Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3: A Pilot Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 13
- 试验地点
- 2
- 主要终点
- scale of rating of ataxia (SARA) score months,
研究概览
简要总结
There are no clinically established treatments which have been proven to delay the disease progression in spinocerebellar ataxia (SCA) 3. Most available treatments are only for symptom alleviation, and thus the majority of patients will eventually progress to needing and wheel chair and eventually bedridden.
As trehalose appear to be potentially promising treatment in SCA, the investigators aim to conduct this study using oral trehalose in our genetically confirmed SCA 3 patients.
详细描述
This prospective single arm interventional study involved 13 genetically confirmed spinocerebellar ataxia (SCA) 3 patients with no concomitant diabetes, over 6 months. Following baseline assessment, patients were instructed to ingest 100g of oral trehalose diluted in 500ml of water or other beverages daily. Assessments were performed at baseline, 2, 4 and 6 months using ataxia rating scales (SARA, SCAFI and INAS) and EQ-5D-3L scale for quality of life assessment.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •DNA diagnosis of SCA 3 in the study subject of his/ her affected family member(s)
- •Consent to participate in the study
- •The age of 18 years and older
排除标准
- •Unconfirmed SCA 3
- •Concomitant disorder(s) that affect SARA and other ataxia measures used in this study
- •Malabsorption of trehalose underlies intolerance to mushrooms, since the lack of absorption results in diarrhoea and intestinal distress.
- •Less than 18 years old
结局指标
主要结局
scale of rating of ataxia (SARA) score months,
时间窗: 2 monthly intervals for 6 months
Assessment of SARA scores by a single assessor
SCA Functional Index Scores
时间窗: 2 monthly intervals for 6 months
Assessment of SCAFI by a single assessor
EQ5D3L - quality of life scores
时间窗: 2 monthly intervals for 6 months
Assessment of quality of life scores
次要结局
- Blood investigation(At baseline and at 6 months)
- Side effects Profile(2 monthly intervals for 6 months)
研究者
Dr Norlinah Mohamed Ibrahim
Professor
National University of Malaysia