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    Summary
    EudraCT Number:2021-002350-90
    Sponsor's Protocol Code Number:EFC17045
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-01-26
    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 number2021-002350-90
    A.3Full title of the trial
    A randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naïve or untreated for at least 6 months
    Studio di fase 3 randomizzato, in doppio cieco, controllato con placebo, della durata di 12 mesi per valutare l’effetto di venglustat sul dolore neuropatico e addominale in adulti di sesso maschile e femminile affetti da malattia di Fabry che siano naïve al trattamento o non trattati da almeno 6 mesi
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry disease
    Studio per valutare l’effetto di venglustat compresse sul dolore neuropatico e addominale in adulti di sesso maschile e femminile affetti da malattia di Fabry
    A.3.2Name or abbreviated title of the trial where available
    .
    .
    A.4.1Sponsor's protocol code numberEFC17045
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1256-9310
    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 SponsorSANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
    B.1.3.4CountryFrance
    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 supportSanofi-Aventis Recherche & Développement
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanofi S.r.l
    B.5.2Functional name of contact pointCONTACT POINT
    B.5.3 Address:
    B.5.3.1Street AddressVIALE LUIGI BODIO 37/B
    B.5.3.2Town/ cityMilano
    B.5.3.3Post code20158
    B.5.3.4CountryItaly
    B.5.4Telephone number800536389
    B.5.5Fax number000000
    B.5.6E-mailinformazioni.medicoscientifiche@sanofi.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/3/14/1310
    D.3 Description of the IMP
    D.3.1Product nameVenglustat
    D.3.2Product code [SAR402671, GZ402671 o GZ/SAR402671]
    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 INNVenglustat malato
    D.3.9.1CAS number 1629063-78-0
    D.3.9.2Current sponsor codeGZ402671
    D.3.9.3Other descriptive nameGZ/SAR402671
    D.3.9.4EV Substance CodeSUB166602
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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
    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
    Fabry Disease
    Malattia di Fabry
    E.1.1.1Medical condition in easily understood language
    Fabry Disease
    Malattia di Fabry
    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 24.1
    E.1.2Level PT
    E.1.2Classification code 10016016
    E.1.2Term Fabry'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 assess the effect of venglustat on neuropathic or abdominal pain in adult Fabry disease patients who are treatment-naïve or untreated for at least 6 months prior to screening.
    Valutare l’effetto di venglustat sul dolore neuropatico o addominale in partecipanti adulti affetti da malattia di Fabry che siano naïve al trattamento o non trattati da almeno 6 mesi prima dello screening.
    E.2.2Secondary objectives of the trial
    - To assess the effect of venglustat on plasma globotriaosylsphingosine (lyso-GL-3) levels
    - To evaluate the effect of venglustat on symptoms of Fabry disease
    - To assess the safety and tolerability of venglustat in patients with Fabry disease
    - To evaluate the pharmacokinetics (PK) of venglustat in patients with Fabry disease
    - Valutare l’effetto di venglustat sui livelli di liso-GL-3 nel plasma
    - Valutare l’effetto di venglustat sui sintomi della malattia di Fabry
    - Valutare la sicurezza e la tollerabilità di venglustat in partecipanti affetti da malattia di Fabry
    - Valutare la PK di venglustat in partecipanti affetti da malattia di Fabry
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male and female adult patients 18 year of age or older, who have had a previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease.
    - Patients who are treatment-naïve or without prior treatment with an approved or experimental therapy for Fabry disease within at least 6 months prior to screening.
    - Elevated plasma globotriaosylsphingosine (lyso GL 3) at screening, or a previous biopsy of any organ that shows deposition of GL 3.
    - Average score of >/=3 (0=no symptom, 10=symptom as bad as you can imagine) on neuropathic pain in upper extremities (item 1), and/or neuropathic pain in lower extremities (item 5) and/or abdominal pain (item 10), as measured by the Fabry Disease Patient-Reported Outcome (FD PRO) at screening.
    - Contraception for male or female participants: not pregnant or breastfeeding; no sperm donating for male participant.
    - A signed informed consent must be provided prior to any study-related procedures
    - Partecipanti adulti di età pari o superiore ai 18 anni di sesso maschile e femminile che hanno ricevuto una diagnosi precedentemente confermata di malattia di Fabry e un’anamnesi di sintomi clinici della malattia di Fabry.
    - Partecipanti naïve al trattamento o senza precedente trattamento con una terapia approvata o sperimentale per la malattia di Fabry non più di 6 mesi prima dello screening.
    - Elevato livello di liso-GL-3 nel plasma allo screening o una precedente biopsia di qualsiasi organo che mostri deposito di GL-3.
    -Punteggio medio >/=3 (0 = nessun sintomo, 10 = il peggior sintomo che si possa immaginare) sul dolore neuropatico alle estremità superiori (Voce 1) e/o sul dolore neuropatico alle estremità inferiori (Voce 5) e/o sul dolore addominale (Voce 10), misurati mediante il Fabry Disease Patient-Reported Outocme (FD-PRO) allo screening.
    - L’uso di contraccettivi da parte di uomini e donne: non in gravidanza o in allattamento; non donatori di sperma per i partecipanti di sesso maschile.
    -Un consenso informato scritto deve essere fornito prima di eventuali procedure correlate allo studio.
    E.4Principal exclusion criteria
    - Any manifestations of Fabry disease that preclude placebo administration.
    - History of transient ischemic attack, stroke, myocardial infarction, heart failure, major cardiovascular surgery, or kidney transplantation.
    - History of ongoing clinically significant cardiac arrhythmia or prior or ongoing treatment for the above.
    - Patients with hepatitis C, HIV, or hepatitis B infection.
    - Neuropathic pain in upper or lower extremities, or abdominal pain not related to Fabry disease.
    - History of seizures currently requiring treatment.
    - Uncontrolled hypertension over the past 12 months prior to screening.
    - Estimated glomerular filtration rate <60 mL/min/1.73m2.
    - Presence of severe depression as measured by Beck's Depression Inventory (BDI)-II >28 and/or a history of an untreated, unstable major affective disorder within 1 year of the screening visit.
    - Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID 19 requiring hospitalization within 6 months of enrollment.
    - Moderate to severe hepatic impairment.
    - History of drug and/or alcohol abuse.
    - History of or active hepatobiliary disease.
    - Liver enzymes (alanine aminotransferase (ALT)/aspartate aminotransferase (AST)) or total bilirubin >2 times the upper limit of normal (ULN).
    - Cortical cataract >/=one-quarter of the lens circumference (Grade cortical cataract [COR-2]) or a posterior subcapsular cataract >/=2 mm (Grade posterior subcapsular cataract [PSC-2]) at the time of screening.
    - Chronic regimen of potentially cataractogenic medications.
    - Initiation of chronic treatment for pain, or change in pain medication regimen, within 3 months prior to randomization.
    - Strong or moderate inducers or inhibitors of cytochrome P450 CYP3A per FDA classification within 14 days or 5 half lives, whichever is longer, prior to randomization.
    - Qualsiasi manifestazione della malattia di Fabry che precluda la somministrazione di placebo.
    - Anamnesi di attacco ischemico transitorio, ictus, infarto del miocardio, insufficienza cardiaca, chirurgia cardiovascolare maggiore, o trapianto renale.
    - Anamnesi di aritmia cardiaca clinicamente significativa in corso, o trattamento precedente o in corso per quanto sopra indicato.
    - Pazienti con epatite C,HIV, o infezioni di epatite B
    - Dolore neuropatico alle estremità superiori o inferiori, o dolore addominale che sia attribuibile a cause diverse dalla malattia di Fabry.
    - Anamnesi di crisi convulsive che attualmente richiedano un trattamento.
    - Ipertensione non controllata negli ultimi 12 mesi precedenti lo screening.
    - Velocità di filtrazione glomerulare stimata <60 ml/min/1,73 m2
    - Presenza di depressione grave misurata mediante classificazione della depressione di Beck (BDI)-II >28 e/o anamnesi di disturbo affettivo maggiore instabile non trattato entro 1 anno dalla visita di screening.
