Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score
研究概览
简要总结
The purpose of this study is to examine the safety and efficacy of Allopregnanolone as a possible treatment for symptoms of Fragile X-associated Tremor/Ataxia Syndrome (FXTAS).
详细描述
This study includes a screening visit with several assessments, followed by an open-label medication trial of Allopregnanolone for 12 weeks and an end-point evaluation to assess for changes. Assessments include blood draws for genetic and safety laboratory testing, neurological and physical exam and medical history, cognitive testing, and motor testing.
Study record was updated in October 2018 to include adverse events and outcome measure reporting. Study record was updated in November 2018 in response to requests to (1) specify time frame of reported outcome measures, (2) clarify that the RASS was a safety monitoring tool, not a prespecified outcome measure, and as such will not be reported as an outcome measure, and (3) upload a version of the study protocol and statistical analysis plan with the required title page and statistical analysis plan information.
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
- 50 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Allopregnanolone
Subjects will receive an intravenous infusion of Allopregnanolone at escalating doses of 2mg, 4mg, and 6mg once weekly over a three week period. The highest dose tolerated without sedation will be held stable for the remaining weekly infusions, for a total of 12 infusions.
干预措施: Allopregnanolone (Drug)
结局指标
主要结局
California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score
时间窗: Baseline/pre-treatment and 14 weeks/post-treatment
California Verbal Learning Test II (CVLT2) is an assessment measuring working memory. Trials 1-5 measure the total number of words remembered after 5 repeated trials and are summed to generate a raw score (called Trial 1-5 Free Recall Total Raw Score) ranging from 0 to 80, with higher scores reflecting better working memory. Mean and standard deviation for raw score at baseline/pre-treatment and at 14 weeks/post-treatment are presented here.
次要结局
- Behavioral Dyscontrol Scale - 2 (BDS-2) Total Score(Baseline/pre-treatment and 14 weeks/post-treatment)
- CATSYS Dot-to-Dot Tremor Intensity (CATSYS DTD TI)(Baseline/pre-treatment and 14 weeks/post-treatment)
- Hippocampal Volume, as Measured by Structural MRI(Baseline/pre-treatment and 14 weeks/post-treatment)
研究者
Randi J. Hagerman, MD
Medical Director, MIND Institute
University of California, Davis