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    Summary
    EudraCT Number:2017-004084-12
    Sponsor's Protocol Code Number:EFC15392
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-06-17
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2017-004084-12
    A.3Full title of the trial
    Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)
    Studio multicentrico, randomizzato, in doppio cieco, controllato con placebo, in due stadi, volto a caratterizzare l¿efficacia, la sicurezza, la tollerabilit¿ e la farmacocinetica di GZ/SAR402671 in pazienti affetti da Rene Policistico Autosomico Dominante (ADPKD) a rischio di rapida progressione.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)
    Studio di Efficacia, Sicurezza, Tollerabilit¿ e Farmacocinetica di GZ/SAR402671 in pazienti affetti da Rene Policistico Autosomico Dominante (ADPKD) a rischio di rapida progressione.
    A.3.2Name or abbreviated title of the trial where available
    SAVE-PKD
    SAVE-PKD
    A.4.1Sponsor's protocol code numberEFC15392
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1202-0775
    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 SponsorGENZYME CORPORATION
    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 supportGenzyme Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanofi S.p.A.
    B.5.2Functional name of contact pointCONTACT POINT
    B.5.3 Address:
    B.5.3.1Street AddressVIALE L. BODIO 37/B
    B.5.3.2Town/ cityMILANO
    B.5.3.3Post code20158
    B.5.3.4CountryItaly
    B.5.4Telephone number800226343
    B.5.5Fax number0239394168
    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/3/18/2122
    D.3 Description of the IMP
    D.3.1Product nameVenglustat
    D.3.2Product code GZ/SAR402671
    D.3.4Pharmaceutical form Capsule, hard
    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
    D.3.9.1CAS number 1401090-53-6
    D.3.9.2Current sponsor codeGZ/SAR402671
    D.3.9.3Other descriptive nameGenz-682452-AU
    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.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 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/3/18/2122
    D.3 Description of the IMP
    D.3.1Product nameVenglustat
    D.3.2Product code GZ/SAR402671
    D.3.4Pharmaceutical form Capsule, hard
    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
    D.3.9.1CAS number 1401090-53-6
    D.3.9.2Current sponsor codeGZ/SAR402671
    D.3.9.3Other descriptive nameGenz-682452-AU
    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 number4
    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 placeboCapsule, hard
    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
    Congenital, hereditary and neonatal diseases
    malattie neonatali, ereditarie e congenite
    E.1.1.1Medical condition in easily understood language
    Congenital, hereditary and neonatal diseases
    malattie neonatali, ereditarie e congenite
    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 LLT
    E.1.2Classification code 10036046
    E.1.2Term Polycystic kidney, autosomal dominant
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the effect of venglustat on the rate of total kidney volume (TKV) growth (Stage 1) and estimated glomerular filtration rate (eGFR) decline in patients at risk of rapidly progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) (Stage 2).
    Determinare l'effetto di venglustat sull'aumento del volume renale totale (TKV) (Stadio 1) e sulla riduzione dell'eGFR in pazienti a rischio di rapida progressione dell'ADPKD (Rene Policistico Autosomico Dominante) (Stadio 2).
    E.2.2Secondary objectives of the trial
    -To determine the effect of venglustat on the rate of renal function decline (Stage 1) and on the rate of TKV growth (Stage 2).
    -To evaluate the pharmacokinetics (PK) of venglustat in Autosomal Dominant Polycystic Kidney Disease patients (Stages 1 and 2).
    - To determine the effect of venglustat on pain and fatigue, based on patient reported diary (Stages 1 and 2).
    -Safety/tolerability objective:
    -To characterize the safety profile of venglustat (Stages 1 and 2).
    -To evaluate the effect of venglustat on mood using Beck Depression Inventory II (BDI-II) (Stages 1 and 2).
    -To evaluate the effect of venglustat on the lens by ophthalmological examination (Stages 1 and 2).
    -Determinare l'effetto di venglustat sul tasso di riduzione della funzionalità renale (Stadio 1) e sull’aumento del volume renale totale TKV (Stadio 2).
    -Valutare la farmacocinetica (PK) di venglustat nei pazienti con ADPKD (Stadi 1 e 2).
    -Determinare l’effetto di venglustat su dolore e affaticamento, in base al diario riferito dal/dalla paziente (Stadi 1 e 2).
    - Obiettivo sicurezza / tollerabilità:
    - Caratterizzare il profilo di sicurezza di venglustat (Stadi 1 e 2).
    - Valutare l'effetto di venglustat sull'umore utilizzando il questionario di Beck sulla depressione (BDI-II) (Stadi 1 e 2).
    - Valutare l'effetto di GZ / SAR402671 sul cristallino mediante esame oftalmologico (Stadi 1 e 2).
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Other types of substudies
    Specify title, date and version of each substudy with relative objectives: Measured Glomerular Filtration Rate Substudy
    The main objective is to determine the effect of GZ/SAR402671 on
    mGFR.

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Tasso misurato di filtrazione glomerulare
    L'obiettivo principale ¿ determinare l'effetto di GZ / SAR402671 su mGFR.
    Non condotto in Italia

    E.3Principal inclusion criteria
    - Male or female adult with ADPKD with age at the time the consent is signed:
    a) between 18 to 50 years (both inclusive) for patients from Stage 1
    b) between 18 to 50 years (both inclusive) for patients from Stage 2 with eGFR between 45 and 89.9 mL/min/1.73 m2 during screening period*
    c) between 18 to 55 years (both inclusive) for patients from Stage 2 with eGFR between 30 and 44.9 mL/min/1.73 m2 during screening period*
    Diagnosis of ADPKD in patients with a family history, will be based on unified Pei criteria.
    In the absence of a family history, the diagnosis will be based on the presence of renal cysts bilaterally, totaling at least 20, in the absence of findings suggestive of other cystic renal diseases.
    - Mayo Imaging Classification of ADPKD Class 1C, 1D or 1E**.
    ** TKV volume must be confirmed by a central reader prior to Visit 3
    - Estimated glomerular filtration rate between 45 to 89.9 mL/min/1.73 m2 during screening period* (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] for Stage 1.
    Estimated glomerular filtration rate between 30 to 89.9 mL/min/1.73 m2 during screening period* (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]) for Stage 2.
    *Eligibility will be confirmed by eGFR value from one of the two first prerandomization eGFR measurements.
    - Stable treatment regimen of antihypertensive therapy for at least 30 days prior to the screening visit for hypertensive patient.
    - Able to read, comprehend and respond to the study questionnaires.
    - Patient has given voluntary written informed consent before performance of any study related procedures not part of standard medical care.
    - Patient does not have access to tolvaptan at the time of study start or tolvaptan is not indicated for treatment of patient according to treating physician (patient does not meet recommended criteria for treatment, refuses to initiate or does not tolerate treatment with tolvaptan).
    -The patient, if female of childbearing potential, must have a negative blood pregnancy test [beta human chorionic gonadotropin (ß-hCG)] at the screening visit and a negative urine pregnancy test at the baseline
    visit.
    - Female patients of childbearing potential and male patients must agree to practice true abstinence in line with their preferred and usual lifestyle or to use double-contraceptive methods (including a highly effective method of contraception for female participants of childbearing potential) for the entire duration of the study and for at least 6 weeks for females and 90 days for males following their last dose of study drug.
    - Pazienti di sesso maschile o femminile con ADPKD con un’ età alla firma del consenso:
    a) compresa tra i 18 e 50 anni (inclusi) per i pazienti dello Stadio 1.
    b) compresa tra 18 e 50 anni (inclusi) per i pazienti dello Stadio 2 con un valore di eGFR durante il periodo di screening compreso tra 45 e 89.9 mL/min/1.73 m2*.
    c) compresa ta 18 e 55 anni (inclusi) per i pazienti dello Stadio 2 con un valore di eGFR durante il periodo di screening compreso tra 30 e 44.9 mL/min/1.73 m2*.
    La diagnosi di ADPK in pazienti con familiarità si baserà sui criteri Pei unificati. In assenza di familiarità, la
    diagnosi si baserà sulla presenza di un totale di almeno 20 cisti renali bilaterali, in assenza di altri riscontri che suggeriscano altre malattie renali cistiche.
    - Stadio 1C, 1D o 1E secondo la classificazione di imaging Mayo dell’ADPKD **
    ** Il volume TKV deve essere confermato dal revisore centrale prima della Visita 3
    - Filtrazione glomerulare stimata tra 45 e 89.9 ml/min/1,73 m2 durante il periodo di screening* (Chronic Kidney Disease Epidemiology Collaboration [equazione CKD-EPI]) per lo Stadio 1.
    Filtrazione glomerulare stimata tra 30 e 89.9 ml/min/1,73 m2 durante il periodo di screening* (Chronic Kidney Disease Epidemiology Collaboration [equazione CKD-EPI]) per lo Stadio 2.
    *L’eleggibilità sarà confermata dal valore di eGFR ottenuto in una delle due valutazioni pre-randomizzazione
    - Terapia antipertensiva stabile per almeno 30 giorni prima della visita di screening per i pazienti ipertesi.
    - Pazienti in grado di leggere, comprendere e rispondere ai questionari di studio.
    - Pazienti che hanno fornito volontariamente il loro consenso prima che venga eseguita qualsiasi procedura di studio, non facente parte della normale pratica clinica.
    - Il paziente non ha accesso a tolvaptan al momento dell'inizio dello studio o per i quali il trattamento con tolvaptan non è indicato secondo la valutazione del medico curante (il paziente non soddisfa i criteri raccomandati per il trattamento o non tollera il trattamento con tolvaptan).
    - Paziente donna potenzialmente fertile, deve avere un risultato negativo al test di gravidanza sul siero [gonadotropina corionica beta (ß-hCG)] alla visita di screening e un test di gravidanza sulle urine negativo alla visita basale.
    - Pazienti in età fertile e pazienti maschi dovranno accettare di praticare astinenza in linea con il loro stile di vita preferito e abituale o utilizzare 2 accettabili metodi di contraccezione (incluso un metodo ontraccettivo altamente efficace per le donne in età fertile) per l'intera durata dello studio e per almeno 6 settimane per le femmine e 90 giorni per i maschi dopo l'ultima dose di farmaco in studio.
    E.4Principal exclusion criteria
    -Systolic blood pressure >160 mm Hg at Run-in and Baseline visits
    -Administration within 3 months prior to the screening visit of tolvaptan or other Polycystic Kidney Disease-modifying agents (somatostatin analogues).
    -Current participation in another investigational interventional study or use of investigational medicinal product (IMP), within 3 months or 5 half lives, whichever is longer, before randomization.
    - The patient has a positive result of any of the following tests: hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (anti HCV) antibodies, anti-human immunodeficiency virus 1 and 2 antibodies (anti HIV1 and anti HIV2 Ab). Patients with a positive hepatitis B surface antibody (HBsAb) test are eligible if other criteria are met (ie, negative tests for: HBsAg, hepatitis B core antibody [HBcAb]). Patients immune due to natural infection (positive hepatitis B surface antibody (HBsAb), negative hepatitis B surface antigen (HBsAg) and positive hepatitis B core antibody [HBcAb]) are eligible if they have negative HBV DNA test.

    - A history of drug and/or alcohol abuse within the past year prior to the screening visit. A history of alcohol dependence within the 5 years prior to the screening visit.
    -The patient is scheduled for in-patient hospitalization including elective surgery, during the study.
    -The patient has a clinically significant, uncontrolled medical condition that, in the opinion of the investigator, would put the safety of the subject at risk through participation, or which would affect the efficacy or safety analysis if the condition exacerbated during the study, or that may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.
    -The patient, in the opinion of the investigator, is unable to adhere to the requirements of the study or unable to undergo study assessments (eg, has contraindications to pupillary dilation or unable to undergo magnetic resonance imaging (MRI ) [For example: patient’s weight exceeds weight capacity of the MRI, ferromagnetic metal prostheses, aneurysm clips, severe claustrophobia, large abdominal/back tattoos, etc]).
    -Any country-related specific regulation that would prevent the patient from entering the study.
    -The patients did not adhere to treatment (<70% compliance rate) in the run-in
    -The patient has, according to World Health Organization (WHO) Grading, a cortical cataract >onequarter of the lens circumference (Grade cortical cataract-2 [COR-2]) or a posterior subcapsular cataract >2 mm (Grade posterior subcapsular cataract-2 [PSC-2]). Patients with nuclear cataracts will not be excluded.
    -The patient is currently receiving potentially cataractogenic medications, including a chronic regimen (more frequently than every 2 weeks) of any route of corticosteroids (including medium and high potency topical steroids) or any medication that may cause cataract, according to the Prescribing Information
    -The patient has received strong or moderate inducers or inhibitors of CYP3A4 within 14 days or 5 half-lives, whichever is longer, prior to randomization. This also includes the consumption of grapefruit, grapefruit juice, or grapefruit containing products within 72 hours of starting GZ/SAR402671 administration
    -The patient is pregnant, or lactating
    -Liver enzymes (alanine aminotransferase [ALT]/aspartate aminotransferase [AST]) or total bilirubin >2 times the upper limit of normal unless the patient has the diagnosis of Gilbert syndrome. Patients with the Gilbert syndrome should have no additional symptoms or signs which suggest hepatobiliary disease and serum total bilirubin level no more than 3mg/dl (51 µmol/L) with conjugated bilirubin less than 20% of the total bilirubin fraction
    -Presence of severe depression as measured by beck depression inventory-II (BDI-II) >28 and/or a history of a major affective disorder within 1 year of the screening visit
    -Known hypersensitivity to GZ/SAR402671 or any component of the excipients
    -PA sistolica>160 mmHg durante la fase di run-in e al basale
    -Assunz. nei 3mesi precedenti la visita di screening di tolvaptan o altri agenti in grado di modificare la malattia policistica renale(analoghi della somatostatina)
    -Partecipazione ad un altro studio o uso di un IMP, entro 3 mesi o 5 emivite, a seconda di quale dei due duri più a lungo, prima della randomizzazione
    -Il pz ha un risultato positivo di uno dei seguenti test: antigene di superficie dell'epatite B(HBsAg), anticorpi anti-virus dell'epatite C(anti HCV),anticorpi anti-virus dell'immunodeficienza umana 1 e 2(anti HIV1 e anti HIV2 Ab).I pz con test anticorpale di superficie positivo per l'epatite B(HBsAb)sono eleggibili se vengono soddisfatti altri criteri(ad es,test negativi per HBsAg,anticorpo core per epatite B).I pz immuni a causa di infezione naturale(anticorpo di superficie dell'epatite B positiva,antigene di superficie dell'epatite B negativa(HBsAg)e anticorpo di nucleo dell'epatite B positiva HBcAb sono eleggibili se hanno test del DNA dell'HBV negativo
    -Storia di abuso di droghe e/o alcol nell'ultimo aa prima della visita di screening. Anamnesi di abuso di alcol entro 5 aa prima della visita di screening
    -Al pz è stato programmato un ricovero in ospedale inclusi ricoveri per chirurgia elettiva,durante lo studio
    -Il pz presenta una condizione medica clinicamente significativa e incontrollata che, con la partecipazione allo studio, secondo l'opinione del PI, metterebbe a rischio la sua sicurezza o che influenzerebbe l'efficacia o l'analisi della sicurezza se la condizione si aggravasse durante lo studio, o che possono interferire in modo significativo con l’aderenza allo studio, comprese tutte le valutazioni prescritte e le attività di follow-up
    -Il pz secondo l’opinione del PI, non è in grado di aderire ai requisiti dello studio o non in grado di sottoporsi a valutazioni dello studio (es.controindicazioni alla dilatazione pupillare o non è in grado di sottoporsi a RM) (ad es:peso del pz superiore alla capacità della RM, protesi metallici ferromagnetici, clip per aneurisma, claustrofobia grave, grandi tatuaggi sull’addome o sulla schiena)
    -Qualsiasi specifico regolamento relativo al paese che impedirebbe al pz di entrare nello studio
    -pz che non sono aderenti al trattamento (<70% compliance) durante il run-in
    - Secondo la classificazione OMS, il pz presenta una cataratta corticale>1 quarto della circonferenza del cristallino (Grado2 della cataratta corticale [COR-2]) o una cataratta sottocapsulare posteriore>2 mm (Grado2 della cataratta sottocapsulare posteriore [PSC-2]). I pz con cataratta nucleare non saranno esclusi
    -pz è in trattamento con farmaci potenzialmente catarattogenici,compreso un regime cronico con corticosteroidi(con freq>a ogni 2 sett)mediante qualsiasi via di somministrazione(inclusi gli steroidi topici a medio ed elevato potenziale)o di qualsiasi farmaco che possa provocare cataratta,in base alle info sulla prescriz.
