A randomized, double-blind, placebo-controlled Phase II proof of concept study evaluating efficacy and safety of Upadacitinib in patients with idiopathic inflammatory myopathies after withdrawal of intravenous immunoglobulins (IVIG) (IVIG-Spare)

Antisynthetase Syndrom, Dermatomyositis, Immune-mediating necrotising myopathie, overlap Myositis, Polymyositis

To assess the proportion of patients in IVIG-free stable disease activity at week 16 after randomization across two study arms comparing Upadacitinib 30 mg vs Placebo.

• Difference in proportion of patients in IVIG-free stable disease activity at week 20 between patients with Upadacitinib versus Placebo, Different proportion of patients with IVIG-free stable disease activity at week 16 and 20 using different definitions of disease worsening: (1) worsening PhGA by ≥ 2 cm NRS and worsening MMT-8 by ≥ 20%, or (2) worsening EmGA by ≥ 2 cm NRS or (3) a worsening of 3 of the 6 core set measures (HAQ-DI, MMT-8, CK, PhGA, PtGA, EmGA) by ≥ 30%., • Difference in time to first flare between patients with Upadacitinib versus Placebo, • Difference of manual muscle test between Upadacitinib and Placebo at week 16 and week 20, • Difference of patient reported outcomes, including 0- 10 numeric rating scale (NRS) of PtGA, EmGA, muscular pain, fatigue, dyspnoe and dysphagia; Quality of life, Health Assessment Questionaire Disability (HAQ-DI) between Upadacitinib and Placebo at week 16 and week 20, • Difference cumulative dose of steroids between patients with Upadacitinib versus Placebo at week 16 and 20, • Difference in laboratory parameters of values (CK, ALT, AST, LDH and aldolase) between Upadacitinib and Placebo at week 16 and week 20, • Differences in safety profile according to number of adverse events and organ systems (haematologic, hepatic, gastrointestinal, infections)

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Austria