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    Summary
    EudraCT Number:2016-003257-15
    Sponsor's Protocol Code Number:A083-02
    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:2018-01-04
    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 number2016-003257-15
    A.3Full title of the trial
    A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy
    Estudio de fase II, randomizado, doble ciego y controlado con placebo, de ACE-083 en pacientes con distrofia muscular facioescapulohumeral
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2 clinical trial that is randomized and controlled by a placebo (similar to the product under investigation but does not have any therapeutic effect) of ACE-083 in Patients with muscular dystrophy (on face, around shoulder blades, in upper arms). The study is a double-blind study, this means that neither you nor your doctor will know if you receive study drug or placebo
    Estudio de fase II, randomizado y controlado con placebo (similar al producto en investigación pero sin efecto terapeútico) de ACE-083 en pacientes con distrofia muscular (en la cara, alrededor de lo omóplatos y en la parte superior de los brazos)
    A.4.1Sponsor's protocol code numberA083-02
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02927080
    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 SponsorAcceleron Pharma 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 supportAcceleron Pharma Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAcceleron Pharma Inc.
    B.5.2Functional name of contact pointKenneth M. Attie
    B.5.3 Address:
    B.5.3.1Street Address128 Sidney Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02139
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1617649 9200
    B.5.5Fax number+1617576 0479
    B.5.6E-mailkattie@acceleronpharma.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 nameACE-083
    D.3.2Product code ACE-083
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNpending
    D.3.9.1CAS number 1650554-49-6
    D.3.9.2Current sponsor codeACE-083
    D.3.9.3Other descriptive nameACE-083
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 placeboSolution for injection
    D.8.4Route of administration of the placeboIntramuscular use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Facioscapulohumeral Muscular Dystrophy
    Distrofia muscular facioescapulohumeral
    E.1.1.1Medical condition in easily understood language
    Muscle disease characterized by muscle weakness and wasting (atrophy). The most affected muscles are the face (facio-), around the shoulder blades (scapulo-), and in the upper arms (humeral).
    Enfermedad muscular caracterizada por debilidad y desgaste muscular(atrofia).Músculos más afectados: cara(facio-),alrededor de los omóplatos (escapulo-),y parte superior de los brazos (humeral).
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10064087
    E.1.2Term Facioscapulohumeral muscular dystrophy
    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
    Part 1:
    To evaluate the safety and tolerability of ACE-083 in patients with facioscapulohumeral muscular dystrophy (FSHD)
    Part 2:
    To determine whether treatment with ACE-083 increases total muscle volume of the injected muscle in patients with FSHD
    Parte 1
    Evaluar la seguridad y la tolerabilidad de ACE-083 en pacientes con distrofia muscular facioescapulohumeral (DMFEH).
    Parte 2
    Determinar si el tratamiento con ACE-083 aumenta el volumen muscular total del músculo inyectado en los pacientes con DMFEH
    E.2.2Secondary objectives of the trial
    Part 1:
    • To determine the recommended dose level(s) of ACE-083 for Part 2
    • To evaluate changes in muscle volume and intramuscular fat fraction of the injected muscle
    • To evaluate changes in strength of the injected muscle
    • To estimate the systemic exposure of ACE 083 when administered as a local muscle injection
    Part 2:
    • To determine whether treatment with ACE-083 decreases intramuscular fat fraction of the injected muscle
    • To determine whether treatment with ACE-083 increases strength of the injected muscle
