E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Fabry disease |
Malattia di Fabry |
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E.1.1.1 | Medical 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 |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10010331 |
E.1.2 | Term | Congenital, familial and genetic disorders |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main 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 |
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E.2.2 | Secondary 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. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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;
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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; |
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E.4 | Principal 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. |
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E.5 End points |
E.5.1 | Primary end point(s) |
• Treatment-emergent AEs and SAEs up to FU1 visit |
EA emergenti dal trattamento e SAE fino alla visita FU1 |
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E.5.1.1 | Timepoint(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) |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Tolerability |
Tollerbilità |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 25 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Canada |
United States |
Austria |
Belgium |
Germany |
Ireland |
Italy |
Netherlands |
Norway |
Poland |
Spain |
United Kingdom |
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E.8.7 | Trial 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
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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 |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |