E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Polymyositis / Dermatomyositis / necrotizing autoimmune myopathy |
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E.1.1.1 | Medical condition in easily understood language |
Polymyositis / Dermatomyositis / necrotizing autoimmune myopathy |
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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 | 17.1 |
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.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10036102 |
E.1.2 | Term | Polymyositis |
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 |
The objective of this study is to evaluate the efficacy and safety of gevokizumab in adult patients with polymyositis or dermatomyositis or necrotizing autoimmune myopathy intolerant or resistant or dependent to systemic oral corticosteroids. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Active polymyositis or dermatomyositis or necrotizing autoimmune myopathy intolerant or resistant or dependent to systemic oral corticosteroids, - With a stable background treatment of oral corticosteroid if any and / or a stable dose of immunosuppressive agent if any. - Male or female, age ≥ 18 (or legal age of majority in the country), - Weight ≥ 40 kg and ≤ 125 kg at selection, - For subjects with reproductive potential, a willingness to use highly effective contraceptive measures. |
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E.4 | Principal exclusion criteria |
- Juvenile dermatomyositis, drug induced myositis, severity criteria such as severe cardiovascular or pulmonary involvement or myositis damage, congenital myopathy, inclusion body myositis, cancer- associated myositis or DM diagnosed for less than 1 year, - Impossibility to perform muscle biopsy for PM, DM sine dermatitis and NAM patients (if required), - Non-ambulatory patients, - Fibromyalgia of type I, - Joint disease or other musculoskeletal condition, - Active TB disease, - History of severe allergic or anaphylactic reactions to monoclonal antibodies, - History of malignancy within 5 years prior to selection, - Known immunodeficiency, - Infectious disease, - Pregnancy, breastfeeding or possibility to become pregnant during the study |
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E.5 End points |
E.5.1 | Primary end point(s) |
-Clinical improvement according to Manual Muscle Testing (MMT-8) score -Clinical improvement according to IMACS group's myositis disease activity core set -Specific dermatologic testing: modified CDASI (Cutaneous Dermatomyositis Disease Area and Severity Index) for DM patients (except patients with DM sine dermatitis) -SF36 -Biomarkers |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
MMT-8 and biomarkers : at selection and every 4 weeks until week 24 visit and until week 48 visit (including withdrawal visit) (only for France: at selection and every 4 weeks until week 24 visit) Others : every 4 weeks from week 0 to week 24 and until week 48 visit (including withdrawal visit) (only for France: every 4 weeks from week 0 to week 24) |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
E.6.11 | Pharmacogenomic | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
followed by an open label gevokizumab administration period (except for France) |
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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 | Yes |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 37 |
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 |
Brazil |
Canada |
Czech Republic |
Denmark |
France |
Germany |
Greece |
Hungary |
Italy |
Poland |
South Africa |
Spain |
Sweden |
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 | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
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 | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |