E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Duchenne Muscular Dystrophy |
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MedDRA Classification |
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 whether treatment with idebenone improves or slows decline in cardiac function in patients with cardiac dysfunction associated with DMD |
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E.2.2 | Secondary objectives of the trial |
To determine whether treatment with idebenone improves or slows decline in muscle strength and/or respiratory function in patients with DMD To evaluate the safety and tolerability of idebenone in patients with DMD To determine whether treatment with idebenone changes biochemical markers reflecting cardiac overload or cardiac degeneration
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
•Patients 10 – 16 years of age at time of enrolment •Male •Presence of cardiac involvement/dysfunction, defined by abnormal peak systolic strain in LV inferolateral wall •Confirmed diagnosis of DMD (out of frame dystrophin gene deletion or absent/< 5% dystrophin protein on muscle biopsy; clinical picture consistent of typical DMD) •If on chronic glucocorticosteroids treatment (deflazacort, prednisone) for DMD (or any other disease): dosage must be stable (unchanged) 6 months prior to inclusion •If on chronic medication for DMD associated cardiomyopathy (β-blocker, diuretics): dosage must be stable (unchanged) 3 months prior to inclusion •Ability to provide reproducible repeat QMT upper limb score within 15% of first assessment score (at Visit 1/Day 1 vs. screening)
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E.4 | Principal exclusion criteria |
•Symptomatic cardiomyopathy or heart failure •Asymptomatic but severe cardiac dysfunction as Screening Visit (( evaluation): FS < 20% and/or EF < 40% •Use of ACE inhibitors •Previous history of ventricular arrhythmias (other than isolated ventricular extrasystole); ventricular arrhythmias presented at Screening •Previous (6 months or less) participation in any other therapeutic trial for DMD •Use of coenzymeQ10, idebenone, creatine, glutamine, oxatomide, or any herbal medicines within the last 6 months •History of significant concomitant illness or significant impairment of renal or hepatic function (serum creatinine and GGT greater than 1.5 times upper limit for age and gender) •Known individual hypersensitivity to idebenone
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E.5 End points |
E.5.1 | Primary end point(s) |
The relative change from Baseline (at Screening) to Week 52 in peak systolic radial strain of left ventricle (LV) inferolateral wall, assessed by Color Doppler Myocardial imaging (CDMI). |
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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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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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End of the trial is at last patient last visit as defined per protocol |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | |