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    EudraCT Number:2019-004066-18
    Sponsor's Protocol Code Number:AL001-3
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2020-03-06
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2019-004066-18
    A.3Full title of the trial
    A Phase 3, Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene
    Estudio de fase 3, multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia y la seguridad de AL001 en pacientes con riesgo o con demencia frontotemporal causada por mutaciones heterocigotas en el gen de la progranulina
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of AL001 in FTD
    Estudio doble ciego y controlado con placebo para evaluar la eficacia y la seguridad de AL001 en DFT
    A.4.1Sponsor's protocol code numberAL001-3
    A.5.4Other Identifiers
    Name:IND numberNumber:135892
    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 SponsorAlector 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 supportAlector Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlector Inc.
    B.5.2Functional name of contact pointKristina Vlaovic
    B.5.3 Address:
    B.5.3.1Street Address131 Oyster Point Boulevard, Suite 600
    B.5.3.2Town/ citySouth San Francisco
    B.5.3.3Post codeCA 94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34900834 223
    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 nameAL001
    D.3.2Product code AL001
    D.3.4Pharmaceutical form Solution for infusion
    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 INNnot available
    D.3.9.1CAS number 2376132-27-1
    D.3.9.2Current sponsor codeAL001
    D.3.9.3Other descriptive nameAL001
    D.3.9.4EV Substance CodeSUB193428
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Frontotemporal Dementia
    Demencia Frontotemporal
    E.1.1.1Medical condition in easily understood language
    A type of dementia that causes problems with behaviour and language
    Un tipo de demencia que causa problemas en el comportamiento y el lenguaje
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10068968
    E.1.2Term Frontotemporal dementia
    E.1.2System Organ Class 10029205 - Nervous system 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 efficacy of AL001 compared with placebo in carriers of progranulin gene (GRN) mutations causative of frontotemporal dementia (FTD) as measured by the Clinical Dementia Rating Dementia Staging Instrument PLUS National Alzheimer’s Disease Coordinating Center frontotemporal lobar degeneration Behavior & Language Domains Sum of Boxes (CDR® plus NACC FTLD-SB).
    Evaluar la eficacia de AL001 en comparación con placebo en portadores de mutaciones en el gen de la progranulina (GRN) causantes de demencia frontotemporal (DFT), determinada mediante el instrumento de estadificación de la demencia de valoración de la demencia clínica MÁS suma de recuadros de los dominios de comportamiento y lenguaje en la degeneración lobular frontotemporal del National Alzheimer’s Disease Coordinating Center (CDR® más FTLD-SB del NACC)
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to evaluate the clinical effects, safety, and tolerability of AL001 compared with placebo in carriers of GRN mutations causative of FTD as measured by:

    Secondary Objective:
    Clinical outcome assessments (COAs)

    Secondary Pharmacodynamic (PD) Objective:
    Disease pathology biomarkers

    Secondary Safety Objective:
    Safety assessments and antidrug antibodies (ADAs)
    Los objetivos secundarios de este estudio son evaluar los efectos clínicos, la seguridad y la tolerabilidad de AL001 en comparación con placebo en portadores de mutaciones en GRN causantes de DFT determinados mediante:

    Objectivo secundario:
    Evaluaciones de resultados clínicos (ERC)

    Objetivo farmacodinámico (FD) secundario:
    Biomarcadores anatomopatológicos de la enfermedad

    Objetivo secundario de seguridad:
    Evaluaciones de la seguridad y anticuerpos contra el fármaco (ACF)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Each participant must meet all of the following criteria to be enrolled in this study.

    Key Inclusion Criteria:
    • Known progranulin genetic mutation causing FTD.
    • CDR plus NACC-FTLD score 0-2.
    • If symptomatic, one of the criteria for the diagnosis of probable behavioral variant FTD or FTD-semantic subtype or FTD-Progressive Nonfluent Aphasia.
    • Study partner who consents to study participation and who cares for/visits the patient daily for at least 5 hours per week.
    • Written informed consent must be obtained and documented (from the patient or, where jurisdictions allow it, from their legal decision maker).
    Los pacientes deberán cumplir todos los criterios siguientes para poder participar en este estudio.

