Interventional Study of Levodopa Replacement on Retinal Function in Oculocutaneous Albinism
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Visual Acuity Change
研究概览
简要总结
The purpose of the study is to evaluate and document physiologic and functional changes in visual performance and retinal function of patients diagnosed with albinism (a dopamine deficiency state) following a trial of oral Levodopa/carbidopa treatment.
详细描述
In this study the investigators propose that the retina itself in albinism is deficient in dopamine, and vision improvement will occur as a result of improved retinal function in response to the deficient neurotransmitter dopamine. This study has a pretest-posttest design in order to determine if improvement in vision is in response to replacement of deficiency (dopamine). The electroretinography (ERG) testing and optical coherence tomography (OCT) will be critical determinants to confirm vision improvement as a result of improved retinal function, but are not primary outcome data. Main outcome measures will be collected at pre-treatment, 1 month, 3 months, and 4 months. Change in visual acuity as measured in logMAR by Snellen or sweep visual evoked potential (SVEP) after 3 months of treatment is the primary outcome. Patients include OCA1a patients, OCA1b, OCA2, and unclassified Oculocutaneous albinism (OCA). OCA1a patients clinically are known to have the worst vision, and physiologically have the lowest (or absent) levels of tyrosinase function (Dopamine Production). All patients will be treated with Levodopa/carbidopa 4mg/kg/day in three divided doses.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 —(Child, Adult, Older Adult)
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •clinical diagnosis of oculocutaneous albinism
- •age over 3 and weight over 25 lbs.
排除标准
- •ocular only albinism
- •ocular pathology other than albinism
- •neurologic disease, history of myocardial infarction, history of clinical depression, pregnancy
研究组 & 干预措施
Levodopa/carbidopa 4mg/kg/day
Treatment drug taken orally three times daily
干预措施: Levodopa/carbidopa (Drug)
结局指标
主要结局
Visual Acuity Change
时间窗: 3 months
Change in visual acuity from baseline to 3 months as measured in logMAR by Snellen or Sweep visual evoked potential (SVEP). logMar lower values equals better visual outcome.
次要结局
未报告次要终点
