D.3.1Product nameAT2221
    D.3.2Product code AT2221
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMIGLUSTAT
    D.3.9.1CAS number 72599-27-0
    D.3.9.2Current sponsor codeAT2221
    D.3.9.4EV Substance CodeSUB20049
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number65
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    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) 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 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.IMP: 3
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name Myozyme
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme Corporation
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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.3.4Pharmaceutical form Powder 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 INNALGLUCOSIDASE ALFA
    D.3.9.1CAS number 420784-05-0
    D.3.9.4EV Substance CodeSUB21275
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Adult Subjects With Late Onset Pompe Disease (LOPD)
    Pacientes adultos con Enfermedad de Pompe de comienzo tardío
    E.1.1.1Medical condition in easily understood language
    A change in the genetic material resulting in lower levels of an enzyme called acid alpha-glucosidase (GAA). The GAA enzyme helps the body to break down glycogen, a type of stored sugar.
    Un cambio en el material genético resultando en bajos niveles deuna enzima denominada alglucosidasa alfa (AGA). La enzima AGA ayuda al cuerpo a descomponer el glucógeno, un tipo de azucar almacenado
    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 10075702
    E.1.2Term Pompe's disease late onset
    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
    The objective is to assess the efficacy of ATB200/AT2221 co-administration on ambulatory function, as measured by the 6 Minute Walk Test (6MWT), compared with alglucosidase alfa/placebo
    El objetivo es evaluar la eficacia de la administración combinada de ATB200/AT2221 respecto a la función de deambulación, según se mide en la prueba de deambulación de 6 minutos (PD6M), en comparación con alglucosidasa α/placebo.
    E.2.2Secondary objectives of the trial
    To assess the efficacy of ATB200/AT2221 co-administration (compared with alglucosidase alfa/placebo) on: pulmonary function as measured by sitting forced vital capacity (FVC)(%predicted),muscle strength, ealth-related patient reported outcomes,motor function,overall clinical impression as assessed by both physician and subject
    To assess the safety, tolerability, and immunogenicity of ATB200/AT2221 co administration compared with alglucosidase alfa/placebo
    To assess the effect of ATB200/AT2221 co-administration on biomarkers of muscle injury and disease substrate compared with alglucosidase alfa/placebo
    To characterize the population PK of ATB200 and alglucosidase alfa ERT experienced subjectsusing plasma total acid GAA protein level by signature peptide assay and plasma AT2221 concentration
    To explore the exposure-response relationship for ATB200/AT2221 and alglucosidase alfa/placebo co-administration
    Please refer to the protocol for the full list of objectives
    Evaluar la eficacia de la administración combinada de ATB200/AT2221 (comparada con alglucosidasa α/placebo) en:fuerza muscular,resultados comunicados por el paciente en relación con la salud, en la función pulmonar,medida como la CVF en sedestación (% previsto), funcion motora,impresión clínica global realizada por el médico y el paciente
    Evaluar la seguridad, la tolerabilidad y la inmunogenicidad de la administración combinada de ATB200/AT2221, en comparación con alglucosidasa α/placebo
    Evaluar el efecto de la administración combinada de ATB200/AT2221 sobre los biomarcadores de lesión muscular y sustrato patológico, en comparación con alglucosidasa α/placebo
    Caracterizar la FC poblacional de ATB200 y alglucosidasa α en pacientes que han recibido anteriormente tratamiento TRE utilizando la concentración plasmática total de AGA según el ensayo de péptidos distintivos y la concentración plasmática de AT2221
    Por favor, referirse al protocolo para la lista completa de objetivos
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject must provide signed informed consent prior to any study-related procedures being performed.
    2. Male and female subjects are ≥ 18 years old and weigh ≥ 40 kg at screening.
    3. Female subjects of childbearing potential and male subjects must agree to use medically accepted methods of contraception during the study and for 90 days after the last dose of study drug.
    4. Subject must have a diagnosis of LOPD based on documentation of one of the following:
    a. deficiency of GAA enzyme
    b. GAA genotyping
    5. Subject is classified as one of the following with respect to ERT status:
    a. ERT-experienced, defined as currently receiving standard of care ERT (alglucosidase alfa) at the recommended dose and regimen (ie, 20 mg/kg dose every 2 weeks) for ≥ 24 months
    b. ERT naïve, defined as never having received investigational or commercially available ERT
    6. Subject has a sitting FVC ≥ 30% of the predicted value for healthy adults (National Health and Nutrition Examination Survey III) at screening.