    Criterios de inclusión fundamentales:
    Mutación genética conocida que cusante de DFT.
    Una puntuación CDR más FTLD del NACC entre 0 y 2
    En participantes sintomáticos, uno de los criterios para el diagnóstico de posible demencia frontotemporal de variante conductual o de subtipo semántico o de afasia progresiva primaria
    El paciente dispone de una persona (“acompañante del estudio”) con la que tiene un contacto frecuente y suficiente (al menos 5 horas a la semana de contacto en persona), que otorgue el consentimiento informado necesario.

    El paciente está dispuesto a otorgar su consentimiento informado. Si el paciente no es competente, un representante legal deberá otorgar su consentimiento informado en su nombre
    E.4Principal exclusion criteria
    Dementia due to a condition other than FTD including, but not limited to, Alzheimer’s disease, Parkinson’s disease, dementia with Lewy bodies, Huntington disease, or vascular dementia.
    Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.
    Current uncontrolled hypertension, diabetes mellitus or thyroid disease.
    Clinically significant heart disease, liver disease or kidney disease.
    History or evidence of clinically significant brain disease other than FTD.
    Females who are pregnant or breastfeeding, or planning to conceive within the study period.
    Any experimental vaccine or gene therapy.
    History of cancer except:
    If considered to be cured
    If not being actively treated with anti-cancer therapy or radiotherapy and, in the opinion of the investigator, not likely to require treatment in the ensuing 3 years
    Is considered to have low probability of recurrence
    For prostate cancer, no significant progression over the previous 2 years
    Current use of anticoagulant medications (e.g., coumadin, heparinoids, apixaban).
    Residence in a skilled nursing facility, convalescent home, or long term care facility at screening; or requires continuous nursing care.
    1.Demencia debida a una enfermedad distinta de la DFT, entre otras, enfermedad de Alzheimer, enfermedad de Parkinson, demencia con cuerpos de Lewy, enfermedad de Huntington o demencia vascular.
    2.El paciente tiene antecedentes conocidos de reacciones alérgicas, anafilácticas u otras reacciones de hipersensibilidad graves a anticuerpos quiméricos, humanos o humanizados o a proteínas de fusión.
    3.El paciente tiene hipertensión, diabetes mellitus o enfermedad de tiroides no controladas
    4.Enfermedad clínicamente significativa cardiaca, hepática o renal
    5.Evidencia de enfermedad cerebral clínicamente significativa distinta a la DFT
    6.Mujeres embarazadas o dando de mamar o que planean tener hijos durante el periodo del estudio
    7.Haber recibido terapia génica o vacunas experimentales
    8.El paciente tiene antecedentes de cáncer, excepto si:
    a.Se considera probable que se cure.
    b.No está recibiendo tratamiento antineoplásico o radioterapia de forma activa y, en opinión del investigador, no es probable que precise tratamiento en los 3 años siguientes.
    c.Se considera que tiene pocas probabilidades de recidiva
    d.En caso de cáncer de próstata, no ha presentado progresión significativa en los 2 últimos años.
    9.Uso de Anticoagulantes (por ejemplo, warfarina, heparinoides o apixabán)
    10.El paciente reside en un centro de cuidados especializados, una clínica de reposo o un centro de cuidados a largo plazo en la selección o requiere cuidados de enfermería continuos.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoint:
    •The CDR® plus NACC FTLD-SB
    Criterio de valoración primario:
    CDR más FTLD del NACC
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and weeks 16, 32 and 48 for symptomatic patients and baseline and weeks 16,32, 48, 64, 80 and 96 for pre symptomatic patients.
    Basal y semanas 16, 32 y 48 para pacientes sintomáticos y basal y semanas 16,32, 48, 64, 80 y 96 para pacientes pre sintomáticos.
    E.5.2Secondary end point(s)
    Secondary Endpoints:
    •Clinical Global Impression-Severity (CGI S)
    •Clinical Global Impression-Improvement (CGI I)
    •The changes from baseline in the scores of the following COAs:
    o Frontotemporal Dementia Rating Scale (FRS)
    o Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)

