Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 46
- 试验地点
- 2
- 主要终点
- Aberrant Behavior Checklist-Social Withdrawal subscale
研究概览
简要总结
In this research study we want to understand the effectiveness of a drug treatment, acamprosate, for interfering symptoms (i.e., inattention/hyperactivity, social impairment) associated with Fragile X Syndrome (FXS).
详细描述
Each subject with FXS will receive 10 weeks of blinded treatment with acamprosate or matching placebo. After completion of the double-blind phase, all subjects will have an opportunity to receive acamprosate as part of the study procedures for 16 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 23 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Diagnostic confirmation of full mutation FXS
- •Age ≥5 years and <23 years
- •General good health as determined by physical exam, medical history and laboratory work up.
排除标准
- •Use of more than two psychotropic medications (medications affecting behavior).
- •Unstable dosing of any psychotropic medication (medication affecting behavior)
- •Problems with kidney functioning
- •Unstable seizure disorder
- •Change in any anti-convulsant drug dosing in the 60 days prior to study entry
- •Prior adequate treatment trial with acamprosate as determined by the study doctor
- •Pregnant or lactating females
研究组 & 干预措施
acamprosate
The maximum dose of acamprosate to be used in this study is 666 mg three times daily (total 1998 mg/day) for subjects weight ≥ 50 kg and 1332mg for subjects that weigh < 50 kg.
干预措施: acamprosate (Drug)
Placebo
Placebo will be prescribed with the same frequency and duration as the acamprosate group.
干预措施: Placebo (Drug)
结局指标
主要结局
Aberrant Behavior Checklist-Social Withdrawal subscale
时间窗: Change from baseline to week 10
The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
次要结局
- Clinical Global Impressions- Improvement (CGI-I)(week 10)
- Aberrant Behavior Checklist- Hyperactivity (ABC-H)(Change from baseline to week 10)
- Aberrant Behavior Checklist-Social Avoidance (ABC-SA)(Change from baseline to week 10)