Evaluating the Neurophysiologic and Clinical Effects of Single-Dose Baclofen, Roflumilast, Memantine, and Placebo in Fragile X Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 45
- 试验地点
- 1
- 主要终点
- Change in EEG Relative Gamma Power
研究概览
简要总结
The aim of this study is to utilize neurophysiologic assessments, behavioral measures and clinical measures to assess how much deficits associated with Fragile X Syndrome from pre-dose to post-dose using pharmacology.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Basic Science
- 盲法
- None
盲法说明
Quadruple masking (participant, care provider, investigator and outcomes assessor)
入排标准
- 年龄范围
- 18 Years 至 45 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects ages 18-45, with FXS who completed the study entitled "Mechanisms and brain circuits underlying fragile X syndrome" (IRB # 2015-8425) or appropriate baseline measures through Biorepository (2013-7327).
- •FXS is defined as full FMR1 mutations (>200 CGG repeats) confirmed by genetic testing.
- •General good health as determined by physical exam, medical history and laboratory work up.
- •Stanford Binet IQ <85
- •Stable dosing of psychotropic drugs for at least 4 weeks.
排除标准
- •Subjects with a history of intolerance to baclofen, roflumilast, or memantine will be excluded.
- •Subjects will also be excluded if they have taken any investigational drug within 3 months, have a history of substance abuse or dependence within 6 months, or significant psychiatric or CNS neurological disease unrelated to FXS.
- •Uncontrolled seizures or history of epilepsy with a seizure in the past 6 months
- •Auditory or visual impairments that cannot be corrected based on visual and auditory screener benchmarks.
- •Moderate to severe renal or hepatic impairment and determined by a study physician incorporating data from exam, medical history and laboratory value evaluation among other data points.
- •Use of barbiturates, benzodiazepines, antiepileptics, or other GABAergic or glutamatergic modulators
- •Current use of: Amifampridine, Butalbital, Codeine, Doxylamine, Ethanol, Hydrocodone, Isocarboxazid, Kava, Metoclopramide, Midazolam, Oxybate, Phenelzine, Promethazine, Thalidomide, Tranylcypromine, Trimethobenzamide, Erythromycin, Ketoconazole, Fluvoxamine, Enoxacin, and Cimetidine.
- •Those taking other psychiatric medications must be on stable doses for 4 weeks before the baseline visit.
- •Pregnancy or breast-feeding. For female subjects of child bearing potential, a urine pregnancy test will be performed.
- •Potential subjects with a creatinine clearance < 50 mL/min will be excluded.
- •Identified medical issues, inability to tolerate study procedures or study drug per the discretion of the Principal Investigator.
研究组 & 干预措施
Experimental Study Participants
Participants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses.
干预措施: Baclofen (Drug)
Experimental Study Participants
Participants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses.
干预措施: Memantine (Drug)
Experimental Study Participants
Participants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses.
干预措施: Roflumilast (Drug)
Control Study Participants
Participants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses.
干预措施: Placebo (Drug)
结局指标
主要结局
Change in EEG Relative Gamma Power
时间窗: Pre-dose, 3-hour post-dose
EEG relative gamma power at rest was calculated as the percent of power in the gamma frequencies relative to the sum of power in all frequency bands, averaged across electrodes, and calculated separately at pre-dose and post-dose timepoints. To assess the impact of drug, the pre-dose relative gamma power was subtracted from post-dose relative gamma power. Higher numbers indicate more relative gamma power post-dose; lower numbