Natural History of the Progression of Choroideremia Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 319
- 试验地点
- 1
- 主要终点
- Change in Best-Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (EDTRS)
研究概览
简要总结
The objective of this natural history study is to gain a better understanding of the progression of choroideremia (CHM) and add to the knowledge base for this rare disease.
详细描述
This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Are willing and able to provide informed consent for participation in the study.
- •Have a clinical phenotype and confirmed genetic diagnosis of CHM.
- •Have active disease clinically visible within the macular region.
- •Are willing and able to undergo ophthalmic examinations once every 4 months for up to 20 months.
- •Have a BCVA better than or equal to 6/60 (20/200; decimal 0.1; LogMAR 1.0; 34-38 ETDRS letters) in at least one eye.
排除标准
- •Have a history of amblyopia in the eligible eye.
- •Have any other significant ocular or non-ocular disease/disorder in the eligible eye which, in the opinion of the investigator, may put the subject at risk because of participation in the study, influence the results of the study or influence the subject's ability to participate in the study.
- •Have participated in an interventional research study in the past 6 months.
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
结局指标
主要结局
Change in Best-Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (EDTRS)
时间窗: Up to Month 20
BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If \<20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved.
次要结局
- Change from Baseline in Reading Performance using International Reading Speed Texts (IReST)(Baseline up to Month 12)
- Change from Baseline in Visual Fields(Baseline up to Month 12)
- Change from Baseline in Retinal Thinning Using Spectral Domain Optical Coherence Tomography (SD-OCT)(Baseline up to Month 20)
- Change from Baseline in Area of Viable Retinal Tissue Using Fundus Autofluorescence(Baseline up to Month 20)
- Change from Baseline in Contrast Sensitivity(Baseline up to Month 12)
- Change from Baseline in Color Vision(Baseline up to Month 12)
- Change from Baseline in Retinal Sensitivity Using Microperimetry(Baseline up to Month 20)