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    Summary
    EudraCT Number:2019-000305-79
    Sponsor's Protocol Code Number:A083-04
    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:2019-04-12
    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 number2019-000305-79
    A.3Full title of the trial
    An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) Previously Enrolled in Study A083-02 and in Patients with Charcot-Marie Tooth (CMT) Disease Types 1 and X Previously Enrolled in Study A083-03
    Estudio de extensión abierto para evaluar los efectos a largo plazo de ACE-083 en pacientes con distrofia muscular facioescapulohumeral (DFEH) que han participado anteriormente en el estudio A083-02 y en pacientes con enfermedad de Charcot-Marie-Tooth (CMT)  de tipos 1 y X que han participado anteriormente en el estudio A083-03
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An extension study to the A083-02 and A083-03 studies that evaluate the long-term effect of the ACE-083 investigational product in patients with Facioscapulohumeral Muscular Dystrophy (FSHD) previously participated in study A083-02 and also in patients with Charcot-Marie Tooth (CMT) disease type 1 and X who previously participated in study A083-03.
    Estudio de extensión de los estudios A083-02 y A083-03 que evalua los efectos a largo plazo del medicamento en investigación ACE-083 en pacientes con distrofia muscular facioescapulohumeral (DFEH) que han participado anteriormente en el estudio A083-02 y en pacientes con enfermedad de Charcot-Marie-Tooth (CMT) de tipos 1 y X que han participado anteriormente en el estudio A083-03
    A.4.1Sponsor's protocol code numberA083-04
    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 Lyophilisate for 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 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
    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
    To evaluate the long-term safety and pharmacodynamic (PD) effects of ACE-083 in patients with facioscapulohumeral muscular dystrophy (FSHD) previously enrolled in Study A083-02 and in patients with Charcot Marie Tooth (CMT) disease types 1 and X (CMT1 and CMTX) previously enrolled in Study A083-03.
    Evaluar la seguridad y los efectos farmacodinámicos (FD) a largo plazo de ACE-083 en pacientes con distrofia muscular facioescapulohumeral (DFEH) que han participado anteriormente en el estudio A083-02 y en pacientes con enfermedad de Charcot Marie Tooth de tipos 1 y X (CMT1 y CMTX) que han participado anteriormente en el estudio A083-03.
    E.2.2Secondary objectives of the trial
    •To evaluate the safety and PD effects of every 4 week (q4w) dosing in the loading phase, and of q4w and q8w dosing in the maintenance phase.
    •To evaluate changes in strength, motor function, and quality of life (patient-reported outcomes) during the maintenance and loading phases of treatment.
    •To evaluate the pharmacokinetics (PK) of ACE-083 when administered as a local muscle injection during the maintenance and loading phases of treatment.
    •Evaluar la seguridad y los efectos farmacodinámicos de la administración cada 4 semanas en la fase de carga y de la administración cada 4 y 8 semanas en la fase de mantenimiento.
    •Evaluar variaciones de la fuerza, función motora y calidad de vida (resultados comunicados por los pacientes) durante las fases de mantenimiento y carga del tratamiento.
    •Evaluar la farmacocinética (FC) de ACE-083 cuando se administra en inyección muscular local durante las fases de mantenimiento y carga del tratamiento.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Completion of treatment with study drug per protocol and completion of the end of treatment (ET) visit in Study A083-02 or Study A083-03
    2. 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 a 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. Hormonal birth control use must be stable for at least 14 days prior to Day 1. 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 they have undergone a vasectomy. Subjects must be counseled about contraception prior to the first dose of ACE-083 and every three months thereafter during the study.
    3. Ability to adhere to the study visit schedule/procedures and to understand and comply with protocol requirements
    4. Signed written informed consent
    1.Finalización del tratamiento con el fármaco del estudio según el protocolo y finalización de la visita de final del tratamiento (FT) en el estudio A083-02 o el estudio A083-03.
