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    Summary
    EudraCT Number:2022-000747-77
    Sponsor's Protocol Code Number:283PD302
    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:2022-07-27
    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 number2022-000747-77
    A.3Full title of the trial
    A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Efficacy and Safety of BIIB122/DNL151 in Participants with Parkinson’s Disease and Pathogenic LRRK2 Variants
    Estudio Fase 3, multicéntrico, aleatorizado, doble ciego, controlado con placebo para determinar la eficacia y seguridad de BIIB122/DNL151 en participantes con Enfermedad de Parkinson y variantes patógenas de LRRK2
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Assess if BIIB122 Tablets are Safe and can Slow Worsening of Early-Stage Parkinson's Disease in Participants with Specific LRRK2 Genetic Variants Between the Ages of 30 and 80 Using the Movement Disorder Society-Unified Parkinson's Disease Rating Scale
    Un estudio para evaluar si los comprimidos BIIB122 son seguros y pueden retrasar el empeoramiento de la enfermedad de Parkinson en etapa temprana en participantes con variantes genéticas específicas de LRRK2 entre las edades de 30 y 80 utilizando la Escala de Calificación de la Enfermedad de Parkinson Unificada de la Sociedad de Trastornos del Movimiento
    A.3.2Name or abbreviated title of the trial where available
    LIGHTHOUSE
    A.4.1Sponsor's protocol code number283PD302
    A.5.4Other Identifiers
    Name:IND/IDE Number:Number:135743
    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 SponsorBiogen Idec Research Limited
    B.1.3.4CountryUnited Kingdom
    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 supportBiogen Idec Research Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBiogen Idec Research Limited
    B.5.2Functional name of contact pointN/A
    B.5.3 Address:
    B.5.3.1Street AddressInnovation House, 70 Norden Road
    B.5.3.2Town/ cityMaidenhead, Berkshire
    B.5.3.3Post codeSL6 4AY
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+34913107110
    B.5.6E-mailclinicaltrials@biogen.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 nameBIIB122
    D.3.2Product code DNL151
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNA
    D.3.9.1CAS number 2170179-24-3
    D.3.9.2Current sponsor codeDNL151, DN0001575, DN1575
    D.3.9.3Other descriptive nameBIIB122
    D.3.9.4EV Substance CodeSUB221129
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Parkinson's Disease
    Enfermedad de Parkinson
    E.1.1.1Medical condition in easily understood language
    Parkinson's Disease
    Enfermedad de Parkinson
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10061536
    E.1.2Term Parkinson's disease
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of BIIB122 225 mg compared with placebo, based on the time to confirmed worsening in MDS-UPDRS Parts II and III combined score
    Evaluar la eficacia de 225 mg de BIIB122 en comparación con placebo, según el tiempo hasta el empeoramiento confirmado de la puntuación combinada de las partes II y III de la escala unificada para la evaluación de la enfermedad de Parkinson de la Sociedad de trastornos del movimiento (MDS-UPDRS)
    E.2.2Secondary objectives of the trial
    - To evaluate the safety and tolerability of BIIB122 225 mg compared with placebo when administered for 96 to 180 weeks
    - To evaluate the efficacy of BIIB122 225 mg compared with placebo, based on the time to confirmed worsening in MDS-UPDRS Part II score
    - To evaluate the efficacy of BIIB122 225 mg compared with placebo, based on change in the MDS-UPDRS Parts II and III combined score, when administered for 96 weeks
    - To evaluate the efficacy of BIIB122 225 mg compared with placebo, based on change in SE-ADL score
    - To evaluate the efficacy of BIIB122 225 mg compared with placebo, based on change in MDS-UPDRS Parts I, II, and III combined score
    - Evaluar la seguridad y la tolerabilidad de 225 mg de BIIB122 en comparación con placebo cuando se administra entre 96 y 180 semanas.
    - Evaluar la eficacia de 225 mg de BIIB122 en comparación con placebo según el tiempo hasta el empeoramiento confirmado de la puntuación de la parte II de la MDS-UPDRS.
    - Evaluar la eficacia de 225 mg de BIIB122 en comparación con placebo según el cambio en la puntuación combinada de las partes II y III de la MDS-UPDRS cuando se administra durante 96 semanas.
    - Evaluar la eficacia de 225 mg de BIIB122 en comparación con placebo según el cambio en la puntuación de la escala de actividades de la vida diaria de Schwab y England (SE-ADL).
    - Evaluar la eficacia de 225 mg de BIIB122 en comparación con placebo según el cambio en la puntuación combinada de las partes I, II y III de la MDS-UPDRS.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Two biomarker substudies, a biofluid substudy and an imaging substudy, may be conducted to evaluate exploratory biofluid and imaging biomarkers in a subset of participants.

