E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with Duchenne Muscular Dystropy Amenable to Exon 51 Skipping |
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E.1.1.1 | Medical condition in easily understood language |
Duchenne muscular dystrophy |
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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 | 19.1 |
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 |
The primary objective of this study is to evaluate the safety and tolerability of eteplirsen in patients with Duchenne muscular dystrophy (DMD) between 4 and 6 years of age who are amenable to exon 51 skipping. |
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E.2.2 | Secondary objectives of the trial |
The secondary objective is to evaluate the effect of eteplirsen on dystrophin expression as measured by the percentage of dystrophin-positive fibers on-treatment as compared to pre-treatment biopsied skeletal muscle tissue.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male 4-6 years of age 2. Diagnosis of DMD, genotypically confirmed 3. Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks 4. Intact right and left biceps muscles or two alternative upper arm muscle groups
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E.4 | Principal exclusion criteria |
1. Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids) 2. Previous or current treatment with any other experimental treatments within 12 weeks or participation in any other clinical trial within 6 months 3. Major surgery within 3 months prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study activities 4. Presence of other clinically significant illness
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E.5 End points |
E.5.1 | Primary end point(s) |
The incidence of adverse events, clinical laboratory abnormalities, abnormalities in vital signs and physical examinations, and abnormalities on ECGs and ECHOs. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Change from Baseline to Week 96 |
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E.5.2 | Secondary end point(s) |
Change from Baseline to Weeks 48 and 96 in percent of dystrophinpositive skeletal muscle fibers (eteplirsen-treated patients only) and drug concentration in plasma. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Change from Baseline to Week 96 |
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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 | Yes |
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 | 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) | 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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
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 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 7 |