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    Summary
    EudraCT Number:2022-001991-32
    Sponsor's Protocol Code Number:BN42489
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-12-23
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2022-001991-32
    A.3Full title of the trial
    A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, DOSE-FINDING STUDY TO EVALUATE THE SAFETY, BIOMARKERS, AND EFFICACY OF TOMINERSEN IN INDIVIDUALS WITH PRODROMAL AND EARLY MANIFEST HUNTINGTON’S DISEASE
    ESTUDIO DE FASE II, ALEATORIZADO, DOBLE CIEGO, CONTROLADO CON PLACEBO Y DE BÚSQUEDA DE DOSIS PARA EVALUAR LA SEGURIDAD, LOS BIOMARCADORES Y LA EFICACIA DE TOMINERSEN EN PACIENTES CON ENFERMEDAD DE HUNTINGTON PRODRÓMICA Y DE APARICIÓN PRECOZ
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals with Prodromal and Early Manifest Huntington’s Disease
    Estudio para evaluar la seguridad, los biomarcadores y la eficacia de tominersen en pacientes con enfermedad de Huntington prodrómica y de aparición precoz
    A.4.1Sponsor's protocol code numberBN42489
    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 SponsorRoche Farma S. A. U. que realiza el ensayo en España y que actúa como representante F. Hoffmann-La Roche Ltd
    B.1.3.4CountrySwitzerland
    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 supportF. Hoffmann-La Roche Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationF.Hoffmann-La Roche Ltd
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+34913257300
    B.5.5Fax number+34913248196
    B.5.6E-mailspain.start_up_unit@roche.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/3/15/1453
    D.3 Description of the IMP
    D.3.2Product code RO7234292
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTominersen
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codeRO7234292
    D.3.9.3Other descriptive nameN/A
    D.3.9.4EV Substance CodeSUB198185
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number6
    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 Yes
    D.3.11.13.1Other medicinal product typemodified antisense oligonucleotide
    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 placeboIntrathecal use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Huntington's disease (HD)
    Enfermedad de Huntington (EH)
    E.1.1.1Medical condition in easily understood language
    HD, is a genetically inherited disorder that causes uncontrolled movements (chorea), emotional problems, and the loss of thinking ability, eventually resulting in death
    EH, es un trastorno genético heredado que provoca movimientos incontrolados (corea), problemas emocionales y la pérdida de la capacidad de pensar, lo que acaba provocando la muerte
    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 PT
    E.1.2Classification code 10070668
    E.1.2Term Huntington's disease
    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 safety of tominersen compared with placebo on the basis of incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale, change from baseline in clinical laboratory results [cerebrospinal fluid (CSF) white blood cell (WBC) and protein], and safety magnetic resonance imaging (MRI)
    - To evaluate CSF mutant huntingtin (mHTT) protein levels in response to tominersen compared with placebo at 9 months
    - To evaluate the efficacy of tominersen compared with placebo on the basis of change from baseline in composite unified huntington's disease rating scale (cUHDRS) (non-U.S.) and total functional capacity (TFC) (U.S.) at 16 months
    - Evaluar la seguridad de tominersen en comparación con el placebo basándose en la iIncidencia e intensidad de los acontecimientos adversos, con determinación de la intensidad según la Escala de gradación de la intensidad de los acontecimientos adversos, variación de los resultados de análisis clínicos con respecto al momento basal (leucocitos y proteína en líquido cefalorraquídeo (LCR)) y Resonancia magnética (RM) de seguridad.
