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    Summary
    EudraCT Number:2018-004670-10
    Sponsor's Protocol Code Number:AG10-333
    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:2021-05-24
    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 number2018-004670-10
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of AG10 in Subjects with Symptomatic Transthyretin Amyloid Polyneuropathy (ATTRibute-PN Trial)
    Studio di fase 3, randomizzato, in doppio cieco, controllato con placebo sull’efficacia e sulla sicurezza di AG10 in soggetti con polineuropatia amiloide da transtiretina sintomatica (ATTRibute-PN Trial)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of the Efficacy and Safety of AG10 in participants with Transthyretin Amyloid Polyneuropathy (ATTR-PN)
    Studio sull’efficacia e sulla sicurezza di AG10 in partecipanti con polineuropatia amiloide da transtiretina sintomatica (ATTR-PN)
    A.3.2Name or abbreviated title of the trial where available
    Efficacy and Safety of AG10 in participants with Transthyretin Amyloid Polyneuropathy (ATTR-PN)
    Efficacia e sicurezza di AG10 in partecipanti con polineuropatia amiloide da transtiretina sintomati
    A.4.1Sponsor's protocol code numberAG10-333
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/330/2018
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorEidos Therapeutics, 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 supportEidos Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEidos Therapeutics, Inc
    B.5.2Functional name of contact pointSr. Director, Clinical Operations
    B.5.3 Address:
    B.5.3.1Street Address101 Montgomery Street, Suite 2000
    B.5.3.2Town/ citySan Francisco, CA
    B.5.3.3Post code94104
    B.5.3.4CountryUnited States
    B.5.4Telephone number0014156513853
    B.5.5Fax number000000
    B.5.6E-mailmmcgovern@eidostx.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 numberEMA/OD/096/18
    D.3 Description of the IMP
    D.3.1Product nameAG10
    D.3.2Product code [AG10 HCl]
    D.3.4Pharmaceutical form Film-coated 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 INNAG10 HCl
    D.3.9.1CAS number 2242751-53-5
    D.3.9.2Current sponsor coden/a
    D.3.9.3Other descriptive name3-(3-(3,5-DIMETHYL-1H-PYRAZOL-4-YL)PROPOXY)-4-FLUOROBENZOIC ACID
    D.3.9.4EV Substance CodeSUB193376
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number400
    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 placeboFilm-coated tablet
    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
    Symptomatic Transthyretin Amyloid Polineuropathy (ATTR-PN)
    Polineuropatia amiloide da Transtiretina sintomatica (ATTR-PN)
    E.1.1.1Medical condition in easily understood language
    Symptomatic Transthyretin Amyloid Polineuropathy (ATTR-PN) is a rare disease that occurs when a protein, called transthyretin, is accumulated in the peripheral nerves.
    La polineuropatia amiloide sintomatica della transtiretina (ATTR-PN) è una malattia rara che si verifica quando una proteina, chiamata transtiretina, si accumula nei nervi periferici.
    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 10057949
    E.1.2Term Familial amyloid polyneuropathy
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the efficacy of AG10 in the treatment of subjects with symptomatic transthyretin amyloid polyneuropathy (ATTR-PN) by evaluating the difference between the AG10 and placebo groups in Modified Neuropathy Impairment Score + 7 (mNIS+7) at 18 months of treatment relative to baseline
    Determinare l’efficacia di AG10 nel trattamento di soggetti affetti da polineuropatia amiloide da transtiretina (ATTR-PN) sintomatica valutando la differenza tra i bracci AG10 e placebo nella Modified Neuropathy Impairment Score + 7 (mNIS+7) a 18 mesi di trattamento rispetto al basale
    E.2.2Secondary objectives of the trial
    To evaluate the effects of AG10 on:
    Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN), modified body mass index (mBMI), and Composite Autonomic Score (COMPASS-31) in subjects with symptomatic ATTR-PN
    Other Secondary:
    • To describe the pharmacodynamic (PD) properties of AG10 as assessed by serial measurements of an established assay of transthyretin (TTR) stabilization
    • To assess the pharmacodynamic effects of AG10 as assessed by circulating prealbumin (TTR) concentration as an in vivo biomarker of stabilization
    • To evaluate the effect of AG10 on health-related quality of life (QOL) and clinical outcome score as assessed by the Dyck/Rankin Score
    • To assess the effect of AG10 on gait speed as a reflection of functional ability
    Valutare gli effetti di AG10 su: Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN), indice di massa corporea modificato (mBMI) e Composite Autonomic Score (COMPASS-31) nei soggetti affetti da ATTR-PN sintomatica
    Altri secondari
    • Descrivere le proprietà farmacodinamiche (PD) di AG10, come valutate mediante le misurazioni seriali di un test consolidato di stabilizzazione della transtiretina (TTR)
    • Valutare gli effetti farmacodinamici di AG10, come valutati in base alla concentrazione di prealbumina (TTR) circolante come biomarcatore in vivo della stabilizzazione
    • Valutare l’effetto di AG10 sulla qualità della vita (QOL) correlata alla salute e sul punteggio degli esiti clinici come valutato mediante il Dyck/Rankin Score
    • Valutare l’effetto di AG10 sulla velocità dell’andatura come riflesso della capacità funzionale
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Be able to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures;
    2. Be male or female < o =18 to = o <90 years of age;
    3. Have Stage I or II symptoms (polyneuropathy disability [PND] =IIIa) of ATTR-PN and an established diagnosis of ATTR-PN as defined by physical exam findings and/or neurophysiological test findings consistent with the diagnosis of ATTR-PN;
    4. Have an NIS of 5 to 130 (inclusive) during screening;
    5. Have a nerve conduction studies (NCS) score [sum of the sural sensory nerve action potential (SNAP), tibial compound muscle action potential (CMAP), ulnar SNAP, ulnar CMAP, and peroneal CMAP] of < o =2 points during screening. NCS is a component of mNIS+7;
    6. Have a mutation consistent with ATTR-PN either documented in medical history or confirmed by genotyping obtained at Screening prior to randomization. *No genetic testing is needed for subjects who are recipients of domino liver transplants;
    7. Have an anticipated survival of < o =2 years, in the opinion of the Investigator;
    8. Have Karnofsky performance status < o =60 %;
    9. Have serum albumin levels >3.0 g/dL;
    10. Female subjects of childbearing potential who engage in heterosexual intercourse must agree to use a highly effective method of contraception beginning with randomization and continuing for 30 days after the last dose of AG10. A man who is sexually active with a woman of childbearing potential and has not had a vasectomy must agree to use a barrier method of birth control. Postmenopausal state female subjects must be confirmed with plasma follicle-stimulating hormone (FSH) at Screening.
