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    Summary
    EudraCT Number:2018-000504-42
    Sponsor's Protocol Code Number:ABT-003
    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:2018-04-09
    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 number2018-000504-42
    A.3Full title of the trial
    A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients with Middle and Advanced Phases of MPS IIIA Disease
    Ensayo Fase I/II abierto, dosis única de transferencia génica de scAAV9.U1A.SGSH (ABO-102) en pacientes con enfermedad MPS IIIA media y avanzada
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Gene Therapy Clinical Trial for Mucopolysaccharidosis IIIA in patient with middle and advanced phases of the disease
    Ensayo Clínico de terapia génica para la Mucopolisacaridosis tipo IIIA en pacientes con enfermedad MPS IIIA media y avanzada
    A.3.2Name or abbreviated title of the trial where available
    ABO-102 for Treatment of Middle and Advanced Phases of MPS IIIA
    ABO-102 para el tratamiento de la enfermedad MPS IIIA media y avanzada
    A.4.1Sponsor's protocol code numberABT-003
    A.5.4Other Identifiers
    Name:Program nameNumber:ABO-102
    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 SponsorAbeona Therapeutics Inc
    B.1.3.4CountrySpain
    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 supportAbeona Therapeutics Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAbeona Therapeutics Inc
    B.5.2Functional name of contact pointHead of European Medical Affairs
    B.5.3 Address:
    B.5.3.1Street AddressAvenida de Manoteras 30, A207-208
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28050
    B.5.3.4CountrySpain
    B.5.4Telephone number+34689166070
    B.5.5Fax number+34911726254
    B.5.6E-mailinfotrials@abeonatherapeutics.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/3/16/1761
    D.3 Description of the IMP
    D.3.1Product namescAAV9.U1A.SGSH
    D.3.2Product code ABO-102
    D.3.4Pharmaceutical form Suspension for injection
    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 INNscAAV9.U1A.SGSH
    D.3.9.2Current sponsor codescAAV9.U1A.SGSH
    D.3.9.3Other descriptive namescAAV9.U1A.SGSH
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30000000000000
    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 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 Yes
    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
    MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, with
    deterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatment
    currently available for the disease.
    MPS IIIA es una enfermedad de depósito lisosomal, causada por un defecto genético de la enzima N-sulfoglucosamina sulfohidrolasa. Los niños parecen normales al nacer, pero la enfermedad es progresiva, con
    el deterioro de las habilidades sociales y de adaptación, la disminución neurocognitiva y la muerte prematura. La muerte se produce normalmente a finales de la segunda o principios de la tercera década. Es de destacar que no existe un tratamiento disponible actualmente para la enfermedad.
    E.1.1.1Medical condition in easily understood language
    Mucopolysaccharidosis type IIIA is a genetic disease in children, caused by the toxicity of an accumulation of substances in the body that generate a progressive deterioration.
    La mucopolisacaridosis tipo IIIA es una enfermedad genética en niños, causada por la toxicidad de un acúmulo de sustancias en el organismo que generan un deterioro progresivo.
