E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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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 | 17.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10012503 |
E.1.2 | Term | Dermatomyositis |
E.1.2 | System Organ Class | 10040785 - Skin and subcutaneous 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 assess the efficacy of different doses of BAF312 on the MMT-8 after 6 months of treatment. |
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E.2.2 | Secondary objectives of the trial |
To assess the effects of different doses of BAF312 on safety, pharmacokinetics and peripheral blood lymphocyte counts in active DM patients
To assess the efficacy of different doses of BAF312 after 3 months of treatment in active DM patients as assessed by manual muscle testing using the MMT-8 scoring system |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Written informed consent must be obtained before any assessment is performed.
• Male and female patients between 18 - 65 (inclusive) years of age who have been defined as "definite" or “probable” based on the criteria of Bohan and Peter (Bohan and Peter 1975) for dermatomyositis at least 12 months before screening
• Patients must have active disease as defined by dermatomyositis skin rash AND Muscle weakness
• Patients must have responded inadequately to previous standard of care or have demonstrated significant toxicity or intolerance to such therapies.
• Patients may be on a stable dose of corticosteroid (up/equal to 20 mg once daily prednisone equivalent)
• Patients currently treated with oral or subcutaneous MTX must have been a stable dose of no more/equal to than 25 mg per week
• Patients currently treated with Azathioprine must have been a stable maintenance dose of no more/equal to 3 mg/kg/day • Negative cancer screening conducted in the 6 months prior to screening visit
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E.4 | Principal exclusion criteria |
• Dermatomyositis patients having overlap myositis or any other type of myositis including paraneoplastic myositis, drug-induced myopathy, necrotizing myositis
• Preexisting severe cardiac or pulmonary conditions, malignancy of any organ system or significant eye diseases.
• Uncontrolled diabetes mellitus or diabetes complicated with organ involvement.
• Pregnant or nursing (lactating) women
• Other protocol-defined inclusion/exclusion criteria apply.
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E.5 End points |
E.5.1 | Primary end point(s) |
To assess the efficacy of different doses of BAF312 after 6 months of treatment in active DM patients as assessed by manual muscle testing using the MMT-8 scoring system. |
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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) |
- To assess the effects of different doses of BAF312 on safety, pharmacokinetics and peripheral blood lymphocyte counts in active DM patients.
- To assess the efficacy of different doses of BAF312 after 3 months of treatment in active DM patients as assessed by manual muscle testing using the MMT-8 scoring system. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- for the first listed secondary endpoint : at every visit (after 10 days followed by every month for 1 year)
- for the second listed secondary endpoint: at 3 months |
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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 | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
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 | 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.8.4.1 | 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 | 12 |
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 |
Canada |
Czech Republic |
Germany |
Hungary |
Japan |
Poland |
United States |
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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.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 24 |
E.8.9.1 | In the Member State concerned days | 14 |
E.8.9.2 | In all countries concerned by the trial months | 26 |