    2.Las mujeres en edad fértil (definidas como aquellas sexualmente maduras que no se hayan sometido a una histerectomía u ooforectomía bilateral o que no sean posmenopáusicas de forma natural durante ≥ 24 meses consecutivos) deberán dar negativo en una prueba de embarazo en orina antes de su inclusión y utilizar métodos anticonceptivos muy eficaces (abstinencia, anticonceptivos orales, método de barrera con espermicida o esterilización quirúrgica) durante la participación en el estudio y hasta 8 semanas después de recibir la última dosis de ACE-083. El uso de anticonceptivos hormonales deberá haberse mantenido estable durante al menos 14 días antes del día 1. Los varones deberán comprometerse a utilizar preservativo durante cualquier contacto sexual con mujeres en edad fértil mientras participen en el estudio y hasta 8 semanas después de recibir la última dosis de ACE-083, aunque se hayan sometido a una vasectomía. Los sujetos deberán recibir asesoramiento sobre anticoncepción antes de recibir la primera dosis de ACE-083 y, posteriormente, cada tres meses durante todo el estudio.
    3.Capacidad de cumplir el calendario de visitas y los procedimientos del estudio y de comprender y cumplir los requisitos del protocolo.
    4.Consentimiento informado por escrito firmado.
    E.4Principal exclusion criteria
    1. Current/active malignancy (e.g., remission less than 5 years’ duration), with the exception of fully excised or treated basal cell carcinoma, cervical carcinoma in-situ, or ≤ 2 squamous cell carcinomas of the skin
    2. Co-morbidities, including symptomatic cardiopulmonary disease, significant orthopedic or neuropathic pain, or other conditions that, in the opinion of the investigator, would limit a subject’s ability to complete strength and/or functional assessments
    3. Type 1 or type 2 diabetes mellitus
    4. Thyroid disorder unless condition is stable with no change in treatment for at least 4 weeks before the first dose and no expected change for duration of study
    5. Renal impairment (serum creatinine ≥ 2 times the upper limit of normal [ULN])
    6. Aspartate transaminase (AST) and/or alanine transaminase (ALT) ≥ 3 times ULN
    7. 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 and for duration of study; single agent low dose aspirin [≤ 100 mg daily] is permitted)
    8. Severe deformity or ankle fixation that would sufficiently limit passive range of motion to affect functional assessments (TA subjects only)
    9. Major surgery within 4 weeks prior to Study Day 1
    10. Chronic pharmacologic doses of systemic corticosteroids (≥ 2 weeks) within 4 weeks before Study Day 1 and for duration of study; intra-articular/topical/inhaled/intranasal physiologic doses of systemic corticosteroids are permitted
    11. Androgens, growth hormone, insulin or oral hormone replacement therapy within 6 months before Study Day 1 and for duration of study; topical physiologic androgen replacement is permitted
    12. Any change in medications potentially affecting muscle strength or function within 4 weeks of Study Day 1 and for duration of study (e.g., creatinine, CoQ10, systemic beta adrenergic agonists)
    13. Previous exposure to any other investigational agent (not including ACE-083) potentially affecting muscle volume, muscle strength, or muscle or nerve function, within 5 half-lives of last dose plus an additional 8-week washout period (or 12 weeks prior to Study Day 1 if half-life is unknown)
    14. 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
    15. Any condition that would prevent the use of MRI or compromise the ability to obtain a clear and interpretable image of the treated muscles (e.g., knee/hip replacement metallic implants)
    16. Known active substance abuse, including alcohol
    17. History of sensitivity to protein pharmaceuticals
    18. Female that is pregnant or lactating/breast-feeding
    1.Neoplasia maligna activa o presente (p. ej., remisión de menos de 5 años de duración), a excepción de carcinoma basocelular, carcinoma cervicouterino in situ o ≤ 2 carcinomas de células escamosasde piel totalmente extirpados o tratados.
    2.Enfermedades concomitantes, como enfermedad cardiopulmonar sintomática, dolor ortopédico o neuropático importante u otros procesos que, en opinión del investigador, podrían limitar la capacidad del sujeto para completar las evaluaciones de la fuerza y/o funcionales.
    3.Diabetes mellitus de tipo 1 o 2.
    4.Trastorno tiroideo a menos que la situación se encuentre estable, sin modificaciones del tratamiento, durante al menos 4 semanas antes de la primera dosis y sin cambios previstos durante el estudio.
