Pilot Study of Acamprosate in Youth With Fragile X Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Clinical Global Impression- Severity Scale (CGI-S)
研究概览
简要总结
Fragile X syndrome (FXS) is the most common inherited form of developmental disability. FXS is inherited from the carrier parent, most often the mothers. FXS is associated with severe interfering behavioral symptoms which include anxiety related symptoms, attention deficit hyperactivity, and aggressive behaviors. Approximately 25-33% of individuals with FXS also meet criteria for autistic disorder. The hypothesis of this study is that treatment with acamprosate will reduce inattention/hyperactivity, language impairment, irritability, social deficits, and cognitive delay in youth with FXS. The purpose of this study is to investigate the effectiveness and tolerability of acamprosate in youth with Fragile X Syndrome and to assess the potential psychophysiological differences between FXS and autism spectrum disorders.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female outpatients between the ages of 5 and 17 years.
- •Confirmed diagnosis of Fragile X Syndrome based upon genetic testing.
- •Stable dosing of all psychotropic medications for at least 2 weeks prior to baseline.
- •Subjects with a stable seizure disorder or history of only childhood febrile seizures will be included.
- •Clinical Global Impression-Severity Score of 4 (Moderately Ill) or greater.
- •Must be in good physical health.
- •Subjects of child bearing age of both genders will be required to utilize birth control as applicable.
排除标准
- •Diagnosis of schizophrenia, another psychotic disorder, bipolar disorder or alcohol or other substance abuse based on Diagnostic and Statistical Manual Fourth Edition-Text Revised (DSM-IV-TR).
- •A significant medical condition such as heart, liver, renal or pulmonary disease or unstable seizure disorder.
- •Females with a positive urine pregnancy test
- •Creatinine clearance of less than
- •Concomitant use of another glutamatergic agent (memantine,riluzole, d-cycloserine, amantadine topiramate, gabapentin, among others.
- •Evidence of hypersensitivity to acamprosate or potentially serious adverse effect.
- •Subjects who, in the opinion of the investigator, are unsuitable in any other way to participate in this study including being unable to comply with the requirements of the study for any reason.
研究组 & 干预措施
Acamprosate
The maximum dose of acamprosate to be used in this study is 1998 mg per day for those subjects weighing greater than 60kg and 1332 mg per day for those less weighing less than 60kg.
干预措施: Acamprosate (Drug)
结局指标
主要结局
Clinical Global Impression- Severity Scale (CGI-S)
时间窗: Week 10
The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1, normal, not at all ill; 2, borderline mentally ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill.
次要结局
- The Aberrant Behavior Checklist (ABC)(Week 10)
- Social Responsiveness Scale(Week 10)
- Peabody Picture Vocabulary(Week 10)
- Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD(Week 10)
- ADHD Rating Scale 4th Edition(Week 10)
- Brain-derived Neurotrophic Factor (BDNF)(Screen and Week 10)
- Vineland Adaptive Behavior Scales-II (VABS-II) Communication Domain(Week 10)