A Phase III, Parallel Design, Double-blind, Randomised, Placebo-controlled, Multi-centre Trial to Evaluate the Efficacy and Safety of Nerandomilast Oral Therapy Added to Standard Background Therapy Over 52 Weeks in Adult Trial Participants With Active Idiopathic Inflammatory Myopathies (VERANDA™-IIM)
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 270
- 试验地点
- 148
- 主要终点
- Total improvement score (TIS) score (continuous) at Week 52
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
- •Disease activity defined by moderate to severe myopathy.
- •The participant must be on background therapy for IIM.
- •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.
- •Signed and dated written informed consent in accordance with ICH-GCP and local legislation prior to admission to the trial.
- •Further inclusion criteria apply.
排除标准
- •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).
- •Severe muscle damage.
- •Further exclusion criteria apply.
研究组 & 干预措施
Placebo
Placebo matching nerandomilast
干预措施: Placebo (Drug)
Nerandomilast
干预措施: Nerandomilast (Drug)
结局指标
主要结局
Total improvement score (TIS) score (continuous) at Week 52
时间窗: 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.
次要结局
- Achievement of a TIS-40 response (yes/no; defined as TIS ≥40) at Week 52(At Week 52.)
- Achievement of a successful tapering of oral corticosteroids (OCS) (yes/no) at Week 52(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.)
- 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.)
- Change from baseline in PtGA at Week 52(At baseline and at Week 52.)
- Change from baseline in CDASI-A at Week 12 (in participants with baseline CDASI-A ≥6)(At baseline and at Week 12.)
- Change from baseline in PhGA at Week 52(At baseline and at Week 52.)
- Change from baseline in HAQ-DI at Week 52(At baseline and at Week 52.)
- Change from baseline in MMT-8 score at Week 52(At baseline and at Week 52.)
- 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 5-D itch at Week 12 (in participants with baseline CDASI-A ≥6)(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.)
- Change from baseline in clinical-reported outcome 1 at Week 12(At baseline and at Week 12.)
- Change from baseline in clinical-reported outcome 2 at Week 12(At baseline and at Week 12.)