    • To determine whether treatment with ACE-083 improves motor function related to the injected muscle
    • To evaluate changes in patient-reported measures of FSHD-HI total score and subscale scores
    • To evaluate the safety and tolerability of ACE-083
    • To estimate the systemic exposure of ACE-083 when administered as a local muscle injection
    Parte 1:
    Determinar el nivel o los niveles de dosis recomendados de ACE-083 para la Parte 2
    Evaluar la variación en el volumen muscular y la fracción de tejido adiposo intramuscular del músculo inyectado
    Evaluar la variación en la fuerza del músculo inyectado
    Estimar la exposición sistémica a ACE-083 cuando se administra mediante inyección local en un músculo
    Parte 2:
    Determinar si el tratamiento con ACE-083 disminuye la fracción de tejido adiposo intramuscular del músculo inyectado
    Determinar si el tratamiento con ACE-083 aumenta la fuerza muscular del músculo inyectado
    Determinar si el tratamiento con ACE-083 mejora la función motora relacionada con el músculo inyectado
    Evaluar los cambios en los resultados comunicados por los pacientes relativos a la puntuación total y las puntuaciones de las subescalas del índice FSHD-HI
    Evaluar la seguridad y tolerabilidad de ACE-083
    Estimar la exposición sistémica a ACE-083 cuando se administra mediante inyección local en un músculo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age ≥ 18 years
    2. Genetically-confirmed FSHD1 or FSHD2 (or a first-degree relative with genetically confirmed FSHD1 or FSHD2) and clinical findings meeting FSHD criteria
    3. Part 1 TA cohorts:
    a. 6-minute walk distance (6MWD) ≥ 150 meters(without a brace)
    b. Left and/or right ankle dorsiflexion Medical Research Council (MRC) manual muscle testing (MMT) grade 3 to 4+, inclusive
    Note: Contralateral side may be MRC MMT grade 3 to 5
    Part 1 BB cohorts:
    a. Left and/or right elbow flexion MRC MMT grade 3 to 4+, inclusive
    Note: Contralateral side may be any MRC MMT grade
    Part 2 TA cohorts:
    a. 6MWD ≥ 150 and ≤ 500 meters (without a brace); a maximum of 20% of enrolled patients with 6MWD ≥ 450 meters will be included
    b. Left and right ankle dorsiflexion MRC MMT grade 3 to 4+, inclusive
    Part 2 BB cohorts:
    a. Left and right elbow flexion MRC MMT grade 3 to 4+, inclusive
    4. Females of childbearing potential (defined as sexually mature women who have not undergone hysterectomy or bilateral oophorectomy, or are not naturally postmenopausal ≥ 24 consecutive months) must have negative urine pregnancy test prior to enrollment and use highly effective birth control methods (abstinence, oral contraceptives, barrier method with spermicide, or surgical sterilization) during study participation and for 8 weeks following the last dose of ACE-083. Males must agree to use a condom during any sexual contact with females of childbearing potential while participating in the study and for 8 weeks following the last dose of ACE-083, even if he has undergone a successful vasectomy. Patients must be counseled concerning measures to be used to prevent pregnancy prior to the first dose of ACE-083.
    5. Ability to adhere to the study visit schedule/procedures, and to understand and comply with protocol requirements
    6. Signed written informed consent
    1. Edad ≥18 años
    2. DMFEH1 o DMFEH1 confirmada genéticamente (o un pariente de primer grado con DMFEH1 o DMFEH2 confirmada genéticamente) y signos clínicos que cumplen los criterios de la DMFEH
    3. Para las cohortes con afectación del músculo TA en la Parte 1:
    a. Distancia recorrida en la prueba de marcha de 6 minutos (PM6M) ≥150 metros sin un aparato ortopédico.
    b. Grado 3 a 4+, inclusive, en las pruebas musculares manuales (MMT, del inglés manual muscle testing) del Medical Research Council (MRC) con dorsiflexión del tobillo izquierdo o derecho (o ambos)
    Nota: El lado contralateral puede tener grado 3 a 5 en las MMT del MRC.
    Para las cohortes con afectación del músculo BB en la Parte 1
    a. Grado 3 a 4+, inclusive, en las MMT del MRC con flexión del codo izquierdo o derecho (o ambos)
    Nota: El lado contralateral puede tener cualquier grado en las MMT del MRC.
    Para las cohortes con afectación del músculo TA de la Parte 2:
    a. Distancia recorrida en la PM6M ≥150 y ≤500 metros (sin dispositivo ortopédico); se incluirán un máximo del 20 % de pacientes con una distancia recorrida en la PM6M ≥450 metros
    b. Grado 3 a 4+, inclusive, en las MMT del MRC con dorsiflexión del tobillo izquierdo y derecho
    Para las cohortes con afectación del músculo BB de la Parte 2:
    a. Grado 3 a 4+, inclusive, en las MMT del MRC con flexión de codo izquierdo y derecho
    4. Las mujeres con capacidad para procrear (es decir, mujeres sexualmente maduras a las que no se les ha realizado una histerectomía o una ooforectomía bilateral, o que no son posmenopáusicas de forma natural durante ≥24 meses consecutivos) deben tener un resultado negativo en una prueba de embarazo en orina antes de la inclusión y utilizar métodos anticonceptivos muy eficaces (abstinencia sexual, anticonceptivos orales, método de barrera con espermicida o esterilización quirúrgica) durante su participación en el estudio y las ocho semanas siguientes a la última dosis de
    ACE-083. Los varones deben aceptar el uso de un preservativo cuando mantengan relaciones sexuales con mujeres con capacidad de procrear mientras participen en el estudio y durante las ocho semanas siguientes a la administración de la última dosis de ACE-083, aunque se les haya realizado con éxito una vasectomía. Antes de la administración de la primera dosis de ACE-083, los pacientes deben recibir asesoramiento sobre los métodos que deben emplear para prevenir los embarazos.
    5. Posibilidad de cumplir con el calendario de visitas y los procedimientos del estudio, y de entender y cumplir los requisitos del protocolo
    6. Consentimiento informado firmado por escrito.
    E.4Principal exclusion criteria
    1. History of active malignancy, with the exception of fully excised or treated basal cell carcinoma, cervical carcinoma in-situ, or ≤ 2 squamous cell carcinomas of the skin
    2. Symptomatic cardiopulmonary disease, significant functional impairment, or other co morbidities that in the opinion of the investigator would limit a patient’s ability to complete strength and/or functional assessments on study
    3. Renal impairment (serum creatinine ≥ 2 times the upper limit of normal [ULN])
    4. Aspartate transaminase (AST) and/or alanine transaminase (ALT) ≥ 3 times ULN
    5. Increased risk of bleeding (i.e., due to hemophilia, platelet disorders, or use of any anticoagulation/platelet modifying therapies up to 2 weeks prior to Study Day 1; low dose aspirin [≤ 100 mg daily] is permitted)
    6. Major surgery within 4 weeks prior to Study Day 1
    7. Chronic systemic corticosteroids (≥ 2 weeks) within 4 weeks before Study Day 1 and for duration of study; intra-articular/topical/inhaled therapeutic or physiologic doses of corticosteroids are permitted
    8. Androgens or growth hormone within 6 months before Study Day 1 and for duration of study; topical physiologic androgen replacement is permitted
    9. Any change in medications potentially affecting muscle strength or function within 4 weeks of Study Day 1 and for duration of study (e.g., creatine, CoQ10, systemic beta-adrenergic agonists)
    10. Previous exposure to any investigational agent potentially affecting muscle volume, strength, or function within 5 half-lives of last dose or 4 weeks of Study Day 1 if half-life is unknown, or any prior exposure to ACE-083
    11. Significant change in physical activity or exercise (e.g., significant increase or decrease in intensity or frequency) within 8 weeks before Study Day 1 or inability to maintain the baseline level of physical activity throughout the study
    12. Any condition that would prevent MRI scanning or compromise the ability to obtain a clear and interpretable scan of the TA or BB muscles, as applicable (e.g., pacemaker, knee/hip replacement, or metallic implants)
    13. Known active substance abuse, including alcohol
    14. History of sensitivity to protein pharmaceuticals
    15. Female that is lactating/breast-feeding
    1. Antecedentes de neoplasia maligna activa, con excepción de carcinoma basocelular extirpado en su totalidad o tratado, carcinoma de cuello uterino in situ o ≤2 carcinomas cutáneos espinocelulares
    2. Enfermedad cardiopulmonar sintomática, deterioro funcional significativo u otras enfermedades concomitantes que, según el dictamen del investigador, limitarían la capacidad del paciente para completar las evaluaciones funcionales y de la fuerza durante el estudio
    3. Deterioro renal (creatinina sérica ≥2 veces el límite superior de la normalidad [LSN])
    4. Concentraciones de aspartato-aminotransferasa (AST) o alanina-aminotransferasa (ALAT) ≥3 veces el LSN
    5. Aumento del riesgo de hemorragia (es decir, debido a hemofilia, trastornos plaquetarios o uso de tratamientos anticoagulantes o modificadores de las plaquetas hasta dos semanas antes del día 1 del estudio; se permite la administración de acetilsalicílico en dosis bajas [≤100 mg diarios])
    6. Cirugía mayor en las cuatro semanas anteriores al día 1 del estudio
    7. Tratamiento prolongado (≥2 semanas) con corticosteroides sistémicos en las cuatro semanas anteriores al día 1 del estudio y durante todo el estudio; se permite la administración de dosis terapéuticas o fisiológicas intraarticulares, tópicas o inhaladas de corticosteroides
    8. Andrógenos u hormona del crecimiento en los 6 meses previos al día 1 del estudio y durante todo el estudio; se permite tratamiento de restitución androgénica en dosis fisiológicas tópicas.
