Clemastine Treatment in Individuals With Williams Syndrome- a Double-blind Placebo Control to Assess the Safety and Efficacy
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Neurocognitive measures
研究概览
简要总结
This study explores the neurobiological etiology of Williams syndrome and potential therapeutic targets for associated social, motor, and cognitive abnormalities. The main translational objective will be to test the effectiveness of Clemasntine on neurocognitive and other associated abnormalities in individuals with Williams syndrome.
详细描述
The overarching aim of the research proposed is to examine the safty of Clemastine use for individuals with Williams syndrome. Further, we wish to examine the relationship between Clemastine treatment and cognitive, motor and behavioral deficits in individuals with Williams syndrome. This study is an open-label study with a blinded randomize withdrawal. Each participant will go through a baseline evaluation (see study outcomes) and will be treated with the FDA approved drug Clemastine, in an age dependent dose (dosage table below). After a substantial improvement will be noticed, the participants will be divided into 2 groups, in a randomized, double-blind, placebo-control study design.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
盲法说明
This study is an open-label study with a blinded randomized withdrawal
入排标准
- 年龄范围
- 6 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Individuals with Williams syndrome, which has been confirmed by genetic testing.
- •Normal values in safety variables (e.g. Normal ECG 120-129/80-84).
- •No change in psychotropic medications and dosage during the last 4 weeks.
- •During the study, no pharmacological change that may impact the study (e.g. ADHD
- •medications).
排除标准
- •Individuals with another genetic disorder besides Williams syndrome.
- •Individuals with Williams syndrome, younger than 6 or older than 30 years old.
- •Significant change in normal values in safety variables (e.g. high or low ECG).
- •Change in medications and dosage during the last 4 weeks prior the beginning and
- •during the study.
- •Pregnancy.
- •Using addictive substances such as alcohol.
研究组 & 干预措施
Open Label
干预措施: Open Label Clemastine with a blinded randomize withdrawal (Drug)
Blinded randomize withdrawal
干预措施: Open Label Clemastine with a blinded randomize withdrawal (Drug)
结局指标
主要结局
Neurocognitive measures
时间窗: December 2024
Wechsler Intelligence Scale: The standard age-appropriate Wechsler scale. Wechsler Intelligence Scale for Children, 4th edition(Wechsler 1991, Wechsler 2003) for subjects 17 years and younger and the Wechsler Adult Intelligence Scale, 3rd edition (WAIS III)(Wechsler 1997) for subjects older than 17 years. NIH ToolBox (NIHTB): the NIHTB is a computerized neurocognitive battery developed by NIH that was designed to include measures of cognitive flexibility and inhibitory control, as well as a measure of working memory. http://www.healthmeasures.net/explore-measurement-systems/nih-toolbox/
次要结局
- Motor and Neurological assessment. Motor and Neurological assessment. Motor and Neurological assessment. Motor and neurolocgial measures(December 2024)
研究者
Prof. Doron Gothelf MD
Head of The Child and Adolescent Psychiatry Unit
Sheba Medical Center