Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin
试验速览
- 阶段
- 3 期
- 入组人数
- 8
- 试验地点
- 4
- 主要终点
- number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin
研究概览
简要总结
Dowling Meara type of epidermolysis bullosa simplex (EBS-DM) is a rare genodermatosis due to keratin 5 and 14 mutation, characterized by skin fragility and spontaneous or post traumatic blisters. Neonatal period and infancy are critical since this autonomic dominant affection usually improves with age. Cyclins seem to be efficient in some cases of EBS but are prohibited in children younger than 8 years old. Erythromycin can be a good alternative in this population due to its antibacterial and anti-inflammatory potential.
The aim of this study is the evaluation of the efficiency of oral erythromycin to decrease the number of cutaneous blisters in severe EBS-DM patients from 6 months to 8 years old after 3 months of treatment.
Primary end point is the number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin.
It is a preliminary study on 8 patients. Treatment is oral erythromycin twice a day during 3 months. Follow up for each patient is 5 months. The duration of the study is 1 year.
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
- 6 Months 至 8 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Severe Dowling Meara EBS patients (2 or more new blisters a day)
- •signature of informed consent
- •Patient of 2 sexes
- •Age from 6 months to 8 years. From this age we consider that the patient will less need this treatment or can take cyclines.
- •Systematic Obtaining of the consent lit(enlightened) by the relatives(parents) of the child, after information about the objectives and the constraints of the study.
- •Agreement of the minor
- •Patient member to the Social Security
排除标准
- •Patient allergic to the erythromycin
- •Patient presenting an intolerance to the fructose, a syndrome of malabsorption some glucose and some galactose or a deficit sucrase-isomaltase
- •Renal and\or hepatic Insufficiency
- •Patient taking a medicine against indicated or misadvised in association with the erythromycin
研究组 & 干预措施
Oral erythromycin
Oral erythromycin
干预措施: Oral erythromycin (Drug)
结局指标
主要结局
number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin
时间窗: at 3 months of treatment
Principal end point is evaluated at inclusion and after one month of treatment, 3 months of treatment and 2 months after the end of the treatment
次要结局
- Secondary end points are : effect of 3 months of oral erythromycin on - Global tolerance of treatment.(at 3 months of treatment)
- Secondary end points are : effect of 3 months of oral erythromycin on - Involved area(at 3 months of treatment)
- Secondary end points are : effect of 3 months of oral erythromycin on - pruritus,(at 3 months of treatment)
研究者
Del Cont Delphine
Dr Christine CHIAVERINI
Centre Hospitalier Universitaire de Nice