    1. Essere in grado di comprendere e firmare un modulo di consenso informato scritto, che deve essere ottenuto prima dell’inizio delle procedure di studio;
    2. Essere soggetti di sesso maschile o femminile di età compresa tra < o =18 e= o <90 anni;
    3. Presentare sintomi di Stadio I o II (disabilità da polineuropatia [PND] =IIIa) di ATTR-PN e una diagnosi accertata di ATTR-PN come definita mediante i risultati dell’esame obiettivo e/o i risultati di test neurofisiologici coerenti con la diagnosi di ATTR-PN;
    4. Avere un punteggio NIS da 5 a 130 (compreso) durante lo screening;
    5. Avere un punteggio di studi sulla conduzione nervosa (NCS) (somma del potenziale d’azione della componente sensitiva del nervo surale [SNAP], del potenziale d’azione muscolare composto [CMAP] tibiale, dello SNAP ulnare, del CMAP ulnare e del CMAP peroneale) di < o =2 punti durante lo screening. NCS è un componente di mNIS+7;
    6. Presentare una mutazione coerente con ATTR-PN documentata nell’anamnesi oppure confermata mediante genotipizzazione ottenuta allo screening prima della randomizzazione. *Nessun test genetico è necessario per i soggetti che sono destinatari di trapianto di fegato domino;
    7. Avere una sopravvivenza prevista di < o =2 anni, a giudizio dello sperimentatore;
    8. Avere Karnofsky performance status < o =60%;
    9. Avere livelli sierici di albumina >3,0 g/dl;
    10. I soggetti di sesso femminile in età fertile che hanno rapporti eterosessuali devono acconsentire a utilizzare un metodo contraccettivo altamente efficace a partire dalla randomizzazione e fino a 30 giorni dopo l’ultima dose di AG10. Un uomo sessualmente attivo con una donna in età fertile e che non è stato sottoposto a vasectomia, deve accettare di utilizzare un metodo barriera di contraccezione. I soggetti di sesso femminile in stato postmenopausale devono essere confermati mediante l’ormone follicolo-stimolante (FSH) nel plasma allo screening.
    E.4Principal exclusion criteria
    1. Had a prior liver transplantation or is planning to undergo liver transplantation with a wild-type organ graft as treatment for symptomatic ATTR-PN during the study period. Note: Recipients of a
    "domino" liver transplant from an ATTR-PN donor who have developed ATTR-PN mediated by their graft are allowed under this protocol, as long as re-transplantation to treat ATTR-PN is not planned during the study period and meets all other eligibility criteria;
    2. Has sensorimotor or autonomic neuropathy not related to ATTR-PN; for example, autoimmune disease or monoclonal gammopathy, malignancy, or alcohol abuse;
    3. Has Vitamin B-12 levels below the lower limit of normal (LLN);
    4. Has clinical evidence of untreated hyper/hypothyroidism;
    5. Has leptomeningeal TTR amyloidosis;
    6. Has Type 1 diabetes;
    7. Has had Type 2 diabetes for =5 years;
    8. Has active hepatitis B or C or known human immunodeficiency virus (HIV) infection;
    9. Has NYHA heart failure classification >Class II;
    10. Had acute coronary syndrome, uncontrolled cardiac arrhythmia, or a stroke within 90 days prior to Screening;
    11. Has estimated glomerular filtration rate (eGFR) by Modification of Diet for Renal Disease (MDRD) formula <30 mL/min/1.73 m2 at Screening;
    12. Has abnormal liver function tests at Screening, defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3 × upper limit of normal (ULN) or total bilirubin >3 × ULN;
    13. Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated;
    14. Has known hypersensitivity to Investigational Medicinal Product
    (IMP) (AG10 or placebo), its metabolites, or formulation excipients;
    15. Is currently undergoing treatment for ATTR-PN with patisiran, inotersen, or other gene silencing agents, marketed drug products lacking a label indication for ATTR-PN (e.g., diflunisal, doxycycline), natural products or derivatives used as unproven therapies for ATTR-PN (e.g., green tea extract, tauroursodeoxycholic acid [TUDCA]/ursodiol), within 14 days, or 90 days for patisiran and 180 days for inotersen prior to dosing. Prior to screening, tafamidis, if already prescribed to potential subjects as part of their established background therapy, is allowed at the labeled dosage and administration of 20 mg/day for the treatment of ATTR-PN with, in the opinion of the Investigator, evidence of disease progression while on tafamidis treatment;
    16. Female subjects who are pregnant or breastfeeding. Breastfeeding females must agree to discontinue nursing before IMP is administered. A negative urine pregnancy test at Screening and on Day 1 prior to randomization are required for female subjects of childbearing potential;
    17. In the judgment of the Investigator or Medical Monitor, has any clinically important ongoing medical condition or laboratory abnormality or other condition that might jeopardize the subject's safety, increase their risk from participation, or interfere with the study;
    18. Participation in another investigational drug or investigational device study within 30 days prior to dosing or 5 half-lives of the investigational drug, whichever is longer, with potential residual effects that might confound the results of this study;
    19. Has any condition that, in the opinion of the Investigator or Medical Monitor, would preclude compliance with the study protocol such as a history of substance abuse, alcoholism, or a psychiatric condition.