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10056890
    E.1.2Term Mucopolysaccharidosis 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
    Primary outcomes of safety and efficacy assessed by measuring biochemical and biophysical changes in middle and advanced phases of MPS IIIA evolution
    Objetivos principales de seguridad y eficacia evaluado a través de los cambios bioquímicos y biofísicos en la fase media y avanzada de la evolución de la enfermedad MPS IIIA
    E.2.2Secondary objectives of the trial
    Change from baseline:
    • in plasma or urine GAG or HS after treatment (AT)
    • in CSF or plasma or leukocyte SGSH enzyme activity levels AT
    • in brain volumes AT
    • in the Developmental Age AT compared to Natural History Study (NHS)
    • in the Cognitive Developmental Age AT compared to NHS
    • in the Adaptive Age Equivalent score AT compared to NHS
    • in the Sanfilippo Behavior Rating Scale
    • in sleep pattern
    • in Pediatric Quality of Life Inventory Core Generic Scales total
    • in parent quality of life
    • Change from baseline in gastrointestinal symptoms
    • in Parent Global Impression Score
    • in Clinical Global Impression Improvement Scale
    • in Parent Symptoms Score Questionnaire
    • in Body Mass Index AT
    • and incidence in abnormalities in standard awake 60-minutes- EEG monitoring
    Preliminary data for the Environmental Risk Assessment
    Cambio desde la visita de inicio:
    • en los niveles de GAGs o HS en orina y plasma después del tratamiento (DdT)
    • en los niveles de actividad enzimática SGSH en plasma, LCR o leucocitos DdT
    • de los volúmenes del cerebro DdT
    • en la edad de desarrollo DdT respecto al estudio de historia natural (EHN)
    • en la edad de desarrollo cognitivo DdT respecto al EHN
    • en la puntuación equivalente adaptativa DdT respecto al EHN
    • en la escala de Sanfilippo
    • en el patrón de sueño de los niños
    • en la puntuación total del cuestionario de Calidad de Vida
    • en la calidad de vida de los padres
    • en los síntomas gastrointestinales
    • en la puntuación de la escala de impresión global de los padres
    • en la puntuación de la escala de impresión clínica global
    • en el cuestionario de puntuación de los síntomas de los padres
    • en el índice de masa corporal DdT
    • e Incidencia en las anormalidades en el estado estándar de vigilia de 60 minutos
    Datos preliminares para la ERA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Diagnosis of MPS IIIA confirmed by the following methods:
    o No detectable or significantly reduced SGSH enzyme activity by leukocyte assay, and
    o Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene
    • Cognitive Development Quotient (DQ) lower than 60 (calculated by Bayley Scales of Infant and Toddler Development - Third Edition)
    • Must be ambulatory, though may receive assistance with ambulation
    • Diagnóstico confirmado de MPS IIIA por los dos métodos siguientes:
    o No se detecta o significativamente reducida actividad de la enzima SGSH en el análisis de leucocitos, y
    o Análisis genómico de la mutación del DNA que demuestre las mutaciones heterozigóticas compuestas u homozigóticas del gen SGSH.
    • Cociente de desarrollo cognitivo (DQ) inferior a 60 (calculado por la escala de Bayley para el desarrollo del bebe y niño pequeño - Tercera Edición)
    • Debe caminar, aunque puede que sea necesario recibir asistencia
    E.4Principal exclusion criteria
    • Inability to participate in the clinical evaluation as determined by Principal Investigator
    • Identification of two nonsense or null variants on genetic testing of the SGSH gene
    • At least one S298P mutation in the SGSH gene
    • Has evidence of an attenuated phenotype of MPS IIIA
    • Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics
    • Active viral infection based on clinical observations
    • Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer, or precludes the child from participating in the protocol assessments and follow up
    • Participants with total anti-AAV9 antibody titers ≥ 1:100 as determined by ELISA binding immunoassay
    • Participants with a positive response for the ELISPOT for T-cell responses to AAV9
    • Serology consistent with exposure to HIV, or serology consistent with active hepatitis B or C infection
    • Bleeding disorder or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated according to local institutional policy
    • Visual or hearing impairment sufficient to preclude cooperation with neurodevelopmental testing
    • Any item (braces, etc.) which would exclude the participant from being able to undergo MRI according to local institutional policy
    • Any other situation that precludes the participant from undergoing procedures required in this study
    • Participants with cardiomyopathy or significant congenital heart abnormalities
    • The presence of significant non-MPS IIIA related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study
    • Abnormal laboratory values Grade 2 or higher as defined in CTCAE v4.0 for GGT, total bilirubin (except in subjects diagnosed with Gilbert’s syndrome), creatinine, hemoglobin, WBC count, platelet count, PT and aPTT
    • Female participant who is pregnant or demonstrates a positive urine or beta-hCG result at screening assessment (if applicable)
    • Any vaccination with viral attenuated vaccines less than 30 days prior to the scheduled date of treatment (and use of prednisolone)
    • Previous treatment by Haematopoietic Stem Cell transplantation
    • Previous participation in a gene/cell therapy or ERT clinical trial
    • Participants who are anticipated to undergo a procedure involving anesthesia within 6 months post- drug administration
    • Dysphagia present at Grade 3 or higher, as defined in CTCAE v4.0
    • Incapacidad para participar en la evaluación clínica, según criterio del Investigador Principal
    • Identificación de dos variantes nulas o sin sentido en el test genético del gen SGSH.
