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    Summary
    EudraCT Number:2023-000185-34
    Sponsor's Protocol Code Number:VAL1221-ITLAFORA-01
    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:2023-02-06
    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 number2023-000185-34
    A.3Full title of the trial
    A SINGLE ARM, OPEN-LABEL, PHASE 2 STUDY TO ASSESS THE SAFETY AND EFFICACY OF VAL-1221 ON SUBJECTS WITH LAFORA DISEASE
    STUDIO IN APERTO DI FASE 2 PER LA VALUTAZIONE DELLA SICUREZZA ED EFFICACIA DI VAL-1221 IN PERSONE CON MALATTIA DI LAFORA
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A SINGLE ARM, OPEN-LABEL, PHASE 2 STUDY TO ASSESS THE SAFETY AND EFFICACY OF VAL-1221 ON SUBJECTS WITH LAFORA DISEASE
    STUDIO IN APERTO DI FASE 2 PER LA VALUTAZIONE DELLA SICUREZZA ED EFFICACIA DI VAL-1221 IN PERSONE CON MALATTIA DI LAFORA
    A.3.2Name or abbreviated title of the trial where available
    VAL1221-ITLAFORA-01
    VAL1221-ITLAFORA-01
    A.4.1Sponsor's protocol code numberVAL1221-ITLAFORA-01
    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 SponsorAZIENDA USL DI BOLOGNA
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportParasail LLC
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportMinistero della Salute
    B.4.2CountryItaly
    B.4.1Name of organisation providing support"Associazione Italiana Lafora", "Associazione Tempo Zero", "Associazione Malattie Rare Mauro Baschirotto"
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIRCCS Istituto delle Scienze Neurologiche di Bologna, Azienda USL di Bologna
    B.5.2Functional name of contact pointIRCCS Ricerca AUSL di Bologna
    B.5.3 Address:
    B.5.3.1Street AddressVia Altura 3
    B.5.3.2Town/ cityBologna
    B.5.3.3Post code40139
    B.5.3.4CountryItaly
    B.5.4Telephone number0514966937
    B.5.5Fax number0000000
    B.5.6E-mailirccs.ricerca@ausl.bologna.it
    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 nameVAL-1221
    D.3.2Product code [VAL-1221]
    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 INNClervonafusp alfa
    D.3.9.1CAS number 2145123-44-8
    D.3.9.2Current sponsor codeVAL-1221
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 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 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.IMP: 2
    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 Yes
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameparacetamolo
    D.3.2Product code [paracetamolo]
    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.3.9.2Current sponsor codeparacetamolo
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 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 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.IMP: 3
    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 Yes
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 namecetirizina
    D.3.2Product code cetirizina
    D.3.4Pharmaceutical form Oral solution
    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.3.9.2Current sponsor codecetrizina
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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 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 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    patients with mid- to late stage Lafora Desease
    pazienti con malattia di Lafora in stadio medio e avanzato
    E.1.1.1Medical condition in easily understood language
    patients with mid- to late stage Lafora Desease
    pazienti con malattia di Lafora in stadio medio e avanzato
    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 10054030
    E.1.2Term Lafora's myoclonic epilepsy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10054030
    E.1.2Term Lafora's myoclonic epilepsy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10054030
    E.1.2Term Lafora's myoclonic epilepsy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10054030
    E.1.2Term Lafora's myoclonic epilepsy
    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
    The primary objective for this study is to evaluate the safety of VAL-1221
    L'obiettivo primario di questo studio è valutare la sicurezza di VAL-1221
    E.2.2Secondary objectives of the trial
    Exploratory efficacy objective
    The secondary objective is the identification and/or evaluation of biomarkers that are predictive of response to VAL-1221, are associated with susceptibility to developing adverse events, can provide evidence of VAL-1221 activity or can increase the knowledge and understanding of disease biology and drug safety
    Obiettivo di efficacia esplorativo
    Obiettivo secondario è l'identificazione e/o la valutazione di biomarcatori predittivi della risposta a VAL-1221, associati alla suscettibilità di sviluppare eventi avversi, in grado di fornire prove dell'attività di VAL-1221 o di aumentare la conoscenza e la comprensione della biologia della malattia e della sicurezza del farmaco
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Documented genetic diagnosis of LD based on pathogenic variants in both alleles of either the EPM2A or the NHLRC1 gene
    2. Between 12 and 28 years of age
    3. Mid- or late-stage in the LD evolution (LD progression scale 2-3)
    4. Able and willing to comply with the study protocol including:
    a. Caregiver/trial partner committed to facilitating patient's involvement in the study who is reliable, competent, and at least 18 years of age.
    b. Adequate cognitive capacity to participate in neuropsychological testing adjusted to their level of functioning
    1. Diagnosi genetica documentata di malattia di Lafora basata su varianti patogene in entrambi gli alleli del gene EPM2A o NHLRC1.
