Randomized Double-Blind Controlled Cross Over Trial of Minocycline in Children With Fragile X Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 66
- 试验地点
- 1
- 主要终点
- Visual Analogue Scale- Behavior 1
研究概览
简要总结
This is a single center study at the UC Davis MIND Institute in patients age 3.5-16 years of age with fragile X syndrome (FXS), funded by a National Fragile X Foundation Grant. It is a controlled trial of minocycline, an antibiotic commonly used in children for infection or for treatment of neurodegenerative disorders. We are investigating its use in FXS because it lowers matrix metalloproteinase 9 (MMP9) levels, which are high in FXS, and it also strengthens brain connections in the animal models of FXS. We hypothesize that minocycline will likely be helpful for language, behavior and/or cognition in fragile X patients.
详细描述
This is a single center study at the UC Davis MIND Institute in patients age 3.5-16 years of age with fragile X syndrome (FXS), funded by a National Fragile X Foundation Grant. It is a controlled trial of minocycline, an antibiotic commonly used in children for infection or for treatment of neurodegenerative disorders. We are investigating its use in FXS because it lowers matrix metalloproteinase 9 (MMP9) levels, which are high in FXS, and it also strengthens brain connections in the animal models of FXS. We hypothesize that minocycline will likely be helpful for language, behavior and/or cognition in fragile X patients.
The aim of this study is to carry out a double-blind placebo controlled trial of minocycline treatment in children with FXS who are 3.5 to 16 years of age. At baseline, we will assess behavior and perceptual and cognitive development. After the children have been treated for 3 months with either minocycline or placebo, they undergo the same baseline testing. They will then cross over and be treated for a second 3 months. We will carry out testing again at the end of the second 3 month period. We will also assess the side effects of minocycline treatment throughout the study.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 42 Months 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must have fragile X syndrome with molecular documentation
- •Current pharmacological treatment regimen has been stable for at least 4 weeks
排除标准
- •Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study
- •subjects who are unable to take oral medication
- •subjects who have been on minocycline previously
- •subjects who are allergic to minocycline or tetracyclines
- •subjects who are pregnant
- •subjects with history of lupus or hepatic dysfunction
研究组 & 干预措施
Placebo pill
All patients will be on placebo for 3 months in this crossover study.
干预措施: Placebo (Drug)
Minocycline
All patients will be on minocycline for 3 months in this crossover trial.
干预措施: minocycline hydrochloride (Drug)
结局指标
主要结局
Visual Analogue Scale- Behavior 1
时间窗: Baseline, 3 months, 6 months
A VAS is used to represent a caregiver's assessment of given behaviors, which were chosen by the parents. Caregivers marked a 10 cm horizontal line representing a visual continuum of each behavior from "worst behavior" to "behavior not a problem." Greater values indicate greater improvement. This measure represents the first behavior that the caregivers noted, out of three.
Clinical Global Impression Scale (CGI)
时间窗: 3 months (post first treatment) and 6 months (post second treatment)
The CGI-I utilizes history from primary caregivers and incorporates it into a seven step clinical rating for follow up throughout treatment, from 1 "very much improved" to 7 "very much worse". Lower scores indicate more improvement. Scores were obtained post treatments. Scores from when the patients were on minocycline either first or second were combined and averaged to determine a least squares mean and placebo scores were obtained in the same manner.
次要结局
- VAS Categorized by Behavior: Aggression/ ADHD(Baseline, 3 months, 6 months)
- Visual Analogue Scale- Behaviors 2(Baseline, 3 months, 6 months)
- Visual Analogue Scale Behavior 3- VAS3(Baseline, 3 months, 6 months)
- VAS Categorized by Behavior:Language/ Cognition(Baseline, 3 months, 6 months)
- VAS Categorized by Behavior: Other(Baseline, 3 months, 6 months)
- Expressive Vocabulary Test-2(Baseline, 3 months and 6 months)
- Vineland Adaptive Behavior Scale-II (VABS-II)Adaptive Behavior Composite Score(Baseline, 3 months, and 6 months)
- Aberrant Behavior Checklist-Community Edition (ABC-C)Composite Score(Baseline, 3 months, and 6 months)
- VAS Categorized by Behavior:Anxiety/ Mood(Baseline, 3 months, 6 months)