Pilot randomised double blinded comparative trial to evaluate the efficacy of topical tacrolimus versus placebo in the management of itch in patients with recessive dystrophic epidermolysis bullosa
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- To evaluate the improvement of pruritus as determined by numeric rating scale and Leuven score for pruritus in patients with recessive dystrophic epidermolysis bullosa with the use of topical tacrolimus 0.03% for 3 months as compared to placebo.
研究概览
简要总结
Epidermolysis bullosa (EB) is a group of inherited skin disorders caused by mutations in genes that affect the dermal-epidermal junction, leading to fragile skin, blistering, scarring, and contractures. Itching (pruritus) is a particularly disabling symptom, especially in children with recessive dystrophic EB (RDEB), worsening the condition by causing more blisters and erosions. This study aims to evaluate the effectiveness of topical tacrolimus in managing pruritus in RDEB patients, with the goal of improving patient outcomes and quality of life.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 盲法
- Participant and Outcome Assessor Blinded
入排标准
- 年龄范围
- 6.00 Year(s) 至 60.00 Year(s)（—）
- 性别
- All
入选标准
- •Patients with a clinical diagnosis of recessive dystrophic epidermolysis bullosa, aged over six years, and experiencing moderate to severe pruritus with a Numeric Rating Scale score greater than 3.
排除标准
- •Patients of recessive dystrophic epidermolysis bullosa not complaining of pruritus.
- •Not willing to participate in study.
结局指标
主要结局
To evaluate the improvement of pruritus as determined by numeric rating scale and Leuven score for pruritus in patients with recessive dystrophic epidermolysis bullosa with the use of topical tacrolimus 0.03% for 3 months as compared to placebo.
时间窗: Baseline, 4 weeks, 8 weeks and 12 weeks
To assess the improvement in disease severity and quality of life as determined by EBDASI and EBQOL in patients with recessive dystrophic epidermolysis bullosa with the use of topical tacrolimus 0.03% for 3 months as compared to placebo
时间窗: Baseline, 4 weeks, 8 weeks and 12 weeks
次要结局
- Determination of severity of neuropathy as determined by nerve conduction and quantitative sensory testing with its correlation with severity of pruritus in patients with recessive dystrophic epidermolysis bullosa.(Quantitative assessment of pruritus related cytokines including IL-31, IL-33, IL-22, IL-4, IL-13, IL-17 and TSLP at baseline and at 3 months in patients with recessive dystrophic epidermolysis bullosa)
研究者
Dr. Neetu Bhari
AIIMS New Delhi