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    Summary
    EudraCT Number:2017-003369-85
    Sponsor's Protocol Code Number:ID-069A301
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-10-07
    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 number2017-003369-85
    A.3Full title of the trial
    A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease
    Studio multicentrico, in doppio cieco, randomizzato, controllato con placebo, a gruppi paralleli per la valutazione dell’efficacia e della sicurezza di Lucerastat in monoterapia orale in soggetti adulti con malattia di Fabry
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study to study the effects of a new oral drug called lucerastat in adults with Fabry disease
    Uno studio clinico per studiare gli effetti di un nuovo farmaco orale chiamato lucerastat in soggetti adulti con malattia di Fabry
    A.3.2Name or abbreviated title of the trial where available
    MODIFY
    MODIFY
    A.4.1Sponsor's protocol code numberID-069A301
    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 SponsorIDORSIA PHARMACEUTICALS LTD
    B.1.3.4CountrySwitzerland
    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 supportIdorsia Pharmaceuticals Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIdorsia Pharmaceuticals Ltd
    B.5.2Functional name of contact pointClinical Trial Disclosure Desk
    B.5.3 Address:
    B.5.3.1Street AddressHegenheimermattweg 91
    B.5.3.2Town/ cityAllschwil
    B.5.3.3Post code4123
    B.5.3.4CountrySwitzerland
    B.5.6E-mailclinical-trials-disclosure@idorsia.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/3/12/1033; EMA/OD/042/12
    D.3 Description of the IMP
    D.3.1Product nameLucerastat
    D.3.2Product code [ACT-434964]
    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 INNLucerastat
    D.3.9.2Current sponsor codeACT-434964
    D.3.9.3Other descriptive nameOGT923
    D.3.9.4EV Substance CodeSUB189593
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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
    Fabry disease
    Malattia di Fabry
    E.1.1.1Medical condition in easily understood language
    Fabry disease is a rare, inherited disease caused by a genetic defect which leads to an accumulation of fatty substance in the body. Symptoms include stomach pain and damage to vital organs
    La malattia di Fabry è una patologia rara, ereditaria, causata da un difetto genetico che porta a accumulare sostanze grasse nel corpo. I sintomi includono dolore addominale e danni agli organi vitali
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    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 determine the effect of lucerastat on neuropathic pain in subjects with Fabry disease (FD).
    Valutare gli effetti di Lucerastat sul dolore neuropatico in soggetti con la malattia di Fabry (FD).
    E.2.2Secondary objectives of the trial
    • To determine the effects of lucerastat on gastro-intestinal (GI) symptoms (abdominal pain and diarrhea) in subjects with FD and GI symptom(s) at baseline.
    • To confirm the effect of lucerastat on biomarkers of FD.
    • To determine the safety and tolerability of lucerastat in subjects with FD.
    • Valutare gli effetti di Lucerastat sui sintomi gastrointestinali (GI) (dolore addominale e diarrea) in soggetti con FD e i sintomi GI al baseline;
    • Confermare gli effetti di Lucerastat sui biomarcatori della FD;
    • Valutare la sicurezza e la tollerabilità di Lucerastat in soggetti con FD.
    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: PK profile sub-study (all sites): A sub-study will be conducted at all sites to determine the PK profile of lucerastat at steady state in a subgroup of approximately 36 subjects with FD with either normal or subnormal renal function (i.e., screening eGFR = 60 mL/min/1.73 m2) or impaired renal function (i.e., screening eGFR < 60 mL/min/1.73 m2). If necessary, blood samples collected for PK may be used for the identification of potential metabolites of lucerastat in this population.
    Subjects consenting to this sub study will have a 12-hour PK profile performed at the Month 1 visit.

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Sarà condotto un sottostudio in tutti i centri per determinare il profilo di farmacocinetica di Lucerastat a uno stadio stabile in un sottogruppo di circa 36 soggetti con Malattia di Fabry sia con funzione renale normale che anormale (es. eGFR di screening = 60 mL/min/1,73 m2) o funzione renale ridotta (es. eGFR di screening > 60 mL/min/1,73 m2). Se necessario, i campioni di sangue raccolti per la farmacocinetica possono essere usati per l’identificazione dei metaboliti di Lucerastat in questa popolazione.
    I soggetti che acconsentono a questo sottostudio avranno un profilo di farmacocinetica di 12 ore alla visita del Mese 1.
    E.3Principal inclusion criteria
    1. Signed and dated ICF prior to any study-mandated procedure.
    2. Male or female subjects; 18-years old and above.
    3. FD diagnosis confirmed with local genetic test results (i.e., presence of at least 1 mutation in GLA, the gene coding for a-
    galactosidase A).
    4. Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening.
    1. ICF sottoscritto e datato prima di qualsiasi procedura richiesta dallo studio.
    2. Soggetti di sesso maschile e femminile di età pari o superiore ai 18 anni.
    3. Diagnosi di FD confermata con i risultati dei test genetici locali (es. presenza di almeno 1 mutazione in GLA, la codifica del gene per a galattosidasi A [a-GalA]).
    4. Dolore neuropatico associato alla malattia di Fabry, come definito dal soggetto, negli ultimi 3 mesi prima dello screening.