    - Positività al test del virus SARS-CoV-2 entro 2 settimane dall’arruolamento o COVID-19 che richiede il ricovero ospedaliero entro 6 mesi dall’arruolamento.
    - Insufficienza epatica da moderata a grave.
    - Anamnesi di abuso di droghe e/o alcol .
    - Anamnesi di malattia epatobiliare in passato o attiva.
    - Enzimi epatici (alanina aminotransferasi [ALT]/aspartato aminotransferasi [AST]) o bilirubina totale >2 volte il limite superiore della norma (ULN).
    - Cataratta corticale >/= un quarto della circonferenza del cristallino (grado della cataratta corticale [COR-2]) o una cataratta sottocapsulare posteriore >/=2 mm (grado di cataratta sottocapsulare posteriore [PSC-2]) al momento dello screening.
    - Regime cronico di farmaci potenzialmente catarattogenici
    - Inizio di trattamento cronico per il dolore, o variazione nel regime farmacologico antidolorifico, nei 3 mesi precedenti alla randomizzazione.
    - Induttori o inibitori forti o moderati del citocromo P450 CYP3A secondo la classificazione dell’ FDA entro 14 giorni o 5 emivite, a seconda di quale sia il periodo più lungo, prima della randomizzazione.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of responders in neuropathic or abdominal pain, as assessed by Fabry Disease Patient-Reported Outcome (FD-PRO), in patients treated with venglustat versus placebo
    Percentuale di responder al dolore neuropatico o addominale, valutato mediante il Fabry Disease Patient-Reported Outcome (FD-PRO), nei partecipanti trattati con venglustat rispetto al placebo
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline to 6 month and 12 months
    dal basale a 6 mesi e 12 mesi
    E.5.2Secondary end point(s)
    1 - Percent change in plasma globotriaosylsphingosine (lyso-GL-3)
    2 - Proportion of responders in tiredness component of FD-PRO
    3 - Change in the percentage of days with at least 1 stool reflecting diarrhea (Bristol Stool Form Scale [BSFS] Type 6 or 7)
    4 - Number of participants with adverse event (AE) and serious adverse event (SAE)
    5 - Change in lens clarity by ophthalmologic examination
    6 - Change in Beck Depression Inventory-II (BDI-II) score
    7 - Plasma venglustat concentrations at prespecified visits over the study duration
    8 - Maximum venglustat plasma concentration (Cmax)
    9 - Time to maximum venglustat plasma concentration (tmax)
    10 - Area under the venglustat plasma concentration versus time curve from time 0 to 24 hours (AUC0-24)
    1 -Variazione percentuale nel livello di liso-GL-3 nel plasma
    2- Percentuale di responder nella componente della stanchezza di FD-PRO
    3- Variazione nella percentuale di giorni con almeno 1 caso di feci che corrispondono a diarrea (BSFS di tipo 6 o 7)
    4- Numero di partecipanti con eventi avversi (AE) ed eventi avversi seri (SAE)
    5- Variazione nella trasparenza del cristallino mediante esame oftalmologico
    6- Variazione nel punteggio BDI-II
    7- Concentrazioni plasmatiche di venglustat in occasione di visite predefinite per tutta la durata dello studio
    8- Massima concentrazione plasmatica di venglustat (Cmax)
    9- Tempo della massima concentazione plasmatica di venglustat (tmax)
    10- Area sotto la concentrazione plasmatica di venglustat rispetto alla curva del tempo da 0 a 24 ore (AUC0-24)
    E.5.2.1Timepoint(s) of evaluation of this end point
    For 1 to 10: From baseline to 6 month and 12 months
    Da 1 a 10 : dal basale a 6 mesi e 12 mesi
    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 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) 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 concerned4
    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 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
    Argentina
    Austria
    Brazil
    Bulgaria
    Canada
    China
    Denmark
    Finland
    France
    Germany
    Greece
    Italy
    Mexico
    Netherlands
    Norway
    Poland
    Romania
    Russian Federation
    Turkey
    Ukraine
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months39
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months44
    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: 123
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 17
    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 state11
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 48
    F.4.2.2In the whole clinical trial 140
    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
    Nessuno
    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 Decision2021-10-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-04
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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