Assessment of response to levodopa in persons with spinocerebellar ataxia 12 with clinical parkinsonism - a single arm interventional study
试验速览
- 阶段
- Phase 3 4
- 状态
- 尚未招募
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- change in MDS UPDRS part III score from baseline at three months of levodopa therapy among patients with genetically proven Spino cerebellar ataxia 12 with clinical parkinsonism.
研究概览
简要总结
SCA 12 Iis a rare autosomal dominant inherited ataxia due to expanded cag trinucleotide repaets in PPP2R2B gene located on chromosome 5q31. It is prevalent among the agarwal community. Currently there is no definite cure for SCA 12. In the absence of definitive curative therapy, symptom amelioration is of utmost importance. Parkinsonian features including rigidity bradykinesia, and rest tremor may be highly disabling and thus addressing them efficiently is likely to positively impact quality of life. Response to levodopa in persons with SCA 12 WITH PARKINSONISM HAS NEVER BEEN SSTUDIED. iN ADDITION dat IMAGING FINDINGS IN sca 12 are not delineated in current literature. Hence we aim to assess response to levodopa therapy in persons with SCA 12 with clinical parkinsonism to assess impact of levodopa therapy on quality of life , non motor symptoms, tremors with levodopa treatment and inform DAT findings.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 盲法
- None
入排标准
- 年龄范围
- 18.00 Year(s) 至 90.00 Year(s)（—）
- 性别
- All
入选标准
- •All adult patients with genetically proven diagnosis of SCA 12 •Presence of clinical features of parkinsonism, as assessed and agreed upon by two movement disorder experts, according to the UK Brain Bank criteria for diagnosis of parkinsonism •Patients on a stable dose of symptomatic medication (e.g. beta-blockers for tremor) for three months prior to enrolment.
排除标准
- •Participants meeting any one of the following will be excluded .
- •1.Patients with history of use of any antipsychotic medications, or drugs that can cause drug-induced parkinsonism like D2-blockers etc.
- •in the past 2.Pregnant and lactating females 3.Chronic diseases like Chronic Kidney Disease, Chronic Liver Disease 4.Known allergies or intolerance to levodopa therapy 5.Patients already on levodopa therapy at the time of recruitment 6.Patients who are unwilling to participate.
结局指标
主要结局
change in MDS UPDRS part III score from baseline at three months of levodopa therapy among patients with genetically proven Spino cerebellar ataxia 12 with clinical parkinsonism.
时间窗: baseline and at three months
次要结局
- Striatal outcomes using DAT scan in persons with SCA 12(at baseline)
- To assess improvement from baseline in Quality of life using SF 36 , Tremors(tetras score), Non motor symptoms score(at baseline and 3 months)
- Correlation between CAG trinucleotide repeats and Levodopa response(at baseline and at 3 months)
研究者
Dr Lekshmi S H
AIIMS New delhi