A Controlled, Double-blind, Crossover Trial of Ganaxolone in Children With Fragile X Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 59
- 试验地点
- 2
- 主要终点
- Clinical Global Impression-Improvement (CGI-I) Scale
研究概览
简要总结
This Phase 2 proof-of-concept study is a double-blind, randomized, placebo-controlled, crossover study to investigate ganaxolone treatment in children with fragile x syndrome (FXS). The objective of the study is to assess the safety, tolerability and efficacy of ganaxolone in the treatment of anxiety and attention in subjects with FXS.
详细描述
This is a single center study conducted at University of California Davis (UCD) MIND Institute. Children with FXS between the ages of 6-17 years, inclusive will be randomized at a 1:1 ratio to receive ganaxolone or placebo treatment for 6 weeks, discontinue treatment and washout for 2 weeks, and then cross over to the opposite treatment for another 6 weeks.
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 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •molecular documentation of FMR1 full mutation
- •ages 6-17 yrs, inclusive
- •sexually active subjects are required to use a medically acceptable form of birth control
排除标准
- •non-English or Spanish speaking subjects
- •concomitant systemic steroid, vigabatrin, felbamate and ketoconazole
- •changes in medications within last 2 months
- •clinically unstable medical disease, progressive CNS disease/disorder
- •history of recurrent status epilepticus
- •unwilling to withhold grapefruit or grapefruit juice for the duration of the study
- •actively suicidal
研究组 & 干预措施
Ganaxolone then Placebo
Participants first received ganaxolone. They were first titrated up to the 12 milligram per kilogram (mg/kg) three times daily (tid) during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, participants received placebo for a duration of 6 weeks (from week 8 to 14).
干预措施: Ganaxolone (Drug)
Ganaxolone then Placebo
Participants first received ganaxolone. They were first titrated up to the 12 milligram per kilogram (mg/kg) three times daily (tid) during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, participants received placebo for a duration of 6 weeks (from week 8 to 14).
干预措施: Placebo (Drug)
Placebo then Ganaxolone
Participants first received placebo. They were first titrated up to the 12 mg/kg tid during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, participants received ganaxolone for a duration of 6 weeks (from week 8 to 14).
干预措施: Ganaxolone (Drug)
Placebo then Ganaxolone
Participants first received placebo. They were first titrated up to the 12 mg/kg tid during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, participants received ganaxolone for a duration of 6 weeks (from week 8 to 14).
干预措施: Placebo (Drug)
结局指标
主要结局
Clinical Global Impression-Improvement (CGI-I) Scale
时间窗: Week 14 (End of Treatment)
The CGI-I scale is a clinician-rated 7-point scale used to assess how much the participant's illness had improved or worsened relative to a Baseline state at the beginning of the intervention. It was rated as: 1. "very much improved"; 2. "much improved"; 3. "minimally improved"; 4. "no change"; 5. "minimally worse"; 6. "much worse"; 7. "very much worse". Higher scores indicated worse condition.
次要结局
- Visual Analogue Scale (VAS)(Week 14 (End of Treatment))
- Aberrant Behavior Checklist (ABC)(Week 14 (End of Treatment))
- Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV)(Week 14 (End of Treatment))
- Pediatric Anxiety Rating Scale (PARS) Total Score(Week 14 (End of Treatment))
- Anxiety, Depression, and Mood Scale (ADAMS)(Week 14 (End of Treatment))