NCT06591806进行中（未招募）不适用
A Multicenter, Prospective, Longitudinal, Observational Study in Children and Adults With Stargardt Disease Related Atrophy Secondary to Biallelic Mutations in the ABCA4 Gene
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 80
- 试验地点
- 4
- 主要终点
- To measure the change from baseline in Fundus Autofluorescence
研究概览
简要总结
This multicenter, prospective, longitudinal, observational study in approximately 80 subjects with Stargardt disease secondary to biallelic mutations in the ABCA4 gene (STGD1) aims to evaluate prognostic factors of disease progression, and to further characterize the patient population for future clinical studies.
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
- 8 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects between 8 and 50 years of age at the time of enrolment.
- •Willingness to adhere to the protocol as evidenced by written informed consent if the subject is 18 years or older. If the subject is under 18 years of age, written assent must be obtained from the subject and written informed consent must be obtained from the subject's legally authorized representative (parent or legal guardian).
- •Confirmed mutation in the ABCA4 gene.
排除标准
- •History of uveitis.
- •Any ocular disease in either eye that may confound assessment of the retina morphologically and functionally.
- •Any pathology of the posterior segment other than ABCA4 retinopathy.
- •Presence of any other genetic mutation(s) that have been associated with retinal or macular dystrophy.
结局指标
主要结局
To measure the change from baseline in Fundus Autofluorescence
时间窗: 24 months
次要结局
未报告次要终点
研究者
研究点 (4)
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