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    Summary
    EudraCT Number:2019-004066-18
    Sponsor's Protocol Code Number:AL001-3
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-15
    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 ConcernedItaly - Italian Medicines Agency
    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.
    Studio di fase 3, multicentrico, randomizzato, in doppio cieco, controllato con placebo, per valutare l’efficacia e la sicurezza di AL001 in soggetti a rischio di o affetti da demenza frontotemporale dovuta a mutazioni eterozigote nel gene della 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.
    Studio in doppio cieco, controllato con placebo, per valutare l’efficacia e la sicurezza di AL001 nella demenza frontotemporale.
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberAL001-3
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04374136
    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/ citySan Francisco
    B.5.3.3Post codeCA 94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number00016508269572
    B.5.6E-mailkristina.vlaovic@alector.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 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 INNAL001
    D.3.9.1CAS number 2376132-27-1
    D.3.9.2Current sponsor codeAL001
    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.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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.
    Demenza frontotemporale.
    E.1.1.1Medical condition in easily understood language
    A type of dementia that causes problems with behaviour and language.
    Un tipo di demenza che causa problemi di comportamento e linguaggio.
    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).
    Valutare l'efficacia di AL001 rispetto a placebo nei portatori di mutazioni del gene progranulina (GRN) che causa una demenza frontotemporale (Frontotemporal Dementia, [FTD]), misurata mediante gli strumenti di stadiazione della demenza in base alla scala di valutazione clinica della demenza (Clinical Dementia Rating, [CDR®]) PIÙ la somma delle caselle nei domini del comportamento e del linguaggio in caso di degenerazione lobare frontotemporale del Centro di coordinamento nazionale della malattia di Alzheimer (National Alzheimer’s Disease Coordinating Center frontotemporal lobar degenration Sum of Boxes, [NACC FTLD-SB]) (CDR® più NACC FTLD-SB).
    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)
    Gli obiettivi secondari di questo studio sono quelli di valutare gli effetti clinici, la sicurezza e la tollerabilità di AL001 rispetto al placebo in portatori di mutazioni di GRN che causano FTD misurata in base a:

    Obiettivo Secondario:
    Valutazioni degli esiti clinici (Clinical Outcome Assessment, [COA])

    Obiettivo Secondario di Farmacodinamica (Pharamcodynamic, [PD]):
    Biomarcatori patologici della malattia

    Obiettivo Secondario di Sicurezza:
    Valutazioni di sicurezza e anticorpi anti-farmaco (Antidrug Antibodies, [ADA])
    E.2.3Trial contains a sub-study No
    E.3Principal 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).
    • Nota mutazione genetica della progranulina che causa FTD.
    • Punteggio CDR più NACC-FTLD 0-2.
    • Se sintomatica, uno dei criteri per la diagnosi di probabile FTD con variante comportamentale o sottotipo semantico della FTD o afasia progressiva nonfluente della FDT.
    • Un partner dello Studio che acconsente alla partecipazione allo studio e che si prende cura del/si reca dal paziente ogni giorno per almeno 5 ore a settimana.
    • Il consenso informato scritto deve essere ottenuto e documentato (dal paziente o, se le giurisdizioni lo consentono, dal proprio decisore legalmente responsabile).
    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.
    Demenza dovuta a una condizione diversa dalla FTD tra cui, ma non limitatamente a, malattia di Alzheimer, malattia di Parkinson, demenza a corpi di Lewy, malattia di Huntington o demenza vascolare.
    Anamnesi nota di gravi reazioni allergiche, anafilattiche o altre reazioni di ipersensibilità ad anticorpi chimerici, umani o umanizzati o proteine di fusione.
    Attuale ipertensione, diabete mellito o malattia della tiroide non controllato/a.
    Malattia cardiaca, malattia epatica o renale clinicamente significativa.
    Anamnesi o evidenza di malattia cerebrale clinicamente significativa diversa dalla FTD.
    Donne in gravidanza o che allattano al seno, oppure che intendono concepire durante il periodo dello studio.
    Qualsiasi vaccino sperimentale o terapia genica.
    Anamnesi di tumore tranne:
    se si ritiene che sia guarito
    se non trattato attivamente con terapia antitumorale o radioterapia e, a giudizio dello sperimentatore, non sembri richiedere il trattamento nei 3 anni successivi
    considerato a bassa probabilità di recidiva
    Per il carcinoma prostatico, nessuna progressione significativa nei 2 anni precedenti. Attuale utilizzo di farmaci anticoagulanti (per es. coumadin, eparinoidi, apixaban).
    Residenza in una struttura di infermieristica specializzata, convalescenza domiciliare o in una struttura di assistenza a lungo termine allo screening, oppure richiede un’assistenza infermieristica continua.
    E.5 End points
    E.5.1Primary end point(s)
    •The CDR® plus NACC FTLD-SB
    • CDR® più NACC FTLD-SB
    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.
    Baseline e settimane 16, 32 e 48 per pazienti sintomatici e baseline e settimane 16,32, 48, 64, 80 e 96 per pazienti pre-sintomatici.
    E.5.2Secondary end point(s)
    •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
    Endpoint secondari:
    • Impressione clinica globale Gravità (CGI S)
    • Punteggio di impressione clinica globale Miglioramento (CGI I)
    • Variazioni rispetto alla baseline nei punteggi delle seguenti COA:
    o Scala di valutazione della demenza frontotemporale (FRS)
    o Batteria riproducibile per la valutazione dello stato neuropsicologico (Repeatable Battery for the Assessment of Neuropsychological Status - RBANS)
    Endpoint di sicurezza secondari:
    • Incidenza, natura e gravità degli eventi avversi e degli eventi avversi seri
    • Anomalie riscontrate nell’esame obiettivo
    • Anomalie riscontrate nell’esame neurologico
    • Variazioni delle funzioni vitali rispetto alla baseline nel tempo
    • Variazioni degli elettrocardiogrammi rispetto alla baseline nel tempo
    • Anomalie riscontrate nella risonanza magnetica
    • Variazioni dei test clinici di laboratorio rispetto alla baseline nel tempo
    • Scala Sheehan-Suicidality Tracking Scale (Sheehan STS)
    • Incidenza degli ADA rispetto a 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.
    • Endpoint secondari: ogni 16 settimane.

    Endpoint di PD: PGRN ogni 24 settimane tramite RMI e misurazioni di NfL ogni 8 settimane e valutazioni di sicurezza ogni 4 settimane.
    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.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 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 concerned4
    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 Information not present in EudraCT
    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
    Canada
    United States
    Belgium
    France
    Germany
    Italy
    Netherlands
    Portugal
    Spain
    Sweden
    Switzerland
    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
    LPLV
    LPLV
    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 months5
    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.3.3.6.1Details 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.
    Se il partecipante allo studio non è competente, un rappresentante legalmente autorizzato deve fornire il consenso informato in sua vece e il partecipante deve esprime il proprio assenso, in conformità alle normative locali, alle linee guida e ai com
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    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).
    Al completamento del periodo di trattamento dello studio in doppio cieco, si prevede di offrire ai partecipanti che risultano idonei uno studio di estensione in aperto (Open Label Extension, [OLE]) con AL001 (da descrivere in un protocollo separato).
    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 Decision2020-05-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-03-31
    P. End of Trial
    P.End of Trial StatusOngoing
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