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    EudraCT Number:2018-002210-12
    Sponsor's Protocol Code Number:ID-069A302
    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
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-002210-12
    A.3Full title of the trial
    A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease
    Studio di estensione, multicentrico, in aperto, non controllato, a braccio singolo, per determinare la sicurezza e la tollerabilità a lungo termine di lucerastat 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 study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease
    Uno studio per determinare la sicurezza e la tollerabilità a lungo termine del lucerastat orale in soggetti adulti con malattia di Fabry
    A.3.2Name or abbreviated title of the trial where available
    Not applicable
    Not applicable
    A.4.1Sponsor's protocol code numberID-069A302
    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.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.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
    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/g milligram(s)/gram
    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. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Fabry disease
    Malattia di Fabry
    E.1.1.1Medical condition in easily understood language
    Fabry disease is a rare,inherited disease caused by genetic fault which leads to an accumulation of fatty substance in the body.Can cause symptoms of pain, stomach symptoms and damage to vital organs.
    La malattia di Fabry è una malattia rara ereditaria causata da un difetto genetico.Porta accumulo di sostanza grassa nel corpo.Può causare sintomi di dolore,sintomi di stomaco,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 long-term safety and tolerability of lucerastat in subjects with Fabry disease
    Determinare la sicurezza e la tollerabilità a lungo termine di lucerastat in soggetti con malattia di Fabry
    E.2.2Secondary objectives of the trial
    To evaluate the effect of lucerastat on renal function and cardiac parameters in subjects with Fabry disease;
    To evaluate the long-term effect of lucerastat on biomarkers of Fabry disease.
    Valutare l'effetto di lucerastat sulla funzionalità renale e sui parametri cardiaci in soggetti con malattia di Fabry;
    Valutare l'effetto a lungo termine di lucerastat sui biomarcatori della malattia di Fabry.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed and dated ICF prior to any study-mandated procedure;
    2. Subject completed the 6-month, double-blind treatment period in study ID 069A301;
    1. ICF firmato e datato prima di qualsiasi procedura richiesta dallo studio;
    2. Il soggetto ha completato il periodo di trattamento di 6 mesi in doppio cieco nello studio ID 069A301;
    E.4Principal exclusion criteria
    1. Pregnant / planning to be become pregnant up to 30 days after study treatment discontinuation or lactating subject;
    2. Subject considered to be at high risk of developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
    3. Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results as per investigator judgment.

    In addition, the subject must not be enrolled in study ID-069A302 if at any time during study ID-069A301, one of the following criteria was met:
    4. Subject’s eGFR per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 15 mL/min/1.73 m2;
    5. Subject experienced an event of acute kidney injury Common Terminology Criteria for Adverse Event (CTCAE) grade 2 or above;
    6. Subject experienced an event of stroke CTCAE grade 3 or above;
    7. Subject experienced an event of heart failure leading to in-patient hospitalization or prolongation of ongoing hospitalization.
    1. Incinta / sta pianificando una gravidanza fino a 30 giorni dopo l'interruzione del trattamento in studio o il soggetto che allatta;
    2. Soggetto considerato ad alto rischio di sviluppare segni clinici di coinvolgimento d'organo entro il periodo di tempo dello studio, secondo il giudizio dello sperimentatore;
    3. Qualsiasi fattore o malattia noto che potrebbe interferire con la compliance al trattamento, la condotta dello studio o l'interpretazione dei risultati secondo il giudizio dello sperimentatore.

    Inoltre, il soggetto non deve essere arruolato nello studio ID-069A302 se in qualsiasi momento durante lo studio ID-069A301 è stato soddisfatto uno dei seguenti criteri:
    4. eGFR del soggetto in base all'equazione della creatinina <15 mL / min / 1,73 m2 della collaborazione epidemiologica di malattia renale cronica;
    5. Il soggetto ha subito un evento di danno renale acuto di grado 2 o superiore ai Criteri comuni di terminologia per gli eventi avversi (CTCAE);
    6. Il soggetto ha subito un evento di ictus di grado 3 CTCAE o superiore;
    7. Il soggetto ha subito un evento di insufficienza cardiaca che ha portato al ricovero ospedaliero o al prolungamento del ricovero in corso.
    E.5 End points
    E.5.1Primary end point(s)
    • Treatment-emergent AEs and SAEs up to FU1 visit
    EA emergenti dal trattamento e SAE fino alla visita FU1
    E.5.1.1Timepoint(s) of evaluation of this end point
    From enrollment to FU1 visit; for up to 25 months (24 months OL treatment period plus 1 month Follow-up)
    Dall'iscrizione alla visita FU1; fino a 25 mesi (periodo di trattamento OL di 24 mesi più 1 mese di follow-up)
    E.5.2Secondary end point(s)
    E.5.2.1Timepoint(s) of evaluation of this end point
    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 Yes
    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
    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. 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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned 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 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
    United States
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The global end of the study corresponds to the last subject’s FU1 or FU2 visit, whichever is applicable.
    La fine globale dello studio corrisponde alla visita FU1 o FU2 dell'ultimo soggetto, a seconda di quale sia applicabile
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months2
    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 months6
    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 Yes
    F.3.3.2Women of child-bearing potential using contraception No
    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 50
    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)
    The investigator/delegate will explain to subjects what treatment(s) / medical care is necessary and available according to medical practice and applicable guidelines.
    Lo sperimentatore / delegato spiegherà ai soggetti quali trattamenti / cure mediche sono necessarie e disponibili secondo la pratica medica e le linee guida applicabili.
    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 Decision2020-09-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-10-22
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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