临床试验/NCT01521793

NCT01521793已完成1 期

An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin: Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01)

QLT Inc.6 个研究点分布在 5 个国家目标入组 27 人开始时间: 2012年1月1日最近更新: 2014年7月28日

适应症

试验速览

阶段: 1 期
状态: 已完成
发起方: QLT Inc.
入组人数: 27
试验地点: 6
主要终点: Visual field

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

The purpose of this study is:

To evaluate the safety of up to 3 additional courses of oral QLT091001 administered once daily for 7 days in subjects treated previously with a single 7-day course of QLT091001 in Study RET IRD 01
To evaluate whether up to 3 additional courses of oral QLT091001 administered once daily for 7 days can maintain or improve visual function.

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

年龄范围: 5 Years 至 65 Years（Child, Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•Subjects with LCA or RP due to RPE65 or LRAT deficiency who received a 7-day treatment course of QLT091001 and completed the Day 30 visit in Study RET IRD 01
•Subjects who meet any one of the following criteria at least 1 month after start of the 7-day treatment course in Study RET IRD 01:
•no increase in GVF (in at least 1 eye): Follow-up GVF increased ≤20% from baseline or
•decrease in GVF (in at least 1 eye): Follow-up GVF decreased below the highest previous response by ˃20% or
•Considered a reasonable candidate for retreatment, based on the Investigator's discretion, in consultation with the independent DSMB and the Sponsor, based on regression or lack of response in other parameters of visual function (e.g., subjective reports of changes in color vision or adaptation to low light) but who do not meet other (GVF) criteria for study entry

排除标准

•Subjects with any clinically important abnormal physical finding at Screening.
•Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane,® Soriatane®) within 6 months of Day 0 and subjects who did not tolerate their previous oral retinoid medication will be excluded regardless of the time of last exposure.
•Subjects with liver failure, uncontrolled thyroid disease, hypersensitivity to retinoids, or hypervitaminosis A
•Subjects who have taken any supplements containing ≥10,000 IU vitamin A within 60 days of screening

结局指标

主要结局

Visual field

时间窗: 12 months

次要结局

Safety will be assessed by evaluating the following: adverse events, clinical laboratory tests, ECG's and vital signs(12 months)

研究者

发起方

QLT Inc.

申办方类型

Industry

责任方

Sponsor

研究点 (6)

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