    • Al menos una mutación S298P en el gen SGSH
    • Tiene evidencia de un fenotipo atenuado de MPS IIIA
    • Presencia de una condición médica concomitante que impida la punción lumbar o el uso de anestesia
    • Infección viral activa basada en observaciones clínicas
    • Enfermedad concomitante o necesidad de tratamiento médico crónico que según opinión del Investigador Principal (IP) cree riesgos innecesarios para la transferencia génica o impide que el niño participe en las evaluaciones del protocolo y en el seguimiento
    • Participantes con niveles de anticuerpos totales anti-AAV9 ≥ 1:100 según lo determinado por el inmunoensayo tipo ELISA
    • Participantes con una respuesta positiva de ELISPOT para la respuesta de células T al AAV9
    • Serología acorde con exposición a VIH, o serología consistente con infección activa de hepatitis B o C
    • Trastorno hemorrágico o cualquier otra afección médica o circunstancia en la que esté contraindicada una punción lumbar (para la obtención de líquido cefalorraquídeo (LCR)) conforme a la política institucional local
    • Deterioro visual o auditivo suficiente para impedir la cooperación en las pruebas de neurodesarrollo
    • Cualquier objeto (aparato, etc) que excluya al participante la posibilidad de someterse a una resonancia magnética conforme a la política institucional local
    • Cualquier otra situación que impida al participante someterse a los procedimientos requeridos en este estudio
    • Participantes con cardiomiopatía o cardiopatías congénitas significantes
    • La presencia de deterioro importante del sistema nervioso central (SNC) no relacionado con MPS IIIA o alteraciones de la conducta que confundan el rigor científico o la interpretación de los resultados del estudio
    • Valores analíticos alterados de grado 2 o mayores según se define en CTCAE v4.0 para GGT, bilirrubina total (excepto en sujetos diagnosticados con el síndrome de Gilbert), creatinina, hemoglobina, recuento de glóbulos blancos, recuento de plaquetas, tiempo de protrombina y tiempo de tromboplastina parcial
    • Participante femenina que está embarazada o muestre beta-HCG positivo en orina en la prueba de evaluación (si aplica)
    • Cualquier vacunación con vacunas virales atenuadas administrada a menos de 30 días antes de la fecha programada de tratamiento (y el uso de prednisolona)
    • Tratamiento previo mediante trasplante de células madre hematopoyéticas
    • Participación previa en una terapia génica/celular o en un ensayo clínico de ERT
    • Participantes que se espera que se sometan a un procedimiento que involucre anestesia dentro de los 6 meses posteriores a la administración del tratamiento
    • Disfagia presente en Grado 3 o superior, según se define en CTCAE v4.0
    E.5 End points
    E.5.1Primary end point(s)
    • Product safety as defined by the incidence, type and severity of treatment-related adverse events and serious adverse events
    • Change from baseline in CSF heparan sulfate levels after treatment
    • Change from baseline in liver and/or spleen volumes after treatment, as measured by magnetic resonance imaging (MRI)
    • Seguridad del producto según se define por la incidencia, tipo y severidad de los eventos adversos relacionados con el tratamiento y de los eventos adversos graves
    • Cambio desde la visita de inicio los niveles de heparan sulfato en LCR después del tratamiento
    • Cambio desde la visita de inicio de los volúmenes del hígado y/o bazo después del tratamiento, medido por resonancia magnética (RMN)
    E.5.1.1Timepoint(s) of evaluation of this end point
    - Month 1, 2, 3, 6, 12, 18, 24
    - Month 1, 6, 12, 24
    - Month 1, 6, 12, 24
    - Mes 1, 2, 3, 6, 12, 18, 24
    - Mes 1, 6, 12, 24
    - Mes 1, 6, 12, 24
    E.5.2Secondary end point(s)
    • Change from baseline in plasma or urine glycocaminoglycans or heparan sulfate after treatment
    • Change from baseline in CSF or plasma or leukocyte SGSH enzyme activity levels after treatment
    • Change from baseline in brain volumes after treatment, as measured by MRI
    • Change from baseline in the Developmental Age after treatment compared to Natural History Study data calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children; Second Edition, based on chronological and developmental age