    9. Cualquier cambio en medicamentos que podrían afectar a la fuerza o la función muscular en las 4 semanas previas al día 1 del estudio y durante todo el estudio (p. ej., creatina, CoQ10, agonistas adrenérgicos β sistémicos)
    10. Exposición previa a cualquier fármaco en investigación que podría afectar al volumen, la fuerza o la función muscular en un plazo de cinco semividas desde la última dosis o en las cuatro semanas
    11. Variación significativa en la actividad o ejercicio físico (p. ej., aumento o disminución considerables de su intensidad o frecuencia) en las ocho semanas anteriores al día 1 del estudio o imposibilidad de mantener el nivel basal de actividad física durante todo el estudio
    12. Cualquier circunstancia que impediría la realización de una RMN o que hiciera peligrar la posibilidad de obtener imágenes claras e interpretables de los músculos TA o TB, según el caso (p. ej. marcapasos, artroplastia de rodilla o cadera, o implantes metálicos)
    13. Consumo activo de sustancias, como alcohol
    14. Antecedentes de sensibilidad a fármacos elaborados con proteínas
    15. Mujeres en periodo de lactancia
    E.5 End points
    E.5.1Primary end point(s)
    Part 1:
    • Presence and nature of AEs including injection site reactions and changes in clinical laboratory parameters
    Part2:
    • Percent change from baseline in total muscle volume of the injected muscle
    Parte 1:
    -Presencia y naturaleza de los EAs incluyendo reacciones en el lugar de inyección y cambios en los parámetros clínicos de laboratorio.
    Parte 2:
    -Cambio porcentual respecto al periodo basal en el volumen muscular total del músculo inyectado
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part 1:
    In Part 1 SRT will meet to review data for each cohort when at least 4 patients within a cohort have completed their Day 43 visit.
    Safety assessments from Day 1 through Day 141/EOS include monitoring of adverse events (AEs), injection site reactions, concomitant medications, clinical laboratory assessments (including hematology, chemistry, and ADA), urinalysis, vital signs, and physical examination findings.
    Part 2:
    Percent change in total muscle volume from baseline of the injected TA or BB 3 weeks after the last dose of the double-blind treatment period.
    En la Parte 1 el ERS se reunirá para revisar los datos de cada cohorte cuando un mínimo de cuatro pacientes de una misma cohorte hayan completado la visita del día 43.
    Las evaluaciones de seguridad desde el día 1 hasta el día 141/FdE incluyen seguimiento de los efectos adversos (EA), reacciones en el sitio de la inyección, medicaciones concomitantes, evaluaciones de laboratorio clínico (incluyendo hematología, química, y ADA), análisis de orina, signos vitales y examen físico.
