临床试验/NCT06563414

NCT06563414招募中不适用

A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)

Krystal Biotech, Inc.1 个研究点分布在 1 个国家目标入组 40 人开始时间: 2024年8月2日最近更新: 2025年7月2日

适应症

试验速览

阶段: 不适用
状态: 招募中
发起方: Krystal Biotech, Inc.
入组人数: 40
试验地点: 1
主要终点: Corneal abrasion frequency

研究概览研究设计入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB).

详细描述

The objective of this study is to characterize the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB). Patients will be followed via remote weekly symptom diaries and monthly questionnaires to assess corneal abrasion symptoms, frequency, and duration.

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

•The subject or their respective legal guardian must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent/Assent Form and must be able to and willing to follow study procedures and instructions.
•Age 6 months or older at time of Informed Consent/Assent.
•Prior genetic diagnosis of DEB due to mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

排除标准

未提供

结局指标

主要结局

Corneal abrasion frequency

时间窗: Baseline through up to 1 year

Frequency of corneal abrasion symptoms

次要结局

Epidermolysis Bullosa Eye Disease Index Symptom Score(Baseline through up to 1 year)

研究者

发起方

Krystal Biotech, Inc.

申办方类型

Industry

责任方

Sponsor

研究点 (1)

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