Open Label Safety and Tolerability Trial of CPP-109 (Vigabatrin) in Adults With Treatment Refractory Tourette's Disorder
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Global Severity Score on the Y-GTSS
研究概览
简要总结
The purpose of this study is to determine if vigabatrin, an unusual anti-seizure medication, will diminish the Tourette Disorder outbursts in young adults whose symptoms have persisted into adulthood and have not responded to usual treatment.
详细描述
The aims of this study are to 1) explore proof of concept that CPP-109 will reduce tics, and 2) to obtain systematic data regarding dosing, safety and tolerability of CPP-109 in adults with treatment refractory TD. We will obtain preliminary data on estimate of effect size for tics using Cohen's d, calculating the difference between the two means (baseline and endpoint scores on the YGTSS), divided by the standard deviation of the difference.
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 35 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects must be between 18 and 35 years of age (inclusive) when informed consent is obtained.
- •Subjects must meet full DSM-IV diagnostic criteria for TD by clinical interview on examination by a physician investigator, and confirmed by the Structured Clinical Interview for DSM (SCID-CT) for clinical trials.
- •Subjects will have failed to respond to an adequate trial, as determined by the investigator, of clonidine, guanfacine, and a first generation (typical) and second-generation (atypical) neuroleptic medication in the past.
- •Tics are causing significant distress or impairment, as determined by the subject and principal investigator, on the current treatment regimen.
- •Laboratory results, including serum chemistries, hematology, and urinalysis, must show no significant abnormalities (significant is defined as laboratory values requiring acute medical intervention).
- •Subjects will not undergo formal IQ testing, but must be of normal intelligence in the judgment of the investigator.
- •Subjects must possess an educational level, degree of understanding and command of the English language to enable them to communicate suitably with the investigators and study coordinator, and to understand the nature of the study.
- •Subjects must be considered reliable.
- •Written informed consent of subjects is obtained.
排除标准
- •Subjects with organic brain disease, for example, traumatic brain injury residua.
- •Subjects with a preexisting ophthalmologic condition.
- •Subjects with or at high risk of other types of irreversible vision loss or who require other drugs associated with serious adverse ophthalmic effects such as retinopathy or glaucoma.
- •Subjects meeting criteria for mental retardation as defined by the DSM-IV-TR.
- •Subjects with a history of seizure disorder (other than febrile seizure).
- •Subjects with history of Sydenham's Chorea.
- •Subjects with autism, schizophrenia, other psychotic disorder, or bipolar disorder.
- •Subjects with a primary diagnosis of a major mood disorder that requires ongoing psychiatric treatment.
- •Subjects with a neurological disorder other than a tic disorder.
- •Subjects with a major medical illness.
- •Female subjects who are unwilling to use birth control or who are pregnant, as determined by serum pregnancy test at baseline assessment, or lactating.
- •Subjects who have a past or current history of substance dependence and/or a current history of substance abuse or who fail baseline toxicology screen.
- •Subjects who have any clinically significant abnormal laboratory result at baseline screening including EKG, or blood tests.
- •Subjects who, in the opinion of the investigator, are unsuitable in any other way to participate in this study.
研究组 & 干预措施
Vigabatrin
3 tablets, bid for 8 weeks
干预措施: vigabatrin (Drug)
结局指标
主要结局
Global Severity Score on the Y-GTSS
时间窗: weekly from baseline to end of study (10weeks)
The Global Severity score is the sum of the Total Tic score and the TD Impairment score. It is rated by the Investigator on the Yale Global Tic Severity Score ( Y-GTSS, a widely accepted measure of drug efficacy in TD. Scale from 0- 100. Higher score indicates more impairment.
次要结局
未报告次要终点
研究者
Barbara J. Coffey
Professor
Icahn School of Medicine at Mount Sinai