    5.Insuficiencia renal (creatinina sérica ≥ 2 veces el límite superior de la normalidad [LSN]).
    6.Aspartato transaminasa (AST) y/o alanina transaminasa (ALT) ≥ 3 veces el LSN.
    7.Riesgo elevado de hemorragia (p. ej., debido a hemofilia, trastornos plaquetarios o uso de cualquier tratamiento anticoagulante o modificador de las plaquetas hasta 2 semanas antes del día 1 del estudio y durante todo el estudio; se permite el uso de ácido acetilsalicílico en dosis bajas [≤ 100 mg/día] en monoterapia).
    8.Deformidad grave o fijación del tobillo que podría limitar la amplitud de movimiento pasivo suficientemente como para afectar a las evaluaciones funcionales (únicamente en los sujetos con evaluación del músculo TA).
    9.Cirugía mayor en las 4 semanas previas al día 1 del estudio.
    10.Dosis farmacológicas crónicas de corticoides sistémicos (≥ 2 semanas) en las 4 semanas previas al día 1 del estudio y durante todo el estudio; se permiten las dosis fisiológicas intraarticulares, tópicas, inhaladas o intranasales de corticoides sistémicos.
    11.Tratamiento de reposición de andrógenos, hormona del crecimiento, insulina u hormonas orales en los 6 meses previos al día 1 del estudio y durante todo el estudio; se permite la reposición fisiológica tópica de andrógenos.
    12.Cualquier modificación de medicamentos que pueda afectar potencialmente a la fuerza o función muscular en las 4 semanas previas al día 1 del estudio y durante todo el estudio (p. ej., creatinina, CoQ10, agonistas betaadrenérgicos sistémicos).
    13.Exposición previa a cualquier otro fármaco en investigación (sin incluir ACE-083) que pueda afectar potencialmente al volumen muscular, la fuerza muscular o la función muscular o nerviosa durante el período equivalente a 5 vidas mediasdesde la última dosis más un período de lavado adicional de 8 semanas (o 12 semanas antes del día 1 del estudio en caso de que se desconozca la vida media).
    14.Variación significativa de la actividad física o el ejercicio (p. ej., aumento o disminución significativo de la intensidad o frecuencia) en las 8 semanas previas al día 1 del estudio o incapacidad para mantener el nivel basal de actividad física durante todo el estudio.
    15.Cualquier situación que impida el uso de RM o dificulte la capacidad de obtener una imagen clara e interpretable de los músculos tratados (p. ej., implantes metálicos de remplazo de articulación de rodilla o cadera).
    16.Abuso activo conocido de sustancias, incluido el alcohol.
    17.Antecedentes de sensibilidad a proteínas farmacéuticas.
    18.Mujeres embarazadas o en período de lactancia.
    E.5 End points
    E.5.1Primary end point(s)
    • Presence and nature of adverse events (AE), including injection site reactions and changes in clinical laboratory parameters
    • Percent change from baseline in muscle volume of injected muscle by magnetic resonance imaging (MRI)
    -Presencia y naturaleza de los acontecimientos adversos (AA), incluidas reacciones en el lugar de inyección y variaciones de los parámetros analíticos de laboratorio.
    -Porcentaje de cambio con respecto al momento basal del volumen muscular del músculo inyectado mediante imagen de resonancia magnética (RM).
    E.5.1.1Timepoint(s) of evaluation of this end point
    • Safety assessments from Day 1 through Day 701/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.
    • MRI:
    - Loading phase: Day 1, 85
    - Maintenance and Follow up Phase: Day 1, 113, 169, 337, 505, 673
    -Las evaluaciones de seguridad desde el día 1 hasta el dia 701/EOS incluyen la monitorización de acontecimientos adversos (AAs), reacciones en el lugar de inyección, medicamentos concomitantes, análisis clínicos (hematología, bioquímica y AACF), análisis de orina, constantes vitales y hallazgos de la exploración física.