    - Evaluar las concentraciones de proteína huntingtina mutante (mHTT) en el LCR en respuesta a tominersen en comparación con el placebo en el mes 9
    - Evaluar la eficacia de tominersen en comparación con el placebo basándose en la variación en la cUHDRS (fuera de EE. UU.) y la escala TFC (en EE. UU.) con respecto al momento basal a los
    16 meses
    E.2.2Secondary objectives of the trial
    - To evaluate the safety of tominersen compared with placebo on the basis of change from baseline in vital signs, electrocardiogram (ECG) parameters, plasma clinical laboratory results, montreal cognitive assessment (MoCA), and proportion of participants with suicidal ideation or behavior as assessed by columbia-suicide severity rating scale (C-SSRS) score at each visit
    -To evaluate the efficacy of tominersen compared with placebo on the basis of change from baseline at 16 months for the assessments of TFC (non-U.S.)/cUHDRS (U.S.), symbol digit modalities test (SDMT) stroop word reading (SWR) and total motor score (TMS)
    - To evaluate change from baseline in CSF neurofilament light chain (NfL) in response to tominersen compared with placebo at 16 months
    - To evaluate the immune response to tominersen
    - Evaluar la seguridad de tominersen en comparación con el placebo en base a la variación con respecto al momento basal de las constantes vitale, variación de los parámetros del electrocardiograma (ECG), variación de los resultados de análisis clínicos en plasma, variación de la MoCA y proporción de participantes con ideas o comportamientos suicidas, según la evaluación de la puntuación en la C-SSRS en cada visita
    - Evaluar la eficacia de tominersen en comparación con el placebo en base a la variación respecto al momento basal a los 16 meses en las evaluaciones: TFC (fuera de EE. UU.) /cUHDRS (en EE. UU.), Symbol Digit Modalities Test (prueba de modalidades de símbolos y dígitos –SDMT - TMF) y Stroop Word Reading (lectura de palabras de Stroop –SWR).
    - Evaluar las concentraciones de cadena ligera de neurofilamentos (NfL) en el LCR en respuesta a tominersen en comparación con el placebo a los 16 meses
    - Evaluar la respuesta inmunitaria a tominersen
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Age 25-50 years
    - Huntington’s disease (HD) gene expansion mutation carrier status
    - Prodromal HD
    - Estimated glomerular filtration rate >= 60 mL/min/1.73 m2 in at least one out of two maximum screening samples
    - Total body weight > 40 kg and a body mass index within the range of 18-32 kg/m2
    - Ability to read the words "red," "blue," and "green" in native language
    - Ability to walk unassisted without a cane or walker and move about without a wheelchair on a daily basis as determined at screening and baseline visit (Day 1)
    - Ability to undergo and tolerate MRI scans
    - Ability to tolerate blood draws and lumbar punctures (LPs)
    - Stable medical, psychiatric, and neurologic status as assessed by physician for at least 12 weeks prior to screening and at the time of enrollment
    - Negative hepatitis B surface antigen test at screening
    - Negative hepatitis C virus (HCV) antibody test at screening, or a positive HCV antibody test followed by a negative HCV ribonucleic acid (RNA) test at screening
    - For female participants of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception and agree to refrain from donating eggs during the treatment period and for 5 months after the final dose of the study drug. Female participants must refrain from donating eggs during this same period
    - For male participants: agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom and agree to refrain from donating sperm during the treatment period and for 5 months after the final dose of the study drug to avoid exposing the embryo. Male participants must refrain from donating sperm during this same period
    - Edad de 25-50 años
    - Portador de mutación de expansión del gen HD (enfermedad de Huntington)
    - EH prodrómica
    - Tasa de filtración glomerular estimada ≥60 ml/min/1,73 m2 en al menos una de dos muestras de selección máximas
    - Peso corporal total >40 kg e índice de masa corporal en el intervalo de 18 32 kg/m2
    - Capacidad para leer las palabras «rojo», «azul» y «verde» en el idioma nativo
    - Capacidad para caminar sin ayuda de bastón ni andador y moverse sin silla de ruedas a diario, según lo determinado en las visitas de selección y basal (día 1)
    - Capacidad de someterse a las RM y tolerarlas
    - Capacidad para tolerar las extracciones de sangre y las punciones lumbares (PL)
    - Estado médico, psiquiátrico y neurológico estable según la evaluación del médico durante al menos 12 semanas antes de la selección y en el momento de la inclusión
    - Resultado negativo en el análisis del antígeno de superficie del virus de la hepatitis B en el período de selección
    - Resultado negativo en el análisis de anticuerpos contra el virus de la hepatitis C (VHC) en la selección, o resultado positivo en el análisis de anticuerpos contra el VHC seguido de un resultado negativo en un análisis de ARN del VHC en la selección
    - Mujeres participantes en edad fértil: compromiso de practicar la abstinencia (abstenerse de mantener relaciones heterosexuales) o de utilizar métodos anticonceptivos y compromiso de no donar óvulos durante el período de tratamiento y durante al menos 5 meses después de la última dosis del fármaco del estudio. Las mujeres participantes deberán abstenerse de donar óvulos durante ese mismo período.