    7. Subject performs two 6MWTs at screening that are valid, as determined by the clinical evaluator, and that meet all of the following criteria:
    a. both screening values of 6MWD are ≥ 75 meters
    b. both screening values of 6MWD are ≤ 90% of the predicted value for healthy adults
    c. the lower value of 6MWD is within 20% of the higher value of 6MWD
    1.Los pacientes deben proporcionar el consentimiento informado firmado antes de realizarse cualquier procedimiento relacionado con el estudio.
    2.Pacientes de ambos sexos de ≥18 años de edad que pesen ≥40 kg en el screening.
    3.Las pacientes en edad fértil y los pacientes varones deben estar de acuerdo en emplear métodos anticonceptivos aceptados desde el punto de vista médico, durante el estudio y hasta 90 días después de recibir la última dosis del fármaco del estudio.
    4.El paciente debe presentar un diagnóstico de EPCT basado en la documentación de uno de los siguientes:
    a.Deficiencia de la enzima AGA
    b.Genotipado del gen AGAA
    5.El paciente está clasificado como uno de los siguientes en cuanto a su relación con el TRE:
    a.con TRE previo, que se define como estar recibiendo actualmente un TRE de referencia (alglucosidasa α) con la dosis y la pauta posológica recomendadas (es decir, una dosis de 20 mg/kg cada 2 semanas) durante ≥24 meses.
    b.sin TRE previo, que se define como no haber recibido nunca antes un TRE en fase de investigación o comercializado.
    6.El paciente presenta una CVF en sedestación de ≥30 % del valor previsto para personas adultas sanas (Encuesta Nacional de Examen de Salud y Nutrición III de Estados Unidos) en el screening.
    7.El paciente realiza dos PD6M en el screening que son válidas, según determine el evaluador clínico, y que cumplen todos los criterios siguientes:
    a.los dos valores de la DR6M en el screening son ≥75 metros.
    b.los dos valores de la DR6M en el screening son ≤90 % del valor previsto en personas adultas sanas.
    c.el valor inferior de la DR6M se encuentra dentro del 20 % del valor superior de la PD6M.
    E.4Principal exclusion criteria
    1. Subject has received any investigational therapy or pharmacological treatment for Pompe disease, other than alglucosidase alfa, within 30 days or 5 half lives of the therapy or treatment, whichever is longer, before Day 1 or is anticipated to do so during the study.
    2. Subject has received gene therapy for Pompe disease.
    3. Subject is taking any of the following prohibited medications within 30 days before Day 1:
    • miglitol (eg, Glyset)
    • miglustat (eg, Zavesca)
    • acarbose (eg, Precose or Glucobay)
    • voglibose (eg, Volix, Vocarb, or Volibo)
    Note: None of these medications have a half-life that, when multiplied by 5, is longer than 30 days.
    4. Subject requires the use of invasive or noninvasive ventilation support for > 6 hours per day while awake.
    5. Subject has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa or AT2221
    6. Subject has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the subject or may compromise his/her ability to comply with or adversely impact protocol requirements. This includes clinical depression (as diagnosed by a psychiatrist or other mental health professional) with uncontrolled or poorly controlled symptoms.
    7. Subject, if female, is pregnant or breastfeeding at screening.
    8. Subject, whether male or female, is planning to conceive a child during the study.
    9. Subject does not have documentation of diagnosis of Pompe disease and refuses to undergo genetic testing
    1.El paciente ha recibido cualquier terapia en investigación o tratamiento farmacológico para la enfermedad de Pompe, aparte de la alglucosidasa α, durante los 30 días o las 5 semividas de la terapia o el tratamiento (el período que sea más largo) previos al Día 1, o si está previsto que lo reciba durante el estudio.
    2.El paciente ha recibido terapia génica para la enfermedad de Pompe.
    3.El paciente está tomando alguno de los medicamentos prohibidos siguientes durante los 30 días previos al Día 1:
    •Miglitol (p. ej., Glyset)
    •Miglustat (p. ej., Zavesca)
    •Acarbosa (p. ej., Precose o Glucobay)
    •Voglibosa (p. ej., Volix, Vocarb o Volibo)
    Nota: Ninguno de estos medicamentos tiene una semivida que, al ser multiplicada por 5, sea superior a 30 días.
