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 |
Dermatomyositis is an uncommon inflammatory disease marked by muscle weakness and a distinctive skin rash. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10001403 |
E.1.2 | Term | Adult dermatomyositis |
E.1.2 | System Organ Class | 100000004858 |
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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 assess the efficacy of IgPro20 SC doses in comparison to placebo in adult subjects with DM, as measured by responder status based on the Total Improvement Score (TIS) assessments at Weeks 17, 21, and 25. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study are to assess the efficacy, with additional clinical outcome measures, of IgPro20 in comparison to placebo, the safety of IgPro20 in comparison to placebo, safety and efficacy at Week 53, and safety after Week 53 to end of study participation of IgPro20. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
male or female subjects ≥ 18 years of age with diagnosis of at least probable idiopathic inflammatory myopathies per European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) Classification Criteria which includes confirmation of dermatomyositis (DM) rash/manifestation, disease activity defined by presence of DM rash / manifestation or an objective disease activity measure, and disease severity defined by Physician global visual analog scale (VAS) with a minimum value of 2.0 cm on a 10 cm scale and MMT-8 ≤ 142 or CDASI total activity score ≥ 14. |
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E.4 | Principal exclusion criteria |
Cancer-associated myositis, evidence of active malignant disease or malignancies diagnosed within the previous 5 years, Physician Global Damage ≥ 3, or clinically relevant improvement between Screening Visit and Baseline |
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E.5 End points |
E.5.1 | Primary end point(s) |
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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) |
1. Mean Total Improvement Score (TIS), 2. Point estimates and 95% CI for mean difference (IgPro20 – placebo) in TIS, 3. Mean changes from Baseline in Manual Muscle Testing (MMT-8), 4. Point estimates and 95% CI for mean change difference (IgPro20 –placebo) in MMT-8, 5. Mean changes from Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) total activity score, 6. Point estimates and 95% CI for mean change difference (IgPro20 – placebo), 7. Number of subjects who are able to reduce the oral corticosteroid dose by ≥ 25% , 8. Percentage and 95% CI of subjects who are able to reduce the oral corticosteroid dose by ≥ 25% 9. Point estimates and 95% CI for the odds ratio (IgPro20:Placebo) of subjects who are able to reduce the oral corticosteroid dose by ≥ 25% 10. Mean TIS, 11. Percentage of subjects achieving TIS ≥ 20, ≥ 40, and ≥ 60 points, 12. Time to first achieving TIS ≥ 20, ≥ 40, and ≥ 60 points on the TIS, 13. Percentage of subjects achieving TIS ≥ 20 points at the end of study period 2, 14. Mean changes in individual CSMs (except muscle enzymes) and CDASI from Baseline, 15. Mean changes in individual CSMs except muscle enzymes and CDASI from Week 25, 16. Number of subjects meeting Definition of Worsening (DOW) at least once, twice, or > twice, 17. Percentage of subjects meeting DOW at least once, twice, or > twice, 18. Time to meeting DOW for the first time, 19. Number of subjects meeting DOW and receiving rescue corticosteroid treatment, 20. Percentage of subjects meeting DOW and receiving rescue corticosteroid treatment, 21. Number of subjects who start oral corticosteroid dose taper, 22. Percentage of subjects who start oral corticosteroid dose taper, 23. Number of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%, ≥ 50%, ≥ 75%, 24. Number of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%, ≥ 50%, ≥ 75%, 25. Percentage of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%, ≥ 50%, ≥ 75%, 26. Percentage of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%, ≥ 50%, ≥ 75%, 27. Percentage of subjects receiving rescue corticosteroid treatment, 28. Percentage of subjects whose rescue corticosteroid treatment is tapered, 29. Time to first intake of rescue corticosteroid treatment, 30. Number of subjects having at least 1 level, 2 levels, and more than 2 levels of improvement from Baseline in mobility, self-care, and usual activities domains of EQ-5D-5L, 31. Percentage of subjects having at least 1 level, 2 levels, and more than 2 levels of improvement from Baseline in mobility, self-care, and usual activities domains of EQ-5D-5L, 32. Number of subjects having no reduction in levels, at least 1 level, 2 levels, and more than 2 levels of improvement in mobility, self-care, and usual activities domains of EQ-5D-5L 33. Percentage of subjects having no reduction in levels, at least 1 level, 2 levels, and more than 2 levels of improvement in mobility, self-care, and usual activities domains of EQ-5D-5L 34. Percentage of subjects with Treatment Emergent Adverse Events (TEAEs), 35. Percentage of subjects with related TEAEs, 36. Percentage of subjects with serious TEAEs, 37. Rate of TEAEs per days with infusion, 38. Rate of TEAEs per days with infusion, by severity, 39. Rate of related TEAEs per days with infusion, 40. Rate of serious TEAEs per days with infusion. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1 to 9. Up to Week 25 10. Week 5 up to Week 53 11. Week 5 up to Week 53 12. Week 5 up to Week 53 13. Up to Week 53 14. Between Week 5 and Week 25 15. Week 29 to Week 53 16. Baseline up to Week 53 17. Baseline up to Week 53 18. Baseline up to Week 53 19. Baseline up to Week 53 20. Baseline up to Week 53 21. Baseline up to Week 53 22. Baseline up to Week 53 23. Baseline up to Week 25 24. Baseline up to Week 53 25. Baseline up to Week 25 26. Baseline up to Week 53 27. Baseline up to Week 25 28. Baseline up to Week 25 29. Baseline up to Week 25 30. Baseline up to Week 53 31. Baseline up to Week 53 32. Week 25 up to Week 53 33. Week 25 up to Week 53 34-40. Throughout the study |
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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 | 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) | 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 | 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 | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 34 |
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 |
Argentina |
Australia |
Japan |
Mexico |
United States |
European Union |
Switzerland |
Russian Federation |
Ukraine |
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 | 8 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 8 |
E.8.9.2 | In all countries concerned by the trial months | 1 |