    RM:
    -Fase de carga: dia 1,85
    -Fase de mantenimiento y seguimiento: Dia 1,113,169,337,505,673
    E.5.2Secondary end point(s)
    • Percent and absolute change from baseline in the intramuscular fat fraction of injected muscle by MRI
    • Percent change from baseline in strength measurements
    • Percent and absolute change from baseline in functional assessments: For tibialis anterior (TA) muscle: 10-meter walk/run, 6-minute walk test, 4-stair climb (subjects from A083-02 only), and 100-meter timed test; for biceps brachii (BB) muscle: mid-level and high level performance of the upper limb (PUL) test
    • Absolute change from baseline in FSHD-health index (FSHD-HI, subjects from A083-02) or CMT health index (CMT-HI, subjects from A083-03) total score and subscale scores
    • PK parameters of ACE-083 serum concentrations over time
    •Porcentaje de cambio y absoluto con respecto al momento basal de la fracción de grasa intramuscular del músculo inyectado mediante RM.
    •Variación porcentual con respecto al momento basal de las mediciones de la fuerza.
    •Variación porcentual y absoluta con respecto al momento basal de las evaluaciones funcionales: en relación con el músculo tibial anterior (TA): prueba de marcha/carrera de 10 metros, prueba de marcha de 6 minutos, subida de 4 tramos de escalera (únicamente en los sujetos procedentes del estudio A083-02) y prueba cronometrada de 100 metros; en relación con el músculo bíceps braquial (BB): prueba de rendimiento de la extremidad superior (PUL) de nivel medio y alto.
    •Variación absoluta con respecto al momento basal de la puntuación total FSHD-HI (DFEHFSHD-índice de salud; sujetos procedentes del estudio A083-02) o CMT-HI (CMT-índice de salud; sujetos procedentes del estudio A083-03) y las puntuaciones de las subescalas.
    •Parámetros farmacocinéticos de las concentraciones séricas de ACE-083 a lo largo del tiempo.
    E.5.2.1Timepoint(s) of evaluation of this end point
    • MRI:
    - Loading phase: Day 1, 85
    - Maintenance and Follow up Phase: Day 1, 113, 169, 337, 505, 673
    • evaluate changes in strength, motor function, and quality of life (patient-reported outcomes) during the maintenance and loading phases of treatment.
    • Percent and absolute change from baseline in functional assessments:
    - Loading Phase: Screening, Day 1, Day 85 (for tibialis anterior (TA) muscle: 10-meter walk/run, 6-minute walk test, 4-stair climb (subjects from A083-02 only))
    - Maintenance Phase and Follow-Up: Day 1, 113, 169, 337, 505, 673, 701
    • FSHD-health index and CMT health index (CMT-HI):
    - Loading Phase: Screening, Day 1, 85
    - Maintenance Phase and Follow-Up: Day 1, 113, 169, 337, 505, 673, 701
    • PK samples:
    - Loading Phase: Day 1, 85
    RM:
    -Fase de carga: dia 1, 85
    -Fase de mantenimiento y seguimiento: Dia 1,113,169,337,505,673
    • evaluar variaciones fuerza,función motora y calidad vida (resultados comunicados por pacientes) durante fases de mantenimiento y carga del tratamiento.
    • Variación porcentual y absoluta respecto al momento basal de evaluaciones funcionales:
    -Fase carga: screening, dia 1, 85 (músculo tibial anterior (TA): prueba de marcha/carrera 10 metros, prueba marcha 6 minutos, subida 4 tramos de escalera (únicamente en sujetos procedentes del estudio A083-02))
    -Fase mantenimiento y seguimiento: Dia 1, 113,169,337,505,673,701
    • DFEH-índice salud y CMT-HI:
    -Fase carga: screening, dia 1, 85
    -Fase mantenimiento y seguimiento: Dia 1, 113,169,337,505,673,701
    • Muestras FC:
    -Fase carga: dia 1,5
    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 Yes
    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 Yes
    E.8.1.7.1Other trial design description
    La Parte 1 del estudio es no-aleatorizada, la Parte 2 es aleatorizada
    Part 1 of the study is non-randomized, Part 2 is randomized
    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 trial9
    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 concerned3
    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 ultimo paciente)
    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 months9
    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 months9
    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: 150
    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 state13
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 13
    F.4.2.2In the whole clinical trial 150
    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 finalización 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 Decision2019-05-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-04-12
    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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