    - Varones participantes: compromiso de practicar abstinencia sexual (abstenerse de mantener relaciones heterosexuales) o utilizar preservativo y de no donar semen durante el período de tratamiento y durante al menos 5 meses después de la última dosis del fármaco del estudio para no exponer al embrión. Los varones participantes deberán abstenerse de donar semen durante ese mismo período
    E.4Principal exclusion criteria
    - History of attempted suicide or suicidal ideation with plan that required hospital visit and/or change in level of care within 12 months prior to screening
    - Active psychosis, confusional state, or violent behavior, including aggression that could cause harm to self or others, over the 12 weeks prior to screening
    - Clinical diagnosis of chronic migraines as per the International Headache Society Classification of Headache Disorders
    - Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 5 months after the final dose of study drug
    - Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter
    - Hydrocephalus
    - History of deep brain stimulation
    - Known human immunodeficiency viruses (HIV) infection
    - An infection requiring oral or intravenous antibiotics within 14 days prior to screening, during screening and prior to randomization
    - Anti-retroviral medications, including anti-retroviral medication taken as prophylaxis within 12 months of study enrollment
    - Current or previous use of an antisense oligonucleotide (ASO) or any huntingtin protein (HTT) lowering therapy
    - Treatment with investigational therapy within 4 weeks or 5 drug-elimination half-lives, whichever is longer, prior to screening
    - Current or previous history of a primary independent psychotic disorder
    - Current use of anti-psychotics for motor symptoms or mood stabilization and/or tetrabenazine, valbenazine, or deutetrabenazine at a dose that has not been stable for at least 12 weeks prior to screening or is anticipated to change between screening and initiation of study treatment
    - Cholinesterase inhibitors, memantine, amantadine, or riluzole use within 12 weeks from initiation of study treatment
    - Current use of an anti-depressant or benzodiazepine at a dose that has not been stable for at least 12 weeks prior to screening or is anticipated to change between screening and initiation of study treatment
    - Current use of supplements used to treat HD symptoms, including in an experimental setting, at a dose that has not been stable for at least 6 weeks prior to screening or is anticipated to change during the study
    - Anti-platelet or anticoagulant therapy within 14 days prior to screening
    - History of bleeding diathesis or coagulopathy
    - Platelet count less than the lower limit of normal
    - History of gene therapy, cell transplantation, or brain surgery
    - Planned brain surgery during the study
    - Concurrent or planned participation in any interventional clinical study, including explicit pharmacological and non-pharmacological interventions. Observational studies are acceptable; however, data collection is not permitted between screening and baseline visits or within the 8 weeks before a study clinic visit
    - Poor peripheral venous access
    - Scoliosis or spinal deformity or surgery making intrathecal (IT) injection not feasible in an outpatient setting and potentially interfering with distribution of tominersen up the neuraxis
    - Malignancy within 5 years prior to screening, except basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated
    - Preexisting intra-axial or extra-axial lesions as assessed by a centrally read MRI scan during the screening period
    - Antecedentes de intento de suicidio o ideas suicidas con un plan que hayan requerido una visita al hospital y/o un cambio en el nivel de atención médica en los 12 meses previos a la selección
    - Psicosis activa, estado de confusión o conducta violenta, incluidas agresiones que puedan causarse daños a sí mismos o a otras personas, en las 12 semanas previas a la selección
    - Diagnóstico clínico de migrañas crónicas según la Clasificación de las cefaleas de la International Headache Society
    - Embarazo o lactancia, o intención de quedarse embarazada durante el estudio o en los 5 meses siguientes a la última dosis del fármaco del estudio