    2. Età compresa tra 12 e 28 anni
    3. Stadio intermedio o tardivo dell'evoluzione della malattia di Lafora (scala di progressione LD 2-3)
    4. Capacità e disponibilità a rispettare il protocollo di studio, tra cui:
    a. Caregiver/partner di studio impegnato a facilitare la partecipazione del paziente allo studio, affidabile, competente e di almeno 18 anni di età.
    b. Adeguata capacità cognitiva per partecipare ai test neuropsicologici adeguati al proprio livello di funzionamento.
    E.4Principal exclusion criteria
    1.Any known genetic abnormality confounding the phenotype
    2.Subjects a. with complete absence of speech OR b. unable to perform any activities of daily living OR c. completely bedridden
    3.Current participation in an interventional or therapeutic study
    4. Pregnancy
    5.Receiving an investigational drug within 90 days of the Baseline Visit
    6.Prior or current treatment with gene or stem cell therapy
    7.Any other diseases which may significantly interfere with the assessment of LD
    8.Have any additional conditions which, in the opinion of the Investigator, would make the subject unsafe for inclusion or could interfere with the subject participating in or completing the study.
    1. Qualsiasi anomalia genetica nota che confonda il fenotipo.
    2.Soggetti a. con assenza completa di linguaggio OPPURE b. incapaci di svolgere qualsiasi attività della vita quotidiana OPPURE c. completamente allettati
    3. Partecipazione attuale a uno studio interventistico o terapeutico.
    4. Gravidanza
    5. Terapia con un farmaco in fase di sperimentazione entro 90 giorni dalla visita di riferimento.
    6.Trattamento precedente o in corso con terapia genica o con cellule staminali
    7. Altre malattie che possono interferire significativamente con la valutazione della malattia di Lafora.
    8. Condizioni aggiuntive che, a giudizio dello sperimentatore, potrebbero rendere il soggetto non sicuro per l'inclusione o interferire con la partecipazione o il completamento dello studio.
    E.5 End points
    E.5.1Primary end point(s)
    - Nature, incidence, seriousness and severity of adverse events
    - Incidence of treatment discontinuation due to adverse events
    - Nature, incidence, seriousness, severity, and timing of infusion-related reactions
    - Mean change in vital signs from baseline over time and incidence of abnormal vital sign measurements
    - Changes in ECG assessments from baseline over time and incidence of abnormal ECG assessments
    - Change from baseline and incidence of laboratory abnormalities (including hematology, clinical chemistry, coagulation, and urinalysis parameters)
    - Natura, incidenza, serietà e gravità degli eventi avversi.
    - Incidenza di interruzione del trattamento a causa di eventi avversi
    - Natura, incidenza, gravità, severità e tempistica delle reazioni correlate all'infusione.
    - Variazione media dei segni vitali rispetto al basale nel tempo e incidenza di misurazioni anomale dei segni vitali
    - Variazioni delle valutazioni ECG dal basale nel tempo e incidenza di valutazioni ECG anomale
    - Variazione dal basale e incidenza di anomalie di laboratorio (compresi i parametri ematologici, di chimica clinica, di coagulazione e di analisi delle urine).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening visit, V1, V2, V3, V4
    Screening visit, V1, V2, V3, V4
    E.5.2Secondary end point(s)
    Epilepsy
    - Change in tonic-clonic seizures frequency comparing the trial period with the 6 months preceding the baseline
    - Change in frequency of epileptiform discharges on EEG
    Cognitive functions
    - Non-worsening or improvement of Montreal Cognitive Assessment (MoCA)
    - Non-worsening or improvement of Leiter Performance Scale 3 (Leiter-3)
    - Non-worsening or improvement of the Visual Motor Integration test (VMI)
    - Non-worsening or improvement of children’s color trail test
    - Non-worsening or improvement of NEPSY-II inhibition subtest
    - Non-worsening or improvement of verbal fluency tests
    - Non-worsening or improvement of Weiss working memory subtest
    - Non-worsening or improvement of Weiss vocabulary subtest
    Motor functions
    - Non-worsening or improvement of 4-Stage Balance Test
    - Non-worsening or improvement of Timed Up and Go (TUG)
    - Non-worsening or improvement of 6-minute walk test (6MWT)
    - Non-worsening or improvement of 10-Meter Walk Test (10MWT)
    - Non-worsening or improvement of 9-hole peg test (9HPT)
    - Non-worsening or improvement of Scale for Assessment and Rating of Ataxia (SARA)
    - Non-worsening or improvement of Unified Myoclonus Rating Scale (UMRS)
    Global assessment and disease burden
    - Non-worsening or improvement of Lafora Disease Performance Scale (LDPS)
    - Non-worsening or improvement of Lafora Epilepsy Severity Scale (LESS)
    - Non-worsening or improvement of Barthel Index
    - Non-worsening or improvement of Vineland-II
    - Change of Clinical Global Impression– Improvement (CGI-I)
    - Change of Patient Global Impression – Change (PGI-C)
    - Change of Caregiver Global Impression – Change (CaGI-C)
    - Non-worsening or improvement of Burden Scale for Family Caregivers (short form); - Blood and CSF biomarkers
    - Brain MRI measures
    - Brain FDG-PET measures
    Epilessia
    - Variazione della frequenza delle crisi tonico-cloniche confrontando il periodo di sperimentazione con i 6 mesi precedenti la valutazione basale
    - Variazione della frequenza delle anomalie epilettiformi sull'EEG

    Funzioni cognitive
    - Non peggioramento o miglioramento del Montreal Cognitive Assessment (MoCA)
    - Non peggioramento o miglioramento della Leiter Performance Scale 3 (Leiter-3)
    - Non peggioramento o miglioramento del Visual Motor Integration test (VMI)
    - Non peggioramento o miglioramento del children’s color trail test
    - Non peggioramento o miglioramento del subtest di inibizione NEPSY-II
    - Non peggioramento o miglioramento dei test di fluenza verbale
    - Non peggioramento o miglioramento del sottotest di Weiss sulla memoria di lavoro
    - Non peggioramento o miglioramento del sottotest di Weiss sul vocabolario

    Funzioni motorie
    - Non peggioramento o miglioramento del test di equilibrio a 4 fasi
    - Non peggioramento o miglioramento del Timed Up and Go (TUG)
    - Non peggioramento o miglioramento del test del cammino di 6 minuti (6MWT)
    - Non peggioramento o miglioramento del test del cammino di 10 metri (10MWT)
    - Non peggioramento o miglioramento del test del 9-hole peg test (9HPT)
    - Non peggioramento o miglioramento della Scala di Valutazione dell'Atassia (SARA)
    - Non peggioramento o miglioramento della Scala Unificata di Valutazione del Mioclono (UMRS)

    Valutazione globale e impatto di malattia
    - Non peggioramento o miglioramento della Lafora Disease Performance Scale (LDPS)
    - Non peggioramento o miglioramento della Lafora Epilepsy Severity Scale (LESS)
    - Non peggioramento o miglioramento dell'Indice di Barthel
    - Non peggioramento o miglioramento di Vineland-II
    - Variazione dell'Impressione Globale Clinica - Miglioramento (CGI-I)
    - Variazione dell'Impressione Globale del Paziente - Cambiamento (PGI-C)
    - Variazione dell'Impressione Globale del Caregiver (CaGI-C)
    - Non peggioramento o miglioramento della Burden Scale for Family Caregivers (forma breve); - Biomarcatori del sangue e del liquor
    - Misure di risonanza magnetica cerebrale
    - Misure di FDG-PET cerebrale
    E.5.2.1Timepoint(s) of evaluation of this end point
    Screening visit, V1, V2, V3, V4; Screening visit, V3
    Screening visit, V1, V2, V3, V4; Screening visit, V3
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    a braccio singolo, in aperto, confrontato con il basale
    single-arm, open label, baseline-controlled
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.7Trial has a data monitoring committee No
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1.1In 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) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    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 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
    minors
    minori
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    cognitive deterioration due to neurodegeneration
    disabilità intellettiva dovuta alla neurodegenerazione
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 6
    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)
    If the therapy proves to be safe and effective and patients need to continue it, the manufacturer undertakes to supply the drug even after the end of the study.
    In any case, patients will return to the care pathway and be followed up at the centre as per clinical practice.
    Se la terapia risulta sicura ed efficace ed i pazienti avessero necessità di proseguirla l'azienda produttrice si impegna a fornire il farmaco anche dopo il termine dello studio.
    In ogni caso i pazienti rientreranno nel percorso assistenziale e saranno seguiti presso il centro come da pratica clinica.
    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 Decision2023-04-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-03-16
    P. End of Trial
    P.End of Trial StatusOngoing
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