肉毒杆菌毒素注射到大褶皱后患有 Hailey Hailey 或 Darier 病的患者生活质量改善的评估。 (ToxHD)
肉毒杆菌毒素注射到大褶皱后患有 Hailey Hailey 或 Darier 病的患者生活质量改善的评估。毒素 Hailey Darier
Hailey Hailey and Darier disease are rare genetic dermatoses. Mutations of 2 genes (ATP2C1 or ATP2A2 respectively) are responsible for the diseases. These genes have a key role in calcium pump; their defect create abnormal link between keratinocytes' desmosomes and induce skin lesions. Clinically, patients present with inflammatory lesions located in the folds. Quality of life is impaired because of pain, pruritus and tendency to infections. Lesions are permanent but acute exacerbations occur in hot seasons because of increased sweating. Usual therapies are often not effective (local treatment, laser, phototherapy). Because sweating is a well established inducing or aggravating factor, botulism toxin could be an effective treatment for these diseases.
Botulism toxin is already used in clinical practice and acts via a decreased sweet secretion. Improvement of skin lesions in Hailey-Hailey or Darier diseases has been previously reported in a few cases but there is no study properly evaluating the benefit of such treatment.
The aim of the project is to study the improvement of quality of life for patients suffering from Hailey-Hailey or Darier diseases after a injections of botulism toxin in large skin folds. The principal objective is to estimate the distribution of the variation of quality of life at M1 vs. baseline.
研究概览
研究类型
注册 (实际的)
阶段
- 阶段1
联系人和位置
学习地点
-
-
-
Toulouse、法国
- Centre Hospitalier Universitaire
-
-
参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:
- Confirmed diagnosis (clinical and histological features) of Hailey Hailey or Darier diseases.
- Moderate to very severe lesions located in large folds
- Patient aged 18 ans or more
- Patient with health coverage
- Patient who have signed the consent form
- Patient proficient into filling out the questionnaires.
Exclusion Criteria:
- Hypersensibility to toxin or excipients
- Myastheny
- Deglutition's problems
- Past medical history of dysphagia or aspiration pneumonia
- Pregnancy (positive B-HCG test performed a maxima 72h before) or breastfeeding
- Mental , physical incapacity to fill in the questionnaires
- Guardianship patients
- Skin infections at the inclusion visit
- Application in the last 7 days at the site of injection of local treatments (apart emollients or antiseptics) or injections of botulism toxin or dynamic phototherapy or laser in the last 6 months.
- Systemic treatment with aminosides in the last 15 days
- Inclusion in another study in the last 2 months.
学习计划
研究是如何设计的？
设计细节
- 主要用途：其他
- 分配：不适用
- 介入模型：单组作业
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的：Botulism Toxin treatment
Injection of 50 UI Botulism toxin for the treated zone |
Injection of 50 UI of botulism toxin for treated zone |
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Evaluation of quality of life measured by change in the DLQI score
大体时间：Day 0 and day 30
|
Variation of DLQI score between Baseline and M1 |
Day 0 and day 30
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
患者满意度评价 使用 IGA 评分“改善整体评估”
大体时间：第 180 天
|
第 180 天
|
|
|
使用视觉类比疼痛量表评估患者治疗的可接受性
大体时间：注射后第0天
|
注射后第0天
|
|
|
通过副作用的发生来评估中长期的可接受性
大体时间：第 30 天
|
第 30 天
|
|
|
通过副作用的发生来评估中长期的可接受性
大体时间：90天
|
90天
|
|
|
通过副作用的发生来评估中长期的可接受性
大体时间：第 180 天
|
第 180 天
|
|
|
根据 IGA 评分等于 0 的无反应患者百分比评估长期疗效
大体时间：第 30 天
|
第 30 天
|
|
|
通过显着复发的延迟评估长期疗效（皮损再次出现证明治疗合理）
大体时间：长达 180 天
|
长达 180 天
|
|
|
通过比较研究前 6 个月期间或研究期间 6 个月期间发生的感染发作次数来评估长期疗效
大体时间：长达 180 天
|
长达 180 天
|
|
|
Evaluation of quality of life measured by change in the DLQI score
大体时间：Day 0 and day 90
|
Variation of DLQI score between Baseline and M3 |
Day 0 and day 90
|
|
Evaluation of quality of life measured by change in the DLQI score
大体时间：Day 0 and day 180
|
Variation of DLQI score between Baseline and M6 |
Day 0 and day 180
|
|
Evaluation of skin improvement in treated areas using change the IGA score
大体时间：Day 0 and Day 30
|
Variation of IGA score between Baseline and M1 |
Day 0 and Day 30
|
|
Evaluation of skin improvement in treated areas using change the IGA score
大体时间：Day 0 and Day 90
|
Variation of IGA score between Baseline and M3 |
Day 0 and Day 90
|
|
Evaluation of skin improvement in treated areas using change the IGA score
大体时间：Day 0 and Day 180
|
Variation of IGA score between Baseline and M6 |
Day 0 and Day 180
|
|
Evaluation of psychosocial impairment at measured by change in the HidroQoL score
大体时间：Day 0 and Day 30
|
Variation of HidroQoL score between Baseline and M1 |
Day 0 and Day 30
|
|
Evaluation of psychosocial impairment measured by change in the HidroQoL score
大体时间：Day 0 and Day 90
|
Variation of HidroQoL score between Baseline and M3 |
Day 0 and Day 90
|
|
Evaluation of psychosocial impairment measured by change in the HidroQoL score
大体时间：Day 0 and Day 180
|
Variation of HidroQoL score between Baseline and M6 |
Day 0 and Day 180
|
|
Evaluation by the investigator of the treated lesions global severity change as assessed by comparison using measurement of the affected area
大体时间：Day 0 and Day 30
|
Variation of treated lesions severity between Baseline and M1 |
Day 0 and Day 30
|
|
Evaluation by the investigator of the treated lesions global severity change as assessed by comparison using measurement of the affected area
大体时间：Day 0 and Day 90
|
Variation of treated lesions severity between Baseline and M3 |
Day 0 and Day 90
|
|
Evaluation by the investigator of the treated lesions global severity change as assessed by comparison using measurement of the affected area
大体时间：Day 0 and Day 180
|
Variation of treated lesions severity between Baseline and M6 |
Day 0 and Day 180
|
合作者和调查者
调查人员
- 首席研究员：Aude MAZA RIOLAND, MD、University Hospital, Toulouse
出版物和有用的链接
研究记录日期
研究主要日期
学习开始
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (估计的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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