E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Duchenne Muscular Dystrophy (DMD) |
Distrofia Muscolare di Duchenne (DMD) |
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E.1.1.1 | Medical condition in easily understood language |
DMD is a genetic disease characterised by rapidly progressive muscle weakness and wasting which leads to severe disability. |
La DMD è una malattia genetica caratterizzata da una rapida e progressiva debolezza e perdita di massa muscolare che porta a grave disabilità |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10013801 |
E.1.2 | Term | Duchenne muscular dystrophy |
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 assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.(up to Visit 4/Week 78) |
Valutare la sicurezza a lungo termine dell'idebenone nei pazienti con DMD che hanno completato lo studio SIDEROS (fino alla visita 4/settimana 78) |
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E.2.2 | Secondary objectives of the trial |
To describe the long-term evolution of respiratory function in idebenonetreated DMD patients who completed the SIDEROS study, classified by background factors including, but not limited to age, DMD history (e.g. time of loss of ambulation, mutation type), type of steroid regimen and study treatment assignment in the SIDEROS study.(up to Visit 4/Week 78) |
Descrivere l’evoluzione a lungo termine della funzione respiratoria in pazienti affetti da DMD trattati con idebenone che hanno completato lo studio SIDEROS classificati per fattori di background come, in modo non esaustivo, età, storia della DMD (per es. perdita della deambulazione, tipo di mutazione), tipo di regime steroideo e assegnazione al trattamento dello studio durante lo studio SIDEROS. (fino alla visita 4/settimana 78) |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Completion of the SIDEROS study at Visit 8/ Week 78. Signed and dated Informed Consent Form. inclusion criteria for the optionsal continued treatment with idebenone beyond Visit 4 of SIDEROS-E study: 1. completion of Visit 4 / week 78 of Sideros-E study 2. Signed and dated Informed Consent Form for continued treatment |
1. Completamento dello studio SIDEROS alla visita 8/settimana 78 2. Datato e firmato il modulo di Consento informato per SIDEROS-E Criteri di inclusione per la continuazione del trattamento con idebenone facoltativa oltre la visita 4/settimana 78 dello studio SIDEROS: 1. Completamento dello studio SIDEROS-E alla visita 4/settimana 78 2. Datato e firmato il modulo di Consento informato per la continuazione del trattamento |
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E.4 | Principal exclusion criteria |
1. Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8). 2. Safety, tolerability or other issues arising during the course of the SIDEROS study which in the opinion of the Investigator may put the patient at significant risk or may interfere significantly with the patient's participation in the SIDEROS-E study. 3. Use of any investigational drug other than the study medication. Exclusion criteria for the optional continued treatment with idebenone beyond Visit 4/ Week 78 of SIDEROS-E study: 1. Premature withdrawal from SIDEROS-E study before Visit 4/ Week 78 2. Any conditions, which, in the opinion of the Investigator might trigger a negative risk-benefit assessment for the patient 3. Use of any Investigational drug other than the study medication |
1. Pazienti che hanno interrotto prematuramente lo studio Studio SIDEROS (cioè non hanno sostenuto le visite dalla V1 alla V8) 2. Sicurezza, tollerabilità o altri problemi emersi durante lo studio SIDEROS che secondo l’opinione dello sperimentatore possano comportare un rischio significativo per il paziente in SIDEROS-E o che possano interferire in modo significativo alla partecipazione del paziente a SIDEROS-E 3. Uso di qualunque farmaco sperimentale diverso da quello dello studio Criteri di esclusione per la continuazione del trattamento con idebenone facoltativa oltre la visita 4/settimana 78 dello studio SIDEROS-E: 1. Ritiro anticipato dallo studio SIDEROS-E prima della visita 4/settimana 78 2. Qualunque condizione che secondo l’opinione dello Sperimentatore possa risultare in una valutazione del beneficio/rischio negativa per il paziente 3. Uso di qualunque farmaco sperimentale diverso da quello dello studio |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary: Standard safety assessments, including number of premature discontinuations of study treatment due to adverse events, incidence and severity of adverse events, actual values and changes from baseline in safety laboratory parameters, vital signs and electrocardiogram (ECG). |
Principale: • Normali valutazioni sulla sicurezza quali numero delle interruzioni anticipate del trattamento previsto dallo studio a causa di eventi avversi, incidenza e gravità degli eventi avversi, valori effettivi e variazioni dalla baseline dei parametri di laboratorio sulla sicurezza, segni vitali ed elettrocardiogramma (ECG). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Electrocardiogram: baseline Adverse events, safety laboratory parameters, vital signs: 26 weeks , 52 weeks, 78 weeks, post-completion follow-up visit |
ECG: baseline Eventi Avversi, parametri di sucurezza di laboratorio, segni vitali: 26, 52, 78 settimane, dopo il completamento della visita di Follow-up |
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E.5.2 | Secondary end point(s) |
Secondary: Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p), Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) and Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p). Feasibility of continued idebenone treatment, measured with number of discontinuations of study treatment due to adverse events, will be the only applicable endpoint for the optional continued treatment period (beyond Visit 4). |
Secondario: • Scostamento dalla baseline della Capacità vitale forzata (FVC) come percentuale della previsione (FVC%p), picco di flusso espiratorio (PEF) come percentuale della previsione (PEF%p) e Volume espirato forzato in 1 secondo (FEV1) come percentuale del valore previsto (FEV1%p). La fattibilità del trattamento con idebenone continuato, misurata con il numero di interruzioni del trattamento in studio a causa di eventi avversi, sarà l'unico endpoint applicabile per il periodo di recupero continuo opzionale (oltre la visita 4). |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Forced Vital Capacity (FVC), Peak Expiratory Flow (PEF), Forced Expiratory Volume in 1 second (FEV1) : baseline, 26 weeks , 52 weeks, 78 weeks . FVC and PEF in optional continued period : 130 weeks, 182 weeks . Adverse events for the optional continued treatment period : 104 weeks, 130 weeks, 156 weeks, 182 weeks |
FVC, PEF, FEV1: baseline, 26, 52, 78 settimane. FVC e PEF nel periodo continuato opzionale: 130 settimane, 182 settimane Eventi avversi per il periodo di trattamento continuato opzionale: 104 settimane, 130 settimane, 156 settimane, 182 settimane |
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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 | No |
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 | No |
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 | Yes |
E.8.1.7.1 | Other trial design description |
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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 | 2 |
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 | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 36 |
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 |
Israel |
United States |
Austria |
Belgium |
France |
Germany |
Italy |
Netherlands |
Spain |
Sweden |
Switzerland |
United Kingdom |
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E.8.7 | Trial 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
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 43 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 43 |
E.8.9.2 | In all countries concerned by the trial days | 0 |