Riluzole in Fragile X Syndrome: A Pilot Study Incorporating Biomarker Assay
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Clinical Global Impression-Improvement (CGI-I)
研究概览
简要总结
The purpose of this study is to investigate the effectiveness and tolerability of riluzole in adults with Fragile X Syndrome.
详细描述
Fragile X Syndrome (FXS) represents the most common inherited form of intellectual disability. FXS is more common in males and the symptoms associated with the disorder are more marked in males. FXS is associated with characteristic physical features, behaviors, and comorbidities. Those with FXS often suffer from behavioral difficulties that include anxiety-related symptoms (shyness, social phobia, obsessive-compulsive disorder (OCD) symptoms), attention deficit hyperactivity symptoms (overarousal, hyperactivity, distractibility, impulsivity) and aggressive/self-injurious behaviors.
Riluzole is approved by the FDA for use in treating amyotrophic lateral sclerosis (ALS) in adults. Recently, riluzole has been the subject of several open-label studies describing the use of the drug in treatment-resistant depression and OCD.
Given the overlap between repetitive behavior in FXS and symptoms of OCD, it is logical to study riluzole in FXS given the compound's promise in ameliorating treatment-refractory symptoms of OCD.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females age 18 years or older.
- •Confirmed molecular diagnosis of Fragile X Syndrome.
- •Clinical Global Impression Severity (CGI-S) score of 3 or greater.
- •Significant interfering repetitive behavior as determined by the principal investigator.
- •Must be in good health as determined by screening procedures including a detailed medical history, and complete physical and neurological examination.
- •Dosing of concomitant medications during the study must remain stable.
排除标准
- •Pregnancy.
- •Concomitant use of another glutamatergic agent (memantine, topiramate, amantadine, among others.
- •Evidence of prior trial of riluzole and/or hypersensitivity/allergic reaction to riluzole.
- •Abnormal baseline liver function tests at screen or by history; or complete blood count abnormalities at screen or by history.
研究组 & 干预措施
Riluzole
Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.
干预措施: Riluzole (Drug)
结局指标
主要结局
Clinical Global Impression-Improvement (CGI-I)
时间窗: Obtained at Week 6
The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. and rated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
时间窗: Obtained at Baseline and Week 6
The CY-BOCS PDD has been utilized in a largescale clinical treatment study of repetitive behavior in idiopathic ASDs. CYBOCS-PDD scores range from 0 to 20 and measure repetitive/compulsive behavior and not obsessions. Higher score indicate worse outcome.
次要结局
- Aberrant Behavior Checklist(Week 6)
- The Clinical Global Impression - Severity Scale(Week 6)
- The ADHD Rating Scale(Week 6)
- The Peabody Picture Vocabulary Test(Week 6)
- The Social Reciprocity Scale(Week 6)
- Extra-cellular Signal-relatedness Kinase (ERK)(Screen and Week 6)