NCT02331173已完成不适用
Clinical Evaluation of Patients With X-linked Retinoschisis (XLRS)
Applied Genetic Technologies Corp3 个研究点 分布在 1 个国家目标入组 66 人开始时间: 2012年11月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 66
- 试验地点
- 3
- 主要终点
- Disease progression in subjects with XLRS
研究概览
简要总结
The purpose of this study is to evaluate subjects with X-linked retinoschisis in a clinical setting to collect data on disease progression.
详细描述
The objective of the study is to evaluate subjects with XLRS in a clinical setting and gather data on disease progression. The data from this study will enhance the understanding of the natural history of this rare disease and will facilitate appropriately powered safety studies in a future gene therapy trial in humans.
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
- 7 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Main Study:
- •Clinical diagnosis consistent with XLRS
- •Documented disease causing RS1 mutation
- •7 years of age or older
- •Able to provide informed consent/assent
- •CAI sub-study:
- •Presence of maculoschisis
排除标准
- •Main Study:
- •Other eye diseases that might affect the results (e.g. history of retinal detachment, glaucoma, cataracts that prohibit imaging, or any other eye pathology that in the opinion of the investigator would preclude enrollment)
- •CAI Sub-study:
- •Exclusion Criteria
- •Already being treated with CAIs
- •Previous documented failure to respond to CAI treatment
- •Any drug-specific contraindication/precaution listed below (from www.micromedex.com):
- •Topical Eye Drop Dorzolamide Hydrochloride
- •Contraindications:
- •hypersensitivity to dorzolamide products, including sulfa allergies
- •Precautions:
- •dorzolamide is a sulfonamide that is absorbed systemically, sulfonamide hypersensitivity reactions may occur
- •angle-closure glaucoma
- •concomitant use of oral carbonic-anhydrase inhibitors
- •conjunctivitis and lid reactions reported with chronic administration
- •moderate to severe renal (CrCl less than 30 mL/min) or hepatic insufficiency
- •ocular infection or inflammation
- •recent ocular surgery
- •Topical Eye Drop Brinzolamide
- •Contraindications:
- •hypersensitivity to any component of the product, including sulfa allergies
- •Precautions:
- •concomitant use of oral carbonic anhydrase inhibitors is not recommended
- •contact lens use; remove contact lenses prior to administration, allow 15 minutes before reinsertion
- •hypersensitivity to sulfonamides; severe reaction may occur; discontinue if signs or symptoms appear
- •low corneal endothelial cell counts; increased risk of corneal edema
- •renal impairment, severe (CrCl less than 30 mL/min); use not recommended
研究组 & 干预措施
Carbonic anhydrase inhibitor sub-study
Subjects with maculoschisis may be offered topical treatment with CAIs. These subjects will be asked to visit the study site at 1 month and 3 months after starting topical treatment. During these additional visits, subjects will undergo a dilated eye exam, BCVA, and SD-OCT imaging to assess efficacy of treatment (i.e. reduction of maculoschisis).
干预措施: Dorzolamide 2% TID or brinzolamide 1% TID (Drug)
结局指标
主要结局
Disease progression in subjects with XLRS
时间窗: Every 6 months for 18 months
次要结局
- Disease progression using microperimetry, a non-standard of care visual function test(Every 6 months for 18 months)
- Disease progression using electroretinograms (ERGs), a non-standard of care visual function test(Every 6 months for 18 months)
- Disease progression using the reading speed test, a non-standard of care visual function test(Every 6 months for 18 months)
- Disease progression using the contrast sensitivity test, a non-standard of care visual function test(Every 6 months for 18 months)
- Disease progression using a quality of life questionnaire(Every 6 months for 18 months)
- Change in maculoschisis while receiving carbonic anhydrase inhibitors(All study visits, Months 1, 3, 6, 12, and 18)
- Change in visual function while receiving carbonic anhydrase inhibitors(All study visits, Months 1, 3, 6, 12, and 18)
研究者
研究点 (3)
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