This study is open to adults aged 18 and older who have a condition known as active idiopathic inflammatory myopathy (IIM) or myositis. People can participate if they have a specific type of myositis. The purpose of this study is to find out whether a medicine called nerandomilast improves IIM symptoms.
Participants are put into 2 groups randomly, which means by chance. One group takes nerandomilast tablets and the other group takes placebo tablets. Placebo tablets look like nerandomilast tablets but do not contain any medicine. Participants take nerandomilast or placebo twice a day for 1 year.
Participants are in the study for about 1 year and 2 months. During this time, they visit the study site at least 16 times. During this time, doctors regularly check the participant's health, IIM symptoms, and take note of any unwanted effects. Participants fill in questionnaires about their IIM symptoms and how IIM impacts their quality of life. The results are compared between the 2 groups to see whether the treatment works.
Male or female adult individuals from ≥18 years of age (or alternative age for adults based on local regulations) on the date of signing the informed consent.
A definite or probable clinical diagnosis of idiopathic inflammatory myopathy (IIM) according to the 2017 American college of rheumatology (ACR)/European alliance of associations of rheumatology (EULAR) classification criteria.
Participants with overlap myositis (OM) must have IIM as the predominant disease.
Participants ≥18 years of age (or alternative age for adults based on local regulations) with juvenile dermatomyositis (JDM) can only be included if they were 15 years old or older at first onset of DM symptoms.
Major surgery (major according to the investigator's assessment, e.g. hip replacement) performed within 6 weeks prior to randomisation or planned during the trial period.
Any documented active or suspected malignancy or history of malignancy within 5 years prior to screening, except appropriately treated basal cell carcinoma of the skin, or in situ squamous cell carcinoma of the skin, or in situ carcinoma of uterine cervix.
Participants who must or wish to continue the intake of restricted medications or any drug considered likely to interfere with the safe conduct of the trial.
Participants diagnosed with advanced interstitial lung disease (ILD).
Parallel
Placebo
DrugPlacebo-matching nerandomilast
Nerandomilast
DrugNerandomilast
Total improvement score (TIS) score (continuous) at Week 52
The TIS composite score includes 6 core set measures: manual muscle testing (MMT8), extra-muscular disease activity (MDAAT), patient global disease activity (PtGA), physician global disease activity (PhGA), health assessment questionnaire disability index (HAQ-DI), and muscle enzymes. An improvement score for each core set measure will be assigned using the absolute percent change, based on predetermined ranges. The TIS will be the sum of the all core set measure, scoring from 0 to 100, with higher scores corresponding to a greater degree of improvement.
Achievement of a TIS-40 response (yes/no; defined as TIS ≥40) at Week 52
Number of participants with 40 TIS points or higher.
Achievement of a successful tapering of oral corticosteroids (OCS) (yes/no) at Week 52
Number of participants achieving a TIS-40 response at Week 52 and ≤5 mg/day of prednisone (or equivalent) at Week 52
Change from baseline in cutaneous dermatomyositis disease area and severity index activity score (CDASI-A) at Week 52 (in participants with baseline CDASI-A ≥6)
The Cutaneous Dermatomyositis Disease Area and Severity Index activity score (CDASI-A) is a questionnaire assessing skin disease activity and damage in patients with dermatomyositis (DM). It ranges from 0 to 100, with higher values indicating more severe active skin involvement.
Change from baseline in extra-muscular disease activity as per myositis disease activity assessment tool (MDAAT) at Week 52
Myositis Disease Activity Assessment Tool (MDAAT) is a questionnaire assessing the disease activity of extra-muscular organ systems and muscle in participants with IIM. It is a combined tool with two parallel scores: the MYOACT VAS and the MITAX. The Myositis disease activity assessment (MYOACT) visual analogue scale (VAS) scores the overall severity of disease activity. The sum of the individual scores ranges from 0 to 70, The higher the more severe. For the Myositis Intention-to-Treat Activity Index (MITAX), each question is answered with either: 0 = not present; 1 = improving; 2 = the same; 3 = worse; 4 = new. The summed scores are summed to obtain a total MITAX score with a range of 0 to 63, the higher the more severe.
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