A Prospective Study to Evaluate the Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Change in wound surface area
研究概览
简要总结
This study is a non-interventional, observational study that will evaluate the natural history of wounds in patients with Dystrophic Epidermolysis Bullosa (DEB) for inclusion into the Krystal Biotech Phase III protocol of B-VEC (previously KB103). Wound recurrence and wound size will be evaluated for up to four months.
详细描述
The objective of this study is to analyze the selected wounds' natural history prior to including the selected wounds in the evaluation of safety and efficacy in Krystal Biotech's Phase III protocol of B-VEC.
Subjects will be enrolled upon obtaining consent and meeting eligibility criteria.
Patient Screening is done on-site, followed by the imaging of at least 1 wound, but up to 15 wounds. All remaining imaging will be performed remotely with a smartphone or tablet application.
Patients are on-trial for approximately four months.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent is provided. Patients 18 years of age and older, and parent(s)/legal guardian(s) of patients younger than 18 years of age, must provide written informed consent prior to participating in the study; additionally, informed assent will be obtained from patients younger than 18 years of age as specified by local requirements.
- •Patient must have a documented diagnosis of DEB based on genetic analysis showing a mutation in COL7A1 or, in the opinion of the investigator, a confirmed diagnosis of DEB based on histologic criteria (antigen mapping or electron microscopy).
- •Age: 6 months and older
- •Patient must have at least 1 wound that is suitable for imaging, in the opinion of the investigator, at the time of enrollment.
- •Patient is willing and able to undergo the protocol-specified procedures.
排除标准
- •In the opinion of the investigator, inclusion poses an unacceptable risk to the patient or interpretation of these study data.
结局指标
主要结局
Change in wound surface area
时间窗: Up to four months
Percent change in wound surface area from baseline will be measured from images uploaded to an imaging application.
次要结局
- Duration of wound closure(Up to four months)
- Time to Closure(Up to four months)