    - Presencia de una derivación implantada para el drenaje de LCR o de un catéter del sistema nervioso central (SNC) implantado
    - Hidrocefalia
    - Antecedentes de estimulación cerebral profunda
    - Infección confirmada por el VIH
    - Infección que exija la administración de antibióticos por vía oral o i.v. en los 14 días previos a la selección, durante el periodo de selección y antes de la aleatorización
    - Medicamentos antirretrovirales, incluido el tratamiento antirretroviral recibido como profilaxis en los 12 meses previos a la inclusión en el estudio
    - Uso actual o previo de un oligonucleótido antisentido (OAS) o cualquier tratamiento reductor de la HTT (incluido tominersen)
    - Tratamiento en investigación en las 4 semanas o en las 5 semividas de eliminación del medicamento antes de la selección, lo que sea más largo
    - Presencia o antecedentes de un trastorno psicótico primario independiente
    -Uso actual de antipsicóticos para síntomas motores o regulación del estado de y/o tetrabenazina, valbenazina o deutetrabenazina en una dosis que no se haya mantenido estable durante al menos 12 semanas antes de la selección o que se prevea un cambio entre la selección y el inicio del tratamiento del estudio
    - Uso de inhibidores de la colinesterasa, memantina, amantadina o riluzol en las 12 semanas previas al inicio del tratamiento del estudio
    - Uso actual de un antidepresivo o benzodiacepina en una dosis que no se haya mantenido estable durante al menos 12 semanas antes de la selección o que se prevea un cambio entre la selección y el inicio del tratamiento del estudio
    - Uso actual de suplementos para tratar los síntomas de la EH, incluso en un contexto experimental, en una dosis que no se haya mantenido estable durante al menos 6 semanas antes de la selección o que se prevea un cambio durante el estudio- Anti-platelet or anticoagulant therapy within 14 days prior to screening
    - Antecedentes de diátesis hemorrágica o coagulopatía
    - Recuento de plaquetas por debajo del límite inferior de la normalidad
    - Antecedentes de terapia génica, trasplante de células o intervención quirúrgica cerebral
    - Intervención quirúrgica cerebral prevista durante el estudio
    - Participación simultánea o prevista en cualquier estudio clínico intervencionista, incluidas intervenciones farmacológicas y no farmacológicas explícitas. Son aceptables los estudios observacionales (p. ej., estudio prospectivo ENROLL-HD), pero no se permite la recopilación de datos entre las visitas de selección y basal ni en las 8 semanas previas a una visita al centro del estudio
    - Acceso venoso periférico deficiente
    - Escoliosis o deformidad o cirugía de la columna que haga la inyección intratecal (i.t.) inviable en el contexto ambulatorio y que podría interferir con la distribución de tominersen en el tubo neural
    - Neoplasia maligna en los 5 años previos a la selección, excepto carcinoma basocelular o espinocelular de piel o carcinoma in situ de cuello uterino tratado con éxito
    - Lesiones intra o extraaxiales preexistentes según la evaluación realizada por una exploración de RM interpretada centralmente durante el período de selección
    E.5 End points
    E.5.1Primary end point(s)
    1. Incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale
    2. Change from baseline in clinical laboratory results (CSF WBC and protein)
    3. Safety MRI
    4. Percentage change from baseline in geometric means of CSF mHTT protein levels at Month 9
    5. Change from baseline in cUHDRS (non-U.S.) and TFC (U.S.) at 16 months
    1. Incidencia e intensidad de los acontecimientos adversos, con determinación de la intensidad según la Escala de gradación de la intensidad de los acontecimientos adversos
    2. Variación de los resultados de análisis clínicos con respecto al momento basal (leucocitos y proteína en LCR)
    3. RM de seguridad
    4. Variación porcentual de las medias geométricas de las concentraciones de proteína mHTT en el LCR con respecto al momento basal en el mes 9
    5. Variación en la cUHDRS (fuera de EE. UU.) y la escala TFC (en EE. UU.) con respecto al momento basal a los 16 meses
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Up to approximately 24 months
    2. From baseline visit (Day 1), and Months 4, 8, 9, 12, 16
