A Phase II Open-Label Study of Deucravacitinib for Refractory Adults With Dermatomyositis/ Juvenile Dermatomyositis
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- To obtain preliminary data regarding the efficacy of a selective TYK2 inhibitor, deucravacitinib, in adult patients with active, treatment-refractory DM/JDM
研究概览
简要总结
Background:
Dermatomyositis (DM) and juvenile dermatomyositis (JDM) are diseases that weaken the immune system. DM and JDM can affect the muscles, skin, joints, and lungs and cause skin rashes and muscle inflammation. Symptoms include weakness, pain, fatigue, and rash. Not everyone responds to current treatments. The FDA has approved a drug called deucravacitinib to treat people with plaque psoriasis. Researchers want to find out if this drug can help people with DM or JDM, too.
Objective:
To test deucravacitinib in people with DM or JDM.
Eligibility:
People aged 18 years and older with DM or JDM.
Design:
Participants will have 9 clinic visits over 28 weeks.
Participants will be screened. They will have a physical exam with blood and urine tests. They will have a test of their heart function. They will complete a short questionnaire about their daily life, pain level, and ability to walk, eat, and do other activities.
Deucravacitinib is a pill taken by mouth twice per day at home. Participants will come to the clinic once every 4 weeks for 24 weeks while they are taking the drug. They will have a final visit 4 weeks after their last dose of the study drug. Blood and urine tests will be repeated during these visits. Each visit may take up to 6 hours.
If the drug is helping them, participants may extend their treatment beyond the first 24 weeks. Then they will visit the clinic every 3 months.
详细描述
Study Description:
Myositis is a heterogeneous family of systemic autoimmune diseases affecting adults and children that can affect the muscle, skin, lungs, joints, and/or gastrointestinal tract. Unfortunately, many patients do not respond completely to current immunosuppressive therapies. Although the pathophysiological mechanisms underlying these diseases are incompletely understood, in adult patients with dermatomyositis (DM) and juvenile dermatomyositis (JDM), a Type 1 interferon gene signature in blood, skin, and muscle correlates with disease activity. Furthermore, recent reports suggest that non-selective inhibitors of the JAK/STAT pathway may be effective in treating patients with refractory DM and JDM. In this study, we will test our hypothesis that inhibition of JAK/STAT pathway using the selective oral TYK2 inhibitor, deucravacitinib, will be efficacious and safe in adult patients with active, treatment-refractory DM/JDM by performing a 24-week, phase II, open-label treatment trial.
At the conclusion of the Treatment Period of the study, all subjects will have the option to receive deucravacitinib in the Extension Portion of the trial.
Objectives:
Primary Objective:
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Drug (Deucravacitinb)
Participants will receive a 6mg pill taken 2 times per day over 24 weeks and an optional extension period
干预措施: Deucravacitinb (Drug)
结局指标
主要结局
To obtain preliminary data regarding the efficacy of a selective TYK2 inhibitor, deucravacitinib, in adult patients with active, treatment-refractory DM/JDM
时间窗: At 24 weeks
The number of DM/JDM patients at least a minimal Clinical response using the total improvement score as defined in 2016 ACR/EULAR Criteria for Minimal, Moderate, and Major Clinical Response in Adult Dermatomyositis and Polymyositis and Juvenile Dermatomyositis
次要结局
- To obtain preliminary data regarding the efficacy of deucravacitinib to improve skin disease activity in adult patients with active, treatment-refractory DM/JDM, as defined by the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)(At 24 weeks)
- To obtain preliminary data regarding the frequency and incidence of adverse events in treatment-refractory dermatomyositis patients treated with deucravacitinib.(Over 28 weeks)