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)
适应症
相关药物
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
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
研究点 (6)
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