Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- Mean change in visual field area from baseline to 48 weeks
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of oral valproic acid to slow the progression of visual function and/or to improve the visual function in patients with retinitis pigmentosa (RP).
Enrolled subjects in valproic acid group will be treated with oral valproic acid 500mg daily for 48 weeks. Visual function and safety will be assess before and after treatment (48 weeks) between valproic acid and control groups.
详细描述
This study is designed as a single-site, interventional, prospective, non-randomized, controlled study of 200 participants. Patients that participate in the study will be assigned to either valproic acid group or control in a 3:1 ratio.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of retinitis pigmentosa (RP) established by night blindness, visual field constriction, marked reduction of electroretinogram, and the clinical signs of RP in fundus examination
- •Best corrected visual acuity of 20/200 or more on a Snellen chart in at least one eye
- •Intact visual field of 5 or more as measured by the kinetic perimetry
- •Understand and sign the IRB-approved informed consent document for the study
- •Body weight: male (40 kg to 100 kg), female (40 kg to 80 kg)
- •Must be able to swallow tablets
- •Female subjects of childbearing potential must commit to practice acceptable methods of contraception
排除标准
- •Pregnant women
- •Lactating mothers
- •Medical problems that make consistent follow-up over the treatment period unlikely (e.g., stroke, myocardiac infarction, malignancy) or severe systemic disease
- •Other ocular disease: retinal disease other than RP or cystoid macular edema, glaucoma, cataract worse than +2PSC or infectious corneal disease
- •Coagulation disorder or bleeding-tendency
- •Liver dysfunction
- •Renal dysfunction
- •History of pancreatitis
- •History of neurological disorders including epilepsy, history of brain injury or any organic brain disorders
- •History of mental disorders including schizophrenia, bipolar disorder, or suicidality
- •Currently receiving valproic acid or other anti-convulsants
- •Has taken one of the following drugs at least 4 weeks prior to enrollment as these drugs are specifically known to affect the progression of RP: vitamin A, lutein, omega-3 fatty acid, or any antioxidant which affect the blood flow of retina or retinal function.
研究组 & 干预措施
Valproic acid
干预措施: Valproic Acid (Drug)
结局指标
主要结局
Mean change in visual field area from baseline to 48 weeks
时间窗: Baseline, week 24, and week 48
Visual field area will be measured using kinetic perimetry (Goldmann perimetry) or static perimetry including the central 30 field.
次要结局
- Mean change in central macular thickness(Baseline, week 24, and week 48)
- Mean change in best corrected visual acuity (BCVA)(Baseline, week 24, and week 48)
- Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude(Baseline and week 48)
- Mean change in total score on vision-related quality of life(Baseline and week 48)
- Mean change in fundus appearance(Baseline and week 48)
- Occurrence of adverse effect related to Valproic acid(Baseline through 48 weeks)
- Mean change in central macular volume(Baseline, week 24, and week 48)
- Changes in clinical laboratory data(Baseline through 48 weeks)
研究者
Hyeong Gon Yu
Professor
Seoul National University Hospital