    4.El paciente precisa el uso de respiración asistida, invasiva o no, durante más de 6 horas al día en vigilia.
    5.El paciente tiene hipersensibilidad a alguno de los excipientes de ATB200, alglucosidasa alfa o AT2221
    6.El paciente padece una enfermedad u otra circunstancia atenuante que, a juicio del Investigador o el monitor médico, podría suponer un riesgo de seguridad innecesario para el paciente, comprometer su capacidad para cumplir los requisitos del protocolo o influir negativamente en dichos requisitos. Esto incluye la depresión clínica (según el diagnóstico de un psiquiatra u otro profesional de la salud mental) con síntomas no controlados o controlados inadecuadamente.
    7.La paciente está embarazada o en período de lactancia durante el screening.
    8.El paciente, hombre o mujer, está planeando concebir un hijo durante el estudio.
    9.El paciente no tiene diagnóstico documentado de enfermedad de Pompe y se niega a realizarse análisis genéticos
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the change from baseline to Week 52 in 6MWD.
    El criterio de valoración principal de la eficacia es el cambio en la DR6M desde el valor basal hasta la Semana 52.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At visits Week 12, 26 and Week 52.
    En las visitas de las Semanas 12, 26 y semana 52.
    E.5.2Secondary end point(s)
    • change from baseline to Week 52 in the manual muscle test score for the lower extremities
    • change from baseline to Week 52 in the total score for the PROMIS – physical function
    • change from baseline to Week 52 in the total score for the PROMIS – fatigue
    • change from baseline to Week 52 in GSGC total score
    • change from baseline to Week 52 in sitting FVC (% predicted)
    • change from baseline to Week 26 in 6MWD
    Other secondary efficacy endpoints are as follows:
    • change from baseline to Week 52 in the following variables related to motor function:
    - time to complete the 10-meter walk (ie, assessment of gait) of the GSGC test
    - time to complete the 4-stair climb of the GSGC test
    - time to complete the Gower’s maneuver of the GSGC test
    - time to arise from a chair as part of the GSGC test
    - time to complete the TUG test
    • change from baseline to Week 52 in the following variables related to muscle strength:
    - manual muscle test score for the upper extremities
    - manual muscle test total score
    - quantitative muscle test value (kg) for the upper extremities
    - quantitative muscle test value (kg) for the lower extremities
    - quantitative muscle test total value (kg)
    • change from baseline to Week 52 in the following variables from patient reported outcome measures:
    - total score for the PROMIS – dyspnea
    - total score for the PROMIS – upper extremity
    - R-PAct Scale total score
    - EQ-5D-5L health status
    • actual value of the subject’s functional status (improving, stable, or declining) pertaining to the effects of study drug in the following areas of life at Week 52, as measured by the Subject’s Global Impression of Change
    - overall physical wellbeing
    - effort of breathing
    - muscle strength
    - muscle function
    - ability to move around
    - activities of daily living
    - energy level
    - level of muscular pain
    • actual value of the subject’s functional status (improving, stable, or declining) at Week 52, as measured by the Physician’s Global Impression of Change
    • change from baseline to Week 52 in the following measures of pulmonary function, as follows:
    - sitting SVC (% predicted)
    - MIP (cmH2O)
    - MIP (% predicted)
    - MEP (cmH2O)
    - MEP (% predicted)
    - SNIP (cmH2O)
    Pharmacodynamic endpoints are as follows:
    • change from baseline to Week 52 in serum CK level
    • change from baseline to Week 52 in urinary Hex4 level
    •el cambio en la puntuación de la prueba muscular manual de las extremidades inferiores desde el momento basal hasta la Semana 52.
    •el cambio en la puntuación total de la función física de PROMIS desde el momento basal hasta la Semana 52.
    •el cambio en la puntuación total del cansancio de PROMIS desde el momento basal hasta la Semana 52.
    •el cambio en la puntuación total de la maniobra de MEGS desde el momento basal hasta la Semana 52.
    •el cambio en la CVF en sedestación (como % predicho) desde el momento basal hasta la Semana 52.
    •el cambio en la DR6M desde el momento basal hasta la Semana 26.