    • Change from baseline in the Cognitive Developmental Age after treatment compared to Natural History Study, calculated using the Bayley Scales of Infant and Toddler Development – Third edition or the Kaufman Assessment Battery for Children. Second Edition, based on developmental age
    • Change from baseline in the Adaptive Age Equivalent score after treatment compared to Natural History Study data, as assessed by parent report using the Vineland Adaptive Behavior Scale II Survey form
    • Change from baseline in the Sanfilippo Behavior Rating Scale
    • Change from baseline in sleep pattern as measured by the modified Children’s Sleep Habits Questionnaire (CSHQ)
    • Change from baseline in Pediatric Quality of Life Inventory (PedsQL™) Core Generic Scales total score
    • Change from baseline in parent quality of life, using the Parenting Stress Index, 4th Edition (PSI-4)
    • Change from baseline in gastrointestinal symptoms using the PedsQL™ Gastrointestinal Symptoms Scales
    • Change from baseline in Parent Global Impression Score • Change from baseline in Clinical Global Impression Improvement Scale
    • Change from baseline in Parent Symptoms Score Questionnaire
    • Change from Baseline in Body Mass Index after treatment
    • Incidence and Change from baseline in abnormalities in standard awake 60-minutes- EEG monitoring
    • Determination of vector shedding analysis in plasma, saliva, urine and feces will provide preliminary data for the Environmental Risk Assessment (ERA)

    Explorative endpoints
    • Change from baseline in brain MRI using DTI technology, compared to changes observed in Natural History Study data
    • Change from baseline in plasma and CSF cytokines
    • Change from baseline in CSF Gangliosides (GM2-GM3)
    • Change from baseline in audiometry evaluations assessed by tympanometry, otoacoustic emissions (OAE) and auditory brainstem response (ABR)
    • Determination of neutralizing antibodies titers against AAV9 as determined by an in vitro infectivity assay
    • Cambio desde la visita de inicio en los niveles de glicocaminoglicanos o heparan sulfato en orina y plasma después del tratamiento
    • Cambio desde la visita de inicio en los niveles de actividad enzimática SGSH en plasma, LCR o leucocitos después del tratamiento
    • Cambio desde la visita de inicio de los volúmenes del cerebro después del tratamiento, medido por imágenes de resonancia magnética (RMN)
    • Cambio desde la visita de inicio en la Edad de Desarrollo después del tratamiento en comparación con los datos del Estudio de Historia Natural calculados por las Escalas Mullen de Aprendizaje Temprano o la Batería de Evaluación Kaufman para niños; Segunda Edición, basada en la edad cronológica y de desarrollo
    • Cambio desde la visita de inicio en la Edad de Desarrollo Cognitivo después del tratamiento en comparación con el Estudio de Historia Natural, calculado usando las Escalas Bayley de Desarrollo de Bebés y Niños Pequeños - Tercera edición o la Evaluación Kaufman para Niños. Segunda edición, basada en la edad de desarrollo
    • Cambio desde la visita de inicio en la puntuación equivalente adaptativa después del tratamiento en comparación con los datos del Estudio de Historia Natural, según la evaluación de los padres utilizando la Encuesta de la Escala Vineland de Conducta Adaptativa II
    • Cambio desde la visita de inicio en la Escala de valoración conductual de comportamiento de Sanfilippo
    • Cambio desde la visita de inicio en el patrón de sueño medido por el Cuestionario de Hábitos de Sueño de los Niños (CSHQ)
    • Cambio desde la visita de inicio en la puntuación total del cuestionario de Calidad de Vida Pediátrica (PedsQL™), puntuación total de las escalas genéricas básicas
    • Cambio desde la visita de inicio en el cuestionario de calidad de vida de los padres, utilizando la escala de Índice de Estrés de los Padres Cuarta Edición (PSI-4)
    • Cambio desde la visita de inicio en los síntomas gastrointestinales usando PedsQL™, escala de Síntomas Gastrointestinales
    • Cambio desde la visita de inicio la puntuación de la escala de impresión global de los padres
    • Cambio desde la visita de inicio la puntuación de la escala de impresión clínica global