    Secondary PD Endpoints:
    •The changes from baseline in the following:
    o Structural volumetric magnetic resonance imaging (MRI) whole and regional brain volume
    o Progranulin protein (PGRN) concentrations in plasma and optional cerebrospinal fluid
    (CSF) o Neurofilament-light chain (NfL)
    concentrations in serum and optional CSF

    Secondary Safety Endpoints:
    •Incidence, nature, and severity of adverse events and serious adverse events
    •Physical examination abnormalities
    •Neurological examination abnormalities
    •Changes in vital signs from baseline over time
    •Changes in electrocardiograms from baseline over time
    •MRI abnormalities
    •Changes in clinical laboratory tests from baseline over time
    •Sheehan-Suicidality Tracking Scale (Sheehan STS)
    •Incidence of ADAs to AL001
    Criterios de Valoración Secundarios:
    •Impresión clínica global-Intensidad (CGI-S)
    •Impresión clínica global-Mejoría (CGI-I)
    •Cambios desde el momento basal en la puntuación de los siguientes ERCs:
    - Escala de valoración de la de la demencia frontotemporal (FRS)
    - Batería repetible para la evaluación del estado neuropsicológico (RBANS)

    Criterios de valoración FD secundarios:
    •Variaciones con respecto al momento basal de lo siguiente:
    - Volumen cerebral total y regional mediante resonancia magnética (RM) volumétrica estructural
    - Concentraciones de proteína progranulina (PGRN) en plasma y líquido cefalorraquídeo (LCR) opcional
    - Concentraciones de cadenas ligeras de neurofilamentos (NfL) en suero y LCR opcional

    Criterios de valoración secundarios de la seguridad:
    •Incidencia, naturaleza e intensidad de los acontecimientos adversos y acontecimientos adversos graves.
    •Anomalías en la exploración física
    •Anomalías en la exploración neurológica
    •Variaciones de las constantes vitales con respecto al momento basal a lo largo del tiempo
    •Variaciones de los electrocardiogramas con respecto al momento basal a lo largo del tiempo.
    •Anomalías en la RM
    •Variaciones de los análisis clínicos con respecto al momento basal a lo largo del tiempo.
    •Escala de seguimiento de la tendencia suicida de Sheehan (Sheehan STS)
    •Incidencia de ACF contra AL001
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary endpoints: every 16 weeks.

    PD endpoints: MRI every 24 weeks PGRN and NfL measurements every 8 weeks and safety assessments every 4 weeks.
    Criterios de valoración secundarios: cada 16 semanas

    Criterios de valoración FD secundarios: RM cerebral cada 24 semanas, medidas de PGRN y NfL cada 8 semanas y evaluaciones de seguridad cada 4 semanas.
    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 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. 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 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 trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA31
    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
    United Kingdom
    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
    Ultima Visita del Ultimo Paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months5
    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: 144
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 36
    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. of subjects incapable of giving consent
    If the study participant is not competent, a legally authorised representative must provide informed consent on their behalf, and the participant must provide assent, in accordance with the local regulations, guidelines, and ethics committees.
    Si el paciente no es competente, un representante legal deberá otorgar su consentimiento informado en su nombre y el paciente deberá otorgar su asentimiento, de conformidad con las normas locales, las directrices y el CEIm
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 116
    F.4.2.2In the whole clinical trial 180
    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)
    Upon completion of the double blind study treatment period, participants who qualify are planned to be offered an open label extension (OLE) study with AL001 (to be described in a separate protocol).
    Después de completar el periodo de tratamiento doble ciego del estudio, está planeado ofrecer a los participantes que cualifiquen una extensión abierta del estudio con AL001 (será descrito en un protocolo aparte)
    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 Decision2020-04-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-19
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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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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