临床试验/NCT01014052

NCT01014052已完成1 期

Phase 1b Study to Evaluate QLT091001 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)

QLT Inc.13 个研究点分布在 5 个国家目标入组 32 人开始时间: 2009年11月1日最近更新: 2013年5月14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: 32
试验地点: 13
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 oral QLT091001
to evaluate whether 7-day treatment with oral QLT091001 can improve visual function in subjects with LCA or RP due to RPE65 or LRAT mutations
to evaluate duration of visual function improvement (if observed)

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 diagnosed with LCA or RP (with a mutation in either RPE65 or LRAT)
•Subjects with LCA must be 5-65 years of age
•Subjects with RP must be 18-65 years of age
•Subjects who have a "best-corrected" visual acuity of 3 letters or better (20/800 Snellen equivalent) or viable photoreceptors on OCT/FAF.

排除标准

•Subjects who are actively participating in an experimental therapy study or who have received experimental therapy within 60 days of Day
•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 a history of diabetes or chronic hyperlipidemia, hepatitis, pancreatitis, or cirrhosis.
•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

Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events(12 months)

研究者

发起方

QLT Inc.

申办方类型

Industry

责任方

Sponsor

研究点 (13)

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