E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Sporadic Inclusion Body Myositis |
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E.1.1.1 | Medical condition in easily understood language |
Sporadic Inclusion Body Myositis |
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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 | 18.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10066407 |
E.1.2 | Term | Inclusion body myositis |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue 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 demonstrate that at least one dose regimen of BYM338 will increase the 6 minute walking distance test relative to placebo at week 52. |
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E.2.2 | Secondary objectives of the trial |
•Change from Baseline in lean body mass (LBM) at Week 52
•Change from Baseline in quadriceps Quantitative Muscle Testing (QMT) at Week 52
•Change from Baseline in Patient-Reported Physical Function at Week 52
•Rate of Fall Events
•Change from Baseline in Short Physical Performance Battery score at Week 52
•Dose-response relationship in the change from Baseline in 6MWD meters to Week 52 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•Diagnosed with sporadic inclusion body myositis;
•Must be able to walk (assistive aids allowed, including intermittent use of wheelchair)
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E.4 | Principal exclusion criteria |
•No other conditions that significantly limit ability to move around;
•Must not be using corticosteroids. Must not have used systemic corticosteroid (at daily dose >=10mg prednisone) for the past 3 months;
•Must meet cardiovascular requirements;
•Must not be pregnant or nursing;
•Must not have a chronic active infection (e.g., HIV, hepatitis B or C, tuberculosis, etc);
Other protocol-defined inclusion/exclusion criteria may apply
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E.5 End points |
E.5.1 | Primary end point(s) |
Change from Baseline in 6 Minute Walking Distance Test (6MWD) meters to Week 52 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
•Change from Baseline in lean body mass (LBM) at Week 52
•Change from Baseline in quadriceps Quantitative Muscle Testing (QMT) at Week 52
•Change from Baseline in Patient-Reported Physical Function at Week 52
•Rate of Fall Events
•Change from Baseline in Short Physical Performance Battery score at Week 52
•Dose-response relationship in the change from Baseline in 6MWD meters to Week 52 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Week 52 for LBM, QMT, patient-reporter physical function, short physical performance battery and dose-response relationship for 6MWD and measured during entire study for falls , safety and tolerability |
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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 | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | Yes |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Tolerability and biomarkers assessments |
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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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 4 |
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.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 18 |
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 | No |
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 |
Belgium |
Denmark |
France |
Germany |
Italy |
Japan |
Netherlands |
Poland |
Switzerland |
United Kingdom |
United States |
|
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 | 2 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | 20 |
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 | 1 |
E.8.9.2 | In all countries concerned by the trial days | 20 |