    -pz ha ricevuto induttori o inibitori,potenti o moderati,dell’enzima CYP3A4 nei 14 gg precedenti la randomizzazione o nel lasso di tempo corrispondente a 5 emivite,a seconda di quale periodo duri di più,incluso il consumo di pompelmo, succo di pompelmo o prodotti contenenti pompelmo entro 72h dall’inizio della somministrazione di GZ/SAR402671
    -pz è in stato di gravidanza o in allattamento
    -Enzimi epatici ([ALT]/[AST]) o bilirubina totale con valori>2 volte il limite superiore di normalità a meno che il pz non abbia una diagnosi di sindrome di Gilbert. I pz con sindrome di Gilbert non devono presentare ulteriori sintomi o segni che suggeriscono una malattia epatobiliare e un livello sierico di bilirubina tot non superiore a 3mg/dl (51µmol/L) con bilirubina coniugata inferiore al 20%della frazione totale di bilirubina
    -Presenza di depressione grave misurata dalla BDI-II>28 e/o storia di disturbo affettivo maggiore entro 1 aa dalla visita di screening
    -Ipersensibilità nota a GZ/SAR402671 o a qualsiasi eccipiente che lo compone
    E.5 End points
    E.5.1Primary end point(s)
    1. Annualized rate of change in total kidney volume (TKV) based on magnetic resonance Imaging (MRI) from baseline to 18 months (Stage 1) 2. Annualized rate of change in estimated glomerular filtration rate (eGFR) (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] equation) from baseline to 24 months (Stage 2)
    1. Tasso annuale di variazione del volume totale renale (TKV) valutato mediante RM dal basale ai 18 mesi (Stadio 1). 2. Tasso annuale di variazione della filtrazione glomerulare (eGFR) (equazione CKD-EPI) dal basale fino a 24 mesi (Stadio 2)
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. From baseline to 18 months 2. From baseline to 24 months
    1. Dal Basale a 18 mesi 2. Dal Basale a 24 mesi
    E.5.2Secondary end point(s)
    1.Annualized rate of change in eGFR (CKD-EPI equation) from baseline to 18 months (Stage 1)
    2.Annualized rate of change in TKV based on MRI from baseline to 18 months (Stage 2)
    3.Change in Brief Pain Inventory (BPI) - Item 3 from the daily symptom diary
    4.Change in Brief Fatigue Inventory (BFI) - Item 3 from the daily symptom diary
    5.Number of adverse events (Stages 1 and 2)
    6. Change in score of BDI-II (Stages 1 and 2)
    7.Change in the lens clarity from baseline by ophthalmological examination (Stages 1 and 2)
    8.Assessment of single time-point plasma concentration (Stages 1 and 2)
    1. Tasso annuale di variazione di eGFR (equazione CKD-EPI) dal basale a 18 mesi (Stadio 1)
    2. Tasso annuale di variazione di TKV valutato mediante RM dal basale a 18 mesi (Stadio 2)
    3. Variazione del dolore (BPI - voce 3) dal diario giornaliero dei sintomi.
    4. Variazione dell’affaticamento (BFI - voce 3) dal diario giornaliero dei sintomi
    5. Numero di eventi avversi (Stadio 1 e 2)
    6. Variazione del punteggio di BDI-II (Stadio 1 e 2)
    7. Variazione della opacizzazione del cristallino rispetto al basale attraverso un esame oftalmologico (Stadio 1 e 2)
    8. Valutazione della concentrazione plasmatica a singolo punto di tempo (Stadio 1 e 2)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. 2. :
    From baseline to 18 months
    3. 4. :
    Stage 1: From baseline to 18 months
    Stage 2: From baseline to 24 months
    5. 6. 7. :
    From baseline to end of treatment +30 days
    8. :
    Stage 1: Day 1, Months 1, 6, and 18
    Stage 2: Months 6 and 24
    1. 2. :
    Dal basale a 18 mesi
    3. 4. :
    Stadio 1: Dal basale a 18 mesi
    Stadio 2: Dal basale a 24 mesi
    5. 6. 7. :
    Dal basale al termine del trattamento + 30 giorni
    8. :
    Stadio 1: Giorno 1, Mesi 1, 6, and 18
    Stadio 2: Mesi 6 e 24
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 EEA33
    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
    China
    Japan
    Korea, Republic of
    Taiwan
    United States
    Austria
    Belgium
    Denmark
    France
    Germany
    Italy
    Netherlands
    Portugal
    Romania
    Spain
    United Kingdom
    Czechia
    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 years4
    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 years4
    E.8.9.2In all countries concerned by the trial months2
    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: 982
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 394
    F.4.2.2In the whole clinical trial 982
    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)
    Patients who completed 24 months of treatment in EFC15392 study may be given the option to enroll into a planned open-label long-term extension study (additional 24 months of treatment with venglustat).
    Ai pazienti che completano 24 mesi di trattamento nello studio EFC15392 potrebbe essere offerta la possibilità di partecipare a un potenziale studio di estensione a lungo termine in aperto (24 mesi addizionali di trattamento con venglustat).
    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 Decision2018-06-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-07-12
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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