An Open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 4
- 主要终点
- Change from baseline in best corrected visual acuity in the treated eye
研究概览
简要总结
The assessment of the efficacy (with respect to preservation of visual function and retinal structure) and safety of a single subretinal injection of AAV2.REP1 in participants with a confirmed diagnosis of choroideremia, as evaluated by various functional and anatomical outcomes measured over a number of time points up to 24 months post-treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
盲法说明
The study is designated as Open Label with no masking. However, in order to minimise bias evaluation of the treated eye and untreated fellow eye (control eye), the ophthalmic assessments (visual acuity, microperimetry, fundus autofluorescence, etc.) will be conducted by an appropriately qualified masked observer once the participant's treated eye has had time to heal after the surgical procedure and has regained its normal appearance and function.
入排标准
- 年龄范围
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Candidate is willing and able to give informed consent for participation in the study.
- •Male aged 18 years or above.
- •Genetic or molecular confirmed diagnosis of choroideremia (REP1 protein deficiency).
- •Active disease visible clinically within the macula region.
- •Best corrected visual acuity better than or equal to 6/60 (20/200; Decimal 0.1; LogMAR 1.0) in the study eye.
排除标准
- •Any female, or a male aged below 18 years.
- •An additional cause for sight loss (e.g. amblyopia) in the eye to be treated.
- •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.
- •Inability to take systemic prednisolone for a period of 45 days.
- •Unwillingness to use barrier contraception methods for a period of three months following gene therapy surgery.
- •Participation in another research study involving an investigational product in the preceding 12 weeks.
结局指标
主要结局
Change from baseline in best corrected visual acuity in the treated eye
时间窗: 2 years
次要结局
- Change from baseline in the central visual field in the treated eye as determined by microperimetry(2 years)
- Change from baseline in the area of surviving retinal pigment epithelium in the treated eye as measured by fundus autofluorescence, compared to the untreated fellow eye (control eye) after randomisation of treatment to one eye or the other(2 years)