TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONS
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Wound size reduction
研究概览
简要总结
The overall purpose of this study is to address whether topical gentamicin therapy is an effective and feasible treatment. Specifically, we will investigate the effect of non-intensive treatment (once daily or every other day) on skin protein expression, as well as quantify the effect on wound healing in patients with EB caused by PSC (part A). Furthermore, we will address in vitro whether gentamicin restores protein expression of genes affected by SSM in fibroblasts derived from skin biopsies obtained from patients with EB caused by SSM (part B). If these in vitro experiments yield positive results, the patients donating the cells will be offered to enter part A of this study. The overall duration of part A in this study is planned to be 18 weeks per patients and consists of a 6 weeks treatment period followed by a 12 week follow up period. Each patient will attend 3 study visits: at week 0, week 6 and week 18. All patients will be included within a time period of 12 months. The overall duration of part B will be up to 8 weeks per patients of which 4-7 weeks are spent to prepare fibroblasts obtained from skin biopsies. Then 5 days of in vitro intervention and subsequent analysis follows. Altogether, the duration of the GENTELBULL study will be 78 weeks or less.
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
盲法说明
Wound size calculations will be performed on digital photos, the outcome assessor will not know which wounds are treated and which are controls.
入排标准
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •The patient has EB caused by nonsense mutation
- •The patient has a symmetrical distribution of wounds which allows for wounds on one side of the body to serve as control wounds, while study therapy is applied to the other side.
- •The patient (if an adult) or parent(s)/guardian(s) is capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- •Women of childbearing potential must use a highly effective contraception method
排除标准
- •Known contact allergy against gentamicin sulfate or other ingredients in the ointment
- •Known intolerance to gentamicin sulfate of any sort
- •Moderate or severely reduced kidney function (eGFR <45)
- •Use other experimental therapy against EB
- •Receiving systemic aminoglycosides during the last 3 months
- •The patient uses muscle relaxant drug(s)
研究组 & 干预措施
Single arm
Each patient serves as its own control: one half is treated with the study treatment in addition to standard wound therapy, the other half receives standard wound therapy only.
干预措施: Gentamicin Sulfate (Drug)
结局指标
主要结局
Wound size reduction
时间窗: 6 weeks
Mean of ratios for wound areas (measured in cm2) at week 6 to that of week 0 of gentamicin-treated wounds, compared to the corresponding mean of ratios for standard care-treated wounds. The observed effect is considered significant if p\<0.05 (unpaired t-test). Of note, each patient may or may not reach the primary end point independent of the other participants
次要结局
- Healed skin robustness(18 weeks)
- Systemic gentamicin levels(6 weeks)
- Protein restoration by gentamicin(6 weeks)
研究者
Øystein Sandanger
Principal Investigator
Oslo University Hospital