Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- Percent Change of Active Blisters and in Total Blister/Erosion Counts
研究概览
简要总结
This is a feasibility study to see if Granulocyte Colony Stimulating Factor (GCSF) is effective as a treatment of Dystrophic Epidermolysis Bullosa (EB). Patients will receive one course of treatment with the study drug. The course will be 7 days in length. After receiving GCSF, patients will be followed at 7 and 30 days following the discontinuation of the drug. Thirty day follow up can be done via telephone communication with the patient or family.
详细描述
Each patient will be given 10 micrograms per kilogram per day of G-CSF subcutaneously for 6 consecutive days. On day 7 each patient will be seen and evaluated in the same manner as on day 0. Patients or their parents (if children are too young to reliably respond themselves) will also be asked to rate the following via a visual analog scale of 1-9- oral pain, pruritus, oral pain, swallowing, and overall sense of well-being. A telephone follow-up will be conducted on all patients 28 days after G-CSF so as to evaluate if the effect noted on day 7 was sustained.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Each patient must have the diagnosis of severe generalized recessive dystrophic EB (formerly known as Hallopeau-Siemens RDEB) confirmed by clinical criteria and either of the following:
- •transmission electron microscopy
- •immunofluorescence antigenic mapping and type VII collagen monoclonal antibody staining
- •COL7A1 mutational analysis
排除标准
- •The patient must not have a history of squamous cell carcinoma or any internal malignancy.
- •Female patients who are pregnant.
- •Patients with active signs and symptoms of infection.
研究组 & 干预措施
Granulocyte Colony Stimulating Factor (GCSF)
GCSF 10mcg/kg/d subcutaneously (SQ) for 7 days
干预措施: Granulocyte Colony Stimulating Factor (GCSF) (Drug)
结局指标
主要结局
Percent Change of Active Blisters and in Total Blister/Erosion Counts
时间窗: 7 days
Percent change of active blisters and in total blister/erosion counts from baseline to 7 days
次要结局
- Surface Area of Nonhealing Erosions(7 days)
- Overall Improved Symptomatology(28 days)
研究者
Haydar Frangoul
Professor of Pediatrics
Vanderbilt University Medical Center