A 44-week Monocentric Open Study Assessing the Efficacy and Safety of Deucravacitinib in Adults With Inflammatory Genodermatoses
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- efficacy of deucravacitinib
研究概览
简要总结
The goal of this clinical trial is to learn about deucravacitinib treatment in adults with inflammatory epidermal genodermatoses . The main question it aims to answer are: describe efficacity and safety of this treatment.
Participants will take treatments and have to use bullets during the study period.
详细描述
The patient will have 8 visits After Confirmation of inclusion criteria and no exclusion criteria, the study will be explain to the patient after signing the inform consent and be included in the study Treatment Period (Period 1); challenge period This period will be the first treatment period that will occur from 16 weeks. During this period the patient will see the doctor at the hospital, he will perform the study procedures The second period : dechallenge (period 2): All patients stop their treatment for 12 weeks.
The second treatment period - rechallenge (period 3) the patient will take the treatment for a second 16 weeks period At week 44 - End of study
At each visit:
The investigator at each visit compliance to the treatment and adverse events, Measure vital signs and perform clinical examination.
The patient will assess the different questionnaires, for the study and have blood sample
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 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject has congenital skin fragility with skin and/or mucosal blisters, a clinical phenotype of EBS-sev and a laboratory confirmed diagnosis of EBS by KRT5 and/or 14 mutation (autosomal only) OR a clinical phenotype of inflammatory ichthyosis (IC) including keratinopathic ichthyosis due to KRT1/10 mutation, ECI with identified genetic mutations (TGM5, NIPAL4, ABCA12, etc...).
- •Subject has at least a mean daily number of 4 new blisters (EBS-sev) OR an ISS > 50/
- •Subject agrees not to use any topical therapies other than the investigator approved.
排除标准
- •Subjects with another form of ICI ie Netherton syndrome, Kid syndrome etc.
- •Infectious/Immune-related Exclusions
- •Medical History and Concurrent Diseases
研究组 & 干预措施
deucravacitinib treatment
challenge-dechallenge -rechallenge design
干预措施: deucravacitinib (Drug)
结局指标
主要结局
efficacy of deucravacitinib
时间窗: week 44
number of new blister conting every day
次要结局
- Safety of deucravacitinib treatment(week 44)