    • Cambio desde la visita de inicio en el Cuestionario de puntuación de los síntomas de los padres
    • Cambio desde la visita de inicio en el Índice de Masa Corporal después del tratamiento
    • Incidencia y cambio desde la visita de inicio en las anormalidades en el estado estándar de vigilia de 60 minutos – monitorizado mediante electroencefalograma
    • Determinación de excreción de vector en plasma, saliva, orina y heces, proporcionará datos preliminares para la Evaluación de Riesgo Ambiental (ERA)

    Variables exploratorias
    • Cambio desde la visita de inicio en la resonancia magnética cerebral utilizando tecnología DTI, comparando con los cambios observados en los datos del Estudio de Historia Natural
    • Cambio desde la visita de inicio en los niveles de citoquinas en plasma y LCR
    • Cambio desde la visita de inicio en los niveles de gangliósidos en LCR [GM2-GM3]
    • Cambio desde la visita de inicio las evaluaciones de audiometría, evaluadas mediante una timpanometría, emisiones otoacústicas (EOA) y respuesta auditiva del tronco cerebral (BAER)
    • Determinación de anticuerpos neutralizantes contra AAV9 según lo determinado de un ensayo de infectividad in vitro
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Month 1, 6, 12,18, 24
    - Month 1, 6, 12, 24
    - Month 1, 6, 12, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 6, 12, 18, 24
    - Month 1 and then monthly until two consecutive samples are negative

    Exploratory endpoints
    - Month 1, 6, 12, 24
    - Month 1, 6, 12, 24
    - Month 1, 6, 12, 24
    - Month 6, 12, 24
    - Month 6, 12, 24
    - Mes 1, 6, 12,18, 24
    - Mes 1, 6, 12, 24
    - Mes 1, 6, 12, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 6, 12, 18, 24
    - Mes 1 y después mensualmente hasta que se obtengan dos muestras negativas

    Variables exploratorias
    - Mes 1, 6, 12, 24
    - Mes 1, 6, 12, 24
    - Mes 1, 6, 12, 24
    - Mes 6, 12, 24
    - Mes 6, 12, 24
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Study in another population with respect to ABT-001
    Estudio en una población diferente al ABT-001
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 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 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
    Australia
    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
    Ultima visita del ultima 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 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 Yes
    F.1.1Number of subjects for this age range: 12
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception 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
    These children have severe cognitive problems making them very difficult to understand an explanation about the study and also to sign the informed consent. Based on a expert opinion, unique ICF for parents or legal representatives is prepared.
    Estos niños tienen problemas cognitivos severos que dificultan su entendimiento de una explicación del estudio y firma del consentimiento informado.Basado en una opinión experta, se ha preparado un único ICF para los padres o representantes legales.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 12
    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 the 24-month visit, participants will be invited to participate in a long term follow up study consisting of annual monitoring for a period of five years. If they do not consent to participate in the new study, PI will collect annual medical records according to the guidelines provided in this protocol
    Después de la visita del mes 24, los sujetos serán invitados a participar en un estudio de seguimiento a largo plazo con visitas anuales de monitorización anual por un periodo de 5 años. Si ellos no aceptan participar en el nuevo estudio, el Investigador Principal recogerá datos médicos de acuerdo a las guías proporcionadas en este protocolo
    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 Decision2019-05-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-05-06
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2022-02-08
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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