    3. At Months 4, 8, 12, 16, early termination of treatment (ETT) and safety follow-up (SFU)
    4. Baseline and Month 9
    5. Baseline and 16 Months
    1. Hasta los 24 meses aproximadamente
    2. Desde la visita basal (día 1) y los meses 4, 8, 9, 12, 16
    3. En los meses 4, 8, 12, 16, visita de finalización de tratamiento prematura (ETT) y visitas de seguimiento de la seguridad (SFU)
    4. Momento basal y mes 9
    5. Momento basal y mes 16
    E.5.2Secondary end point(s)
    1. Change from baseline in vital signs
    2. Change from baseline in ECG parameters
    3. Change from baseline in plasma clinical laboratory results
    4. Change from baseline in MoCA
    5. Proportion of participants with suicidal ideation or behavior as assessed by C-SSRS score at each visit, including detailed focus on any individual cases identified as having severe ideation or behavior during the study conduct
    6. Change from baseline at 16 months for the assessments of TFC (non-U.S.)/cUHDRS (U.S.), SDMT, and SWR
    7. Change from baseline in CSF NfL levels at 16 months
    8. Incidence of anti-drug antibodies (ADAs) at specified timepoints relative to the prevalence of ADAs at baseline
    9. Titers will be determined if ADAs are identified
    1. Variación de las constantes vitales con respecto al momento basal
    2. Variación de los parámetros del ECG con respecto al momento basal
    3. Variación con respecto al momento basal de los resultados de análisis clínicos en plasma
    4. Variación de la MoCA con respecto al momento basal
    5. Proporción de participantes con ideas o comportamientos suicidas, según la evaluación de la puntuación en la C-SSRS en cada visita, con especial atención a todos los casos individuales en los que se identifique ideación o comportamiento grave durante la
    realización del estudio
    6. Variación respecto al momento basal a los 16 meses en TFC (fuera de EE. UU.) /cUHDRS (en EE. UU.), SDMT y SWR
    7. Variación de las concentraciones de NfL en LCR respecto al momento basal a los 16 meses
    8. Incidencia de ACF en puntos temporales especificados con respecto a su prevalencia en el momento basal
    9. Los títulos se determinarán si se identifican ACF
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-3. Baseline visit (Day 1), and Months 4, 8, 9, 12, 16, ETT and SFU
    4. Baseline and Months 4, 8, 12, 16, ETT and SFU
    5. Up to approximately 24 months
    6-7. Baseline to 16 Months
    8-9. Baseline, and Months 4, 8, 12, 16, ETT and SFU
    1-3. Visita basal (día 1), los meses 4, 8, 9, 12, 16, ETT y SFU
    4. Visita basal, los meses 4, 8, 9, 12, 16, ETT y SFU
    5. Hasta aproximadamente 24 meses
    6-7. Momento basal hasta los 16 meses
    8-9. Visita basal , los meses 4, 8, 9, 12, 16, ETT y SFU
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    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 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) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA35
    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
    Canada
    Japan
    New Zealand
    United Kingdom
    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 ultimo paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    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: 360
    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 No
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2022-12-23. Yes
    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 state51
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 200
    F.4.2.2In the whole clinical trial 360
    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)
    The Sponsor will offer continued access to Roche IMP (RO7234292) free of charge to eligible patients in accordance with the Roche Global Policy on Continued Access to Investigational Medicinal Product as outlined in the protocol section 6.6.
    El promotor ofrecerá acceso continuado el medicamento en estudio de Roche (RO7234292) de forma gratuita a los pacientes elegibles de acuerdo con la Politica Gloal de Roche de Acceso Continuado a medicamentos en investigadión como se indica en la sección 6.6 del 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 Decision2022-12-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-12-19
    P. End of Trial
    P.End of Trial StatusOngoing
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