A Double-Blind, Placebo-Controlled, Crossover, Flexible-Dose Evaluation of the Efficacy, Safety and Tolerability of STX209 in the Treatment of Irritability in Subjects With Fragile X Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 63
- 试验地点
- 12
- 主要终点
- Aberrant Behavior Checklist Irritability Subscore
研究概览
简要总结
The study objective is to explore the efficacy, safety and tolerability of STX209 for treatment of irritability in subjects with FSX. We hypothesize that STX209 will improve irritability and other typical problem behaviors associated with fragile X syndrome. We also hypothesize that STX209 will be safe and well tolerated.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 40 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects 12 to 40 years of age eventually expanding to 6 years of age
- •Molecular documentation of the fragile X mutation.
- •Clinical Global Impression - Severity (CGI-S) rating for problem behavior of moderate or higher at screening and at Visit 1
- •An Aberrant Behavior Checklist (ABC-C) Irritability Subscale score >12 and at least 3 items on the Irritability Subscale rated at least moderate or above.
- •Current treatment with no more than three psychoactive medications, including anti-epileptics.
- •Current pharmacological treatment regimen has been stable for at least 4 weeks.
排除标准
- •Subjects with a history of seizure disorder who are not currently receiving treatment with antiepileptics.
- •Subjects with any condition, including alcohol and drug abuse, which might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. This includes, but is not limited to impairment of renal function, evidence or history of malignancy or any significant hematological, endocrine, cardiovascular, respiratory, hepatic, or gastrointestinal disease.
- •Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
- •Subjects who are currently receiving treatment with racemic baclofen.
- •Subjects currently treated with vigabatrin or tiagabine.
- •Subjects taking another investigational drug currently or within the last 30 days.
研究组 & 干预措施
STX209
STX209 variable dose from 1mg bid to 10mg tid, capsule, oral, 4 weeks
干预措施: STX209 (Drug)
Placebo
variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
Aberrant Behavior Checklist Irritability Subscore
时间窗: After 4 weeks of treatment
The Aberrant Behavior Checklist-Community Edition (ABC-C) is a 58-item questionnaire composed of five different independent subscales. The questionnaire is completed by the parent/caregiver and lists aberrant behaviors and asks about the severity of the problem. ABC-Irritability is one of the subscales and comprises of 15 items. Minimum score is 0, maximum is 45. A decreased score indicates few aberrant behaviors and clinical improvement. The entire ABC-C assessment is administered at baseline and then at the end of each Intervention Period (4 weeks after Baseline).
次要结局
未报告次要终点