    Los demás criterios de valoración secundarios de la eficacia son los siguientes:
    •el cambio desde el momento basal hasta la Semana 52 en las siguientes variables relacionadas con la función motora:
     - tiempo que tarda en recorrer una distancia de 10 metros (es decir, evaluación de la marcha) según la prueba de MEGS.
     - tiempo que tarda en subir 4 escaleras según la prueba de MEGS.
     - tiempo que tarda en completar la maniobra de Gower según la prueba de MEGS.
     - tiempo que tarda en levantarse de una silla según la prueba de MEGS.
     - tiempo que tarda en completar la PCLC.
    •el cambio desde el momento basal hasta la Semana 52 en las siguientes variables relacionadas con la fuerza muscular:
     - puntuación en la prueba muscular manual de las extremidades superiores
     - puntuación total en la prueba muscular manual
     - valor cuantitativo (kg) en la prueba muscular de las extremidades superiores
     - valor cuantitativo (kg) en la prueba muscular de las extremidades inferiores
     - valor cuantitativo (kg) total en la prueba muscular
    •el cambio desde el momento basal hasta la Semana 52 en las siguientes variables relacionadas con los resultados comunicados por el paciente:
     - puntuación total en la evaluación de la disnea según PROMIS
     - puntuación total en la evaluación de las extremidades superiores según PROMIS
     - puntuación total en la escala AEEP-R
     - estado de salud según el EQ-5D-5L
    •valor actual del estado funcional del paciente (mejorando, estable o empeorando) referente a los efectos del fármaco del estudio en las siguientes áreas de la vida en la Semana 52, determinado según la impresión global del cambio percibida por el paciente
     - bienestar físico general
    - esfuerzo para respirar
     - fuerza muscular
     - función muscular
     - capacidad para desplazarse
     - actividades de la vida cotidiana
     - nivel de energía
     - intensidad del dolor muscular
    •valor actual del estado funcional del paciente (mejorando, estable o empeorando) en la Semana 52, determinado según la impresión global del cambio percibido por el médico
    •el cambio desde el momento basal hasta la Semana 52 en las siguientes mediciones de la función pulmonar:
     - CVL en sedestación (como % predicho)
     - PIM (cm H2O)
     - PIM (como % predicho)
     - PEM (cm H2O)
    - PEM (como % predicho)
     - PIN (cm H2O)
    Los criterios de valoración de la farmacodinámica son los siguientes:
    •el cambio en la concentración sérica de la CK desde el momento basal hasta la Semana 52
    •el cambio en la concentración de la Hex4 en orina desde el momento basal hasta la Semana 52
    E.5.2.1Timepoint(s) of evaluation of this end point
    Almost all timepoint evaluations happen at visit week 52. Change from baseline for 6MWT is also measured at visit week 26.
    La mayoría de las evaluciones son a la visita de la semana 52. Cambio en la DR6M desde el valor basal hasta la Semana 52 se medirá también en la Semana 26
    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 Yes
    E.6.13.1Other scope of the trial description
    Effect on biomarkers of muscle injury and disease substrate
    Explore the exposure-response relationship for ATB200/AT2221 and alglucosidase alfa/placebo co-administration
    Efecto sobre biomarcadores de lesión muscular y sustrato de enfermedades.
    Explorar la relación exposición-respuesta para ATB200 / AT2221 y la coadministración de alglucosidasa alfa / placebo
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    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 EEA30
    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
    Argentina
    Australia
    Austria
    Belgium
    Bosnia and Herzegovina
    Bulgaria
    Canada
    Denmark
    France
    Germany
    Greece
    Hungary
    Italy
    Japan
    Korea, Republic of
    Netherlands
    Poland
    Romania
    Slovakia
    Slovenia
    Spain
    Sweden
    Taiwan
    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
    LPLV (November 2020)
    ultima visita del último paciente (Noviembre 2020)
    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: 90
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 60
    F.4.2.2In the whole clinical trial 110
    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 participation in the trial, patients will have the option to be treated with the current standard of care therapy or to enrol into an Open-Label Extension study and continue receiving the investigational treatment.
    Después de participar en el ensayo, los pacientes tendrán la opción de ser tratados con la terapia estándar actual o inscribirse en un estudio de Extensión en abierto y continuar recibiendo el tratamiento de investigación.
    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 Decision2019-02-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-12-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-12-15
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