ISRCTN15602229已完成未知
An open label Phase 2 clinical trial of retinal gene therapy for choroideremia using an adeno-associated viral vector (AAV2) encoding Rabescort protein 1 (REP1)
IHR Evaluation, Trials and Studies Coordinating Centre (NETSCC)0 个研究点目标入组 30 人开始时间: 2016年4月28日最近更新:
适应症
试验速览
- 阶段
- 未知
- 状态
- 已完成
- 发起方
- 入组人数
- 30
研究概览
简要总结
2024 Results article in https://doi.org/10.3310/WARA5730 (added 05/06/2024)
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- Male
入选标准
- •1. Willing and able to give informed consent for participation in the study
- •2. Male aged 18 years or above
- •3. Genetic or molecular confirmed diagnosis of choroideremia (REP1 protein deficiency)
- •4. Active disease visible clinically within the macula region
- •5. Best corrected visual acuity equal to or worse than 6/6 (20/20; Decimal 1.0; LogMAR 0) but better than or equal to 6/60 (20/200; Decimal 0.1; LogMAR 1.0) in the study eye
排除标准
- •1. Any female, or a male aged below 18 years
- •2. An additional cause for sight loss (e.g. amblyopia) in the eye to be treated
- •3. Any other significant ocular and non-ocular disease or disorder which, in the opinion of the investigator, may put the participants at risk because of participation in the study
- •4. Inability to take systemic prednisolone for a minimum of 3 weeks
- •5. Unwillingness to use barrier contraception methods for a period of three months following gene therapy surgery, if relevant
- •6. Participation in another research study involving an investigational product in the preceding 12 weeks
研究者
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