    Parte 2:
    Cambio porcentual respecto al periodo basal en el volumen muscular total inyectado TA o BB BB 3 semanas después de la última dosis del período de tratamiento doble ciego
    E.5.2Secondary end point(s)
    Part 1:
    • dose selection for part 2 will be done following completion of dosing for all part 1 patients
    • Percent change from baseline in total muscle volume of the injected muscle
    • Percent and absolute change from baseline in intramuscular fat fraction of the injected muscle
    • Percent change from baseline in strength measurements
    • Systemic exposure by changes in serum concentrations over time
    Part 2:
    • Dose recommendation from SRT
    • Percent and absolute change from baseline in intramuscular fat fraction of the injected muscle
    • Percent change from baseline in strength measurements
    • Percent change from baseline in functional assessments
    • Percent change from baseline in FSHD-HI total score as well as percent change in FSHD-HI subscale scores
    • Presence and nature of AEs including injection site reactions and changes in clinical laboratory parameters
    • Systemic exposure by change in serum concentrations over time
    Parte 1:
    -La selección de dosis para la parte 2 se realizará una vez completada la administración para todos los pacientes de la parte 1
    -Cambio porcentual respecto al periodo basal en el volumen muscular total del músculo inyectado.
    -Cambio porcentual y absoluto respecto al periodo basal en la fracción de tejido adiposo intramuscular del músculo inyectado
    -Cambio porcentual respecto al periodo basal en las mediciones de resistencia
    -Exposición sistémica por cambios en las concentraciones séricas en el tiempo
    Parte 2:
    -Recomenación de dosis del ERS
    -Cambio porcentual y absoluto respecto al periodo basal en la fracción de tejido adiposo intramuscular del músculo inyectado
    -Cambio porcentual respecto al periodo basal en las mediciones de resistencia
    -Cambio porcentual respecto al periodo basal en las evaluaciones funcionales
    -Cambio porcentual respecto al periodo basal en las puntuaciones totales en la escala FSHD-HI así como el cambio porcentual en la puntuación de la subescala FSHD-HI
    -Presencia y naturaleza de los EAs incluyendo reacciones en el lugar de inyección y cambios en los parámetros clínicos de laboratorio.
    -Exposición sistémica por cambios en las concentraciones séricas en el tiempo
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part 1:
    •SRT data review per cohort when at least 4 patients in a cohort completed Day 43 visit.
    •Total muscle volume of the injected muscle assessed throughout the study
    •Intramuscular fat fraction of the injected muscle assessed by MRI at Day 1, 43, 106, 141
    •Strength measurements assessed from Day -28 through Day 141
    •Systemic exposure assessed from Day 1 through Day 141
    Part 2:
    •Intramuscular fat fraction assessment by MRI at Day 1, 43, 106, 190, 295, 358, 393.
    •Strength measurements from Day -28 through Day 393
    •Functional assessments from Day -28 through Day 393
    •FSHD-HI total score and FSHD-HI subscale scores assessed from Day -28 through Day 393
    •AEs assessed from Day 1 through Day 393
    •Systemic exposure, serum PK assessment at Day 1, 2, 8, 85, 86, 190, 295, 358, 393
    Parte1
    Revisión de datos/cohorte cuando un mínimo de 4 pacientes de una misma cohorte hayan completado visita día 43
    Volumen muscular total del músculo inyectado evaluado durante el estudio
    Fracción de tejido adiposo intramuscular del músculo inyectado evaluada mediante RMNdías1,43,106,141
    Mediciones fuerza evaluadas de día -28 a día141
    Exposición sistémica evaluada de día 1 a día141
    Parte 2
    Fracción tejido adiposo intramuscular del músculo inyectado evaluada mediante RMN días 1,43,106,190,295,358,393
    -Mediciones fuerza de día -28 a día 393
    Evaluaciones funcionales de día -28 a día 393
    Puntuación total escalaFSHD-HIy puntuación en subescala FSHD-HIevaluadas de día -28 a día393
    EAs evaluados de dia1a día393
    Exposición sistémica,evaluación PK días 1,2,8,85,86,190,295,358,393
    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 Yes
    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
    Tolerability
    Tolerabilidad
    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 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 Yes
    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 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 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 No
    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 States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    UVUP (última visita último paciente)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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: 92
    F.1.3Elderly (>=65 years) No
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 92
    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)
    Patients will return to standard-of-care after study completion.
    Los pacientes volverán a la práctica clínica habitual después de la terminación del 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 Decision2018-04-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-03-14
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2019-09-17
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