    The objective of the biofluid substudy is to evaluate the change in central and peripheral biomarkers over time. Collection will include LPs for CSF collection and blood sampling. All biofluid substudy participants are planned to participate in the CSF portion of the substudy. A subset of those participants is planned to participate in the PBMC portion of the substudy. Samples will be evaluated for measures of LRRK2 pathway, lysosomal function, and exploratory measures relevant to PD.

    The objective of the imaging substudy is to quantify changes in nigral-striatal dopaminergic integrity over time using PET and MRI. The substudy will be conducted with participants who are willing and able to travel to central imaging facilities from participating study sites. The overall screening window will be increased by 7 days to a maximum of 42 days for participants in the imaging substudy to allow enough time to complete assessments. Study site participation will be based on PET radiotracer availability and site geography, at the discretion of the Sponsor. PET imaging will use the 18F-AV133 radioligand that binds VMAT-2. MRI may include, and not be limited to structural, neuromelanin-sensitive, and susceptibility-weighted MRI sequences (to be detailed in the Imaging/MRI Manual) to allow PET quantification and evaluation of exploratory MRI biomarkers.

    Participation in either substudy is optional for participants, where approved per local regulations. All participants who opt to participate in either of the substudies must provide additional written, informed consent. Participation in a substudy is optional and does not exclude participation in the other substudy.
    Se pueden realizar dos subestudios de biomarcadores, un subestudio de líquidos corporales y un subestudio de técnicas de diagnóstico por la imagen para evaluar biomarcadores exploratorios de líquidos corporales y de técnicas de diagnóstico por la imagen en un subconjunto de participantes.

    El objetivo del subestudio de líquidos corporales es evaluar el cambio en los biomarcadores centrales y periféricos a lo largo del tiempo. La Español Se pueden realizar dos subestudios de biomarcadores, un subestudio de líquidos corporales y un subestudio de técnicas de diagnóstico por la imagen para evaluar biomarcadores exploratorios de líquidos corporales y de técnicas de diagnóstico por la imagen en un subconjunto de participantes.

    El objetivo del subestudio de líquidos corporales es evaluar el cambio en los biomarcadores centrales y periféricos a lo largo del tiempo. La recogida incluirá PL para la recogida de LCR y la obtención de muestras de sangre. Está previsto que todos los participantes del subestudio de líquidos corporales participen en la parte de LCR del subestudio. Está previsto que un subconjunto de estos participantes participe en la parte de CMSP del subestudio. Las muestras se evaluarán para determinar las medidas de la vía de LRRK2, la función lisosómica y las medidas exploratorias relevantes para la EP.

    El objetivo del subestudio de técnicas de diagnóstico por la imagen es cuantificar los cambios en la integridad dopaminérgica nigroestriatal a lo largo del tiempo mediante TEP y RM. El subestudio se llevará a cabo con participantes que estén dispuestos a y sean capaces de desplazarse a las instalaciones centrales de técnicas de diagnóstico por la imagen desde los centros del estudio participantes. El intervalo de selección global se incrementará en 7 días hasta un máximo de
    42 días para que los participantes del subestudio de técnicas de diagnóstico por la imagen tengan tiempo suficiente para completar las evaluaciones. La participación de los centros del estudio se basará en la disponibilidad del radiomarcador de TEP y la geografía del centro, a discreción del promotor. La TEP utilizará el radioligando 18F-AV133 que se une a VMAT-2. La RM puede incluir, entre otras, secuencias de RM estructurales, sensibles a la neuromelanina y ponderadas por susceptibilidad (que se detallarán en el manual de RM/técnicas de diagnóstico por la imagen) para permitir la cuantificación mediante TEP y la evaluación de biomarcadores exploratorios de RM.

