Idiopathic Inflammatory Myopathies
Conditions
Brief summary
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.
Interventions
DRUGPlacebo
Placebo-matching nerandomilast
DRUGNerandomilast
Nerandomilast
Sponsors
Boehringer Ingelheim
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. 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. 2. 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. 3. Participants with overlap myositis (OM) must have IIM as the predominant disease. 4. 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. 5. Disease activity defined by moderate to severe myopathy. 6. The participant must be on background therapy for IIM. 7. Woman (or women) of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control for a period of 28 days before treatment initiation, throughout the trial, and for a period of at least 5 days after the last dose of investigational medicinal product (IMP). WOCBP taking oral contraceptives also have to use one barrier method. 8. Signed and dated written informed consent in accordance with ICH-GCP and local legislation prior to admission to the trial. 9. Further inclusion criteria apply.
Exclusion criteria
1. 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. 2. 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. 3. 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. 4. Participants diagnosed with advanced interstitial lung disease (ILD). 5. Severe muscle damage. 6. Further
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Total improvement score (TIS) score (continuous) at Week 52 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Achievement of a TIS-40 response (yes/no; defined as TIS ≥40) at Week 52 | 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 | 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) | At baseline and at Week 52. | 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 | At baseline and 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. |
| Change from baseline in PtGA at Week 52 | At baseline and at Week 52. | Patient global disease activity (PtGA) measures the participant's self-assessment of the disease. It is composed of a 100 mm visual analogue scale. Patients will mark their assessment between "extremely poor" (0) and "excellent" (100). The difference between 0 (extremely poor) and the patient assessment is the PtGA, where higher distances indicate a better health status. |
| Change from baseline in clinical-reported outcome 1 at Week 12 | At baseline and at Week 12. | — |
| Change from baseline in PhGA at Week 52 | At baseline and at Week 52. | Physician global activity (PhGA) captures the physician's assessment of the participant's overall health. It is composed of a 100 mm visual analogue scale. Physicians will mark their assessment between "extremely poor" (0) and "excellent" (100). The difference between 0 (extremely poor) and the physician assessment is the PhGA, where higher distances indicate a better health status. |
| Change from baseline in HAQ-DI at Week 52 | At baseline and at Week 52. | The health assessment questionnaire disability index (HAQ-DI) assesses difficulties in performing activities of daily living across 8 domains. Each domain is rated on a scale of 0-3. The higher the score, the higher the disability. |
| Change from baseline in MMT-8 score at Week 52 | At baseline and at Week 52. | Manual Muscle Testing (MMT-8) assesses the muscle strength of 8 proximal, distal, and axial muscle groups. Each item is rated from 0 to 10, with the total score ranging from 0 to 150, where higher scores mean better muscle strength condition. |
| Achievement of a TIS-60 response (yes/no; defined as TIS ≥60) at Week 52 | At baseline and at Week 52. | — |
| Change from baseline in clinical-reported outcome 2 at Week 12 | At baseline and at Week 12. | — |
| Change from baseline in the most abnormal baseline muscle enzymes (aldolase, CK, AST, ALT, or LDH) at Week 52 | At baseline and at Week 52. | CK means creatine kinase, AST means aspartate aminotransferase, ALT means alanine aminotransferase, and LDH means lactate dehydrogenase. |
Countries
Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, China, France, Germany, Greece, Hungary, Italy, Japan, Mexico, Netherlands, Poland, Portugal, Romania, Serbia, South Korea, Spain, Taiwan, Thailand, United States
Contacts
CONTACTBoehringer Ingelheim
Outcome results
None listed