    E.4Principal exclusion criteria
    1. Pregnant, planning to be become pregnant up to 30 days after study treatment discontinuation or lactating subject;
    2. Severe renal insufficiency defined as an estimated glomerular filtration rate (eGFR) per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 30 mL/min/1.73 m2 at screening;
    3. Subject on regular dialysis for the treatment of chronic kidney disease;
    4. Subject has undergone, or is on a waiting list for, or is scheduled to undergo kidney or other organ transplantation;
    5. Known and documented transient ischemic attack, stroke, unstable angina or myocardial infarction within 6 months prior to screening;
    6. Clinically significant unstable cardiac disease in the opinion of the investigator (e.g., uncontrolled symptomatic arrhythmia, New York Heart Association class III or IV congestive heart failure);
    7. Any other subject at high risk for developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
    8. Subject planned for imminent initiation of treatment with ERT.
    1. In gravidanza/in gravidanza programmata fino a 30 giorni dopo l’interruzione del trattamento di studio o soggetto in allattamento;
    2. Insufficienza renale acuta definita come velocità di filtrazione glomerulare (eGFR) stimata mediante l’equazione della creatinina secondo la Chronic Kidney Disease Epidemiology Collaboration < 30 mL/min/1,73 m2 allo screening (come riportato dal laboratorio centrale);
    3. Soggetto sottoposti regolarmente a dialisi per il trattamento dell’insufficienza renale cronica;
    4. Il soggetto è stato sottoposto, o è in lista di attesa, o ha un trapianto programmato di reni o altri organi;
    5. Attacco ischemico transitorio noto e documentato, ictus, angina instabile o infarto del miocardio nei 6 mesi prima dello screening;
    6. Malattia cardiaca instabile clinicamente rilevante secondo lo sperimentatore (es. aritmia sintomatica instabile, insufficienza cardiaca congestizia di classe III o IV secondo la New York Heart Association);
    7. Ogni altro soggetto ad alto rischio di sviluppo di segni clinici di coinvolgimento di organi nel periodo di durata dello studio, secondo il giudizio dello sperimentatore;
    8. Il soggetto ha programmato di iniziare un trattamento con terapia enzimatica sostitutiva (ERT) nell’imminenza.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is a response to study treatment on neuropathic pain, defined as a reduction from baseline to Month 6 of at least 30% in the "modified" Brief Pain Inventory-Short Form item 3 (BPI-SF3) score of "neuropathic pain at its worst in the last 24 hours".
    L’endpoint primario di efficacia è una risposta al trattamento di studio del dolore neuropatico, definito come riduzione dal baseline al Mese 6 di almeno il 30% nel risultato “modificato” BPI-SF3 del “dolore neuropatico al suo punto massimo nelle ultime 24 ore”.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline to Month 6
    Dal baseliine al Mese 6
    E.5.2Secondary end point(s)
    • Change from baseline to Month 6 in the average daily 11-point Numerical Rating Scale (NRS-11) score of "abdominal pain at its worst in the last 24 hours" in subjects with GI symptoms at baseline.
    • Change from baseline to Month 6 in the number of days with at least one stool of a Bristol Stool Scale (BSS) consistency Type 6 or 7 in subjects with GI symptoms at baseline.
    • Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3).
    • Variazioni dal baseline al Mese 6 nel risultato della scala di valutazione numerica media quotidiana a 11-punti (NRS-11) di “dolore addominale al suo punto massimo nelle ultime 24 ore” in soggetti con sintomi GI al baseline.
    • Variazioni dal baseline al Mese 6 nel numero di giorni con almeno un episodio di feci, secondo la Bristol Stool Scale (BSS), di consistenza di Tipo 6 o 7 in soggetti con sintomi GI al baseline;
    • Variazioni dal baseline al Mese 6 di globotriaosilceramide (Gb3) nel plasma.
    E.5.2.1Timepoint(s) of evaluation of this end point
    From baseline to Month 6
    Dal baseline al Mese 6
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    Tollerabilità
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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
    Austria
    Belgium
    Canada
    Finland
    Germany
    Ireland
    Italy
    Netherlands
    Norway
    Poland
    Spain
    Switzerland
    United Kingdom
    United States
    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:
    If the last subject enters the OLE study, the end of the trial corresponds to the EOT visit. If the last subject does not enter the OLE study, the end of the trial corresponds to the FU1 visit, FU2 visit, or to the last visit of the PTOP, whichever is last.
    LVLS:
    Se l’ultimo soggetto partecipa allo studio di estensione in aperto (OLE), la durata complessiva dello studio corrisponde alla visita EOT dell’ultimo soggetto. Se l’ultimo soggetto non partecipa allo studio OLE, la durata complessiva dello studio corrisponde all’ultima visita di follow up di sicurezza post-trattamento (FU1) del soggetto, alla visita FU2, o all’ultima visita del periodo di osservazione post-trattamento (PTOP), qualunque sia l’ultima.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    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 years3
    E.8.9.2In all countries concerned by the trial months5
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 97
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 11
    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 60
    F.4.2.2In the whole clinical trial 108
    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)
    Subjects who completed the Month 6 visit will be proposed to enroll in a separate OLE study
    Ai soggetti che completeranno la visita del Mese 6 verrà proposto l'arruolamento in un diverso studio in aperto.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-05-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-07-16
    P. End of Trial
    P.End of Trial StatusCompleted
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