    La participación en cualquiera de los subestudios es opcional para los participantes, cuando lo apruebe la normativa local. Todos los participantes que opten por participar en cualquiera de los subestudios deben proporcionar un consentimiento informado por escrito aparte. La participación en un subestudio es opcional y no excluye la participación en el otro subestudio.
    E.3Principal inclusion criteria
    Key Inclusion Criteria:
    - Clinical diagnosis of PD meeting the Movement Disorder Society Clinical Diagnostic Criteria within 5 years of the Screening Visit, inclusive, and at least 30 years of age at the time of diagnosis
    - Modified Hoehn and Yahr scale, Stages 1 to 2.5 (in OFF state), inclusive, at Screening
    - MDS-UPDRS Parts II and III (in OFF state) combined score <=40 at Screening
    - Screening genetic test results verifying the presence of a pathogenic leucine-rich repeat kinase 2 (LRRK2) variant
    Criterios de inclusión principales:
    • Diagnóstico clínico de EP que cumpla los criterios de diagnóstico clínico de la Sociedad de Trastornos del Movimiento en los 5 años anteriores a la visita de selección, inclusive, y tener al menos 30 años de edad en el momento del diagnóstico.
    • Estadios 1 a 2.5 de la escala modificada de Hoehn y Yahr (en ausencia de respuesta), ambos inclusive en la selección.
    • Puntuación combinada de las partes II y III de la MDS-UPDRS (en ausencia de respuesta) de <=40 en la selección.
    • Resultados de las pruebas genéticas de selección que verifiquen la presencia de una variante patógena de la cinasa rica en repeticiones de leucina 2 (LRRK2).
    E.4Principal exclusion criteria
    Key Exclusion Criteria:
    - Clinically significant neurologic disorder other than PD, including, but not limited to, stroke, dementia, or seizure within 5 years of Screening Visit, in the opinion of the Investigator
    - Clinical evidence of atypical parkinsonism (e.g., multiple-system atrophy or progressive supranuclear palsy) or evidence of drug-induced parkinsonism
    Criterios de exclusión principales:
    • Trastorno neurológico clínicamente significativo distinto de la EP, incluidos, entre otros, accidente cerebrovascular, demencia o crisis epilépticas, en los 5 años anteriores a la visita de selección, en opinión del investigador.
    • Indicios clínicos de parkinsonismo atípico (p. ej., atrofia multisistémica o parálisis supranuclear progresiva) o indicios de parkinsonismo inducido por fármacos.
    E.5 End points
    E.5.1Primary end point(s)
    Time to Confirmed Worsening in Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Parts II and III Over the Treatment Period.
    Tiempo hasta el empeoramiento confirmado en las partes II y III de la escala unificada para la evaluación de la enfermedad de Parkinson de la Sociedad de trastornos del movimiento (MDS-UPDRS) a lo largo del periodo de tratamiento.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to Week 180
    Hasta la semana 180.
    E.5.2Secondary end point(s)
    AEs: Day 1 up to Week 187; SAEs: Screening up to Week 187:
    - Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Up to Week 180:
    - Time to Confirmed Worsening in MDS-UPDRS Part II Score Over the Treatment Period

    Baseline up to Week 96:
    - Change From Baseline in MDS-UPDRS Parts II and III Combined Score

    Up to Week 180:
    - Time to Confirmed Worsening in Schwab and England Activities of Daily Living Scale (SE-ADL) Score Over the Treatment Period

    Baseline up to Week 96:
    - Change From Baseline in MDS-UPDRS Parts I, II, and III Combined Score
    AA: día 1 hasta la semana 187; AAG: selección hasta la semana 187:
    - Número de participantes con acontecimientos adversos surgidos durante el tratamiento (AAST) y acontecimientos adversos graves (AAG).

    Hasta la semana 180:
    - Tiempo hasta el empeoramiento confirmado de la puntuación de la parte II de la MDS-UPDRS a lo largo del periodo de tratamiento.

    Desde el inicio hasta la semana 96:
    - Cambio con respecto al inicio en la puntuación combinada de las partes II y III de la MDS-UPDRS.

    Hasta la semana 180:
    - Tiempo hasta el empeoramiento confirmado en la escala de actividades de la vida diaria de Schwab y England (SE-ADL) a lo largo del periodo de tratamiento.

    Desde el inicio hasta la semana 96:
    - Cambio con respecto al inicio en la puntuación combinada de las partes I, II y III de la MDS-UPDRS.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Timepoints mentioned in section E.5.2
    Puntos temporales mencionados en el apartado E.5.2
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group 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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA24
    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
    Brazil
    Canada
    Egypt
    Israel
    Tunisia
    United States
    France
    Spain
    Germany
    Italy
    Belgium
    Norway
    Portugal
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years8
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days28
    E.8.9.2In all countries concerned by the trial years8
    E.8.9.2In all countries concerned by the trial months9
    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: 278
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 122
    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 state21
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 125
    F.4.2.2In the whole clinical trial 400
    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)
    None
    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 Decision2022-10-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-10-03
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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