    1. Aver avuto un precedente trapianto di fegato o aver pianificato un intervento di trapianto di fegato con un innesto d’organo wild-type come trattamento per ATTR-PN sintomatica durante il periodo dello studio.
    Nota: I destinatari di un trapianto di fegato “domino” da un donatore ATTR-PN che hanno sviluppato ATTR-PN mediata dall’innesto sono consentiti nel presente protocollo, purché il successivo trapianto per trattare ATTR-PN non sia pianificato durante il periodo dello studio ed essi/e soddisfino tutti gli altri criteri di idoneità;
    2. Neuropatia sensomotoria o autonomica non correlata a ATTR-PN; per esempio, malattia autoimmune o gammopatia monoclonale, neoplasia maligna o abuso di alcol;
    3. Avere i livelli di vitamina B-12 al di sotto del limite inferiore della norma ( (LLN);
    4. Avere evidenze cliniche di iper/ipotiroidismo non trattato;
    5. Amiloidosi leptomeningea (AL) da TTR;
    6. Diabete di tipo 1;
    7. Aver avuto diabete di tipo 2 per =5 anni;
    8. Epatite B o C attiva o infezione nota da virus dell’immunodeficienza umana (HIV);
    9. Classificazione di insufficienza cardiaca NYHA >Classe II;
    10. Aver avuto una sindrome coronarica acuta, un’aritmia cardiaca non controllata o un ictus nei 90 giorni precedenti lo screening;
    11. Velocità di filtrazione glomerulare stimata (eGFR) in base alla formula Modification of Diet for Renal Disease (MDRD) <30 ml/min/1,73 m2 allo screening;
    12. Anomalie dei test di funzionalità epatica allo screening, definite come alanina aminotransferasi (ALT) o aspartato aminotransferasi (AST) >3 × il limite superiore della norma (ULN) o bilirubina totale >3 × l’ULN;
    13. Aver avuto una neoplasia maligna negli ultimi 2 anni, ad eccezione del carcinoma della pelle a cellule basali o squamose o del carcinoma in situ della cervice che siano stati trattati con esito positivo;
    14. Presentare ipersensibilità nota al farmaco sperimentale (IMP) (AG10 o placebo), ai suoi metaboliti o agli eccipienti contenuti nella formulazione;
    15. Essere attualmente in trattamento per ATTR-PN con patisiran, inotersen o altri agenti di silenziamento genico, prodotti farmaceutici disponibili in commercio privi di indicazione in etichetta per il trattamento della ATTR-PN (per es., diflunisal, doxiciclina), prodotti naturali o derivati utilizzati come terapie non comprovate per ATTR-PN (per es., estratto di tè verde, acido tauroursodesossicolico [TUDCA]/ursodiolo), entro 14 giorni o 90 giorni per patisiran e 180 giorni per inotersen prima della somministrazione. Prima dello screening, tafamidis, se già prescritto ai potenziali soggetti nell’ambito della loro terapia di base consolidata, è consentito al dosaggio e alla somministrazione riportati in etichetta di 20 mg/giorno per il trattamento di ATTR-PN con, a giudizio dello sperimentatore, evidenze di progressione della malattia durante il trattamento con tafamidis;
    16. Soggetti di sesso femminile in gravidanza o allattamento. Le donne in allattamento devono acconsentire a interrompere l’allattamento prima della somministrazione dell’IMP. Ai soggetti di sesso femminile in età fertile è richiesto un test di gravidanza sulle urine negativo allo screening e il Giorno 1 prima della randomizzazione;
    17. A giudizio dello sperimentatore o del Medical Monitor, qualsiasi patologia medica in corso di rilevanza clinica o anomalia di laboratorio o altra patologia che possa mettere a rischio la sicurezza del soggetto, aumentare il rischio associato alla partecipazione o interferire con lo studio;
    18. Partecipazione, entro 30 giorni prima della somministrazione o 5 emivite del farmaco sperimentale, adottando il periodo più lungo, a un altro studio su un farmaco sperimentale o un dispositivo sperimentale con potenziali effetti residui che potrebbero confondere i risultati di questo studio;
    19. Qualsiasi condizione che, a giudizio dello sperimentatore o del Medical Monitor, potrebbe impedire la conformità al protocollo dello studio, come un’anamnesi di abuso di sostanze, alcolismo ocondiz psichiat
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to Month 18 in mNIS+7 score
    Variazione al Mese 18 di trattamento rispetto al basale nel mNIS+7
    E.5.1.1Timepoint(s) of evaluation of this end point
    baseline, month 9 and month 18
    Basale, mese 9 e mese 18
    E.5.2Secondary end point(s)
    1. Change from baseline to Month 18 in Norfolk QOL-DN
    2. Change from baseline to Month 18 in mBMI,
    3. Change from baseline to Month 18 in prealbumin levels
    4. Change from baseline to Month 18 in COMPASS-31
    5. Change from baseline to Month 18 of treatment in PD endpoints using established assay of TTR stabilization including Fluorescent Probe Exclusion (FPE) assay.
    6. Change from baseline to Month 18 of treatment in Dyck/Rankin score at each assessed time point.
    7. Change from baseline to Month 18 of treatment in gait speed.
    1. Variazione al Mese 18 rispetto al basale nei Norfolk QOL-DN
    2. Variazione al Mese 18 rispetto al basale nel mBMI
    3. Variazione al Mese 18 rispetto al basale nei livelli di prealbumina
    4. Variazione al Mese 18 rispetto al basale nel COMPASS-31
    5. Variazione al Mese 18 di trattamento rispetto al basale negli endpoint PD mediante un test consolidato della stabilizzazione di TTR, tra cui il test Fluorescent Probe Exclusion (FPE)
    6. Variazione rispetto al basale al Mese 18 di trattamento nel Dyck/Rankin Score ad ogni punto temporale valutato.
    7. Variazione rispetto al basale al Mese 18 di trattamento nella velocità dell’andatura
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Baseline, 9 and 18 months
    2. Baseline, 9 and 18 months
    3. Baseline, 3, 6, 9, 13, 15 and 18 months
    4. Baseline, 9 and 18 months
    5. Baseline, 9 and 18 months
    6. Baseline, 9 and 18 months
    7. Baseline, 9 and 18 months
    1. Basale, 9 e 18 mesi
    2. Basale, 9 e 18 mesi

    3. Basale, 3, 6, 9, 13, 15 e 18 mesi
    4. Basale, 9 e 18 mesi
    5. Basale, 9 e 18 mesi
    6. Basale, 9 e 18 mesi
    7. Basale, 9 e 18 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 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) 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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    Brazil
    Canada
    Mexico
    New Zealand
    Russian Federation
    Taiwan
    Turkey
    Bulgaria
    France
    Germany
    Hungary
    Italy
    Netherlands
    Poland
    Portugal
    Spain
    Argentina
    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 years3
    E.8.9.1In the Member State concerned months7
    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 months7
    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: 87
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 58
    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 state35
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 145
    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)
    All subjects who complete the double-blinded treatment period may be eligible to participate in an open label extension study of long-term AG10 treatment
    Tutti i soggetti che completano il periodo di trattamento in doppio cieco possono essere ammessi a partecipare a uno studio di estensione in aperto del trattamento AG10 a lungo termine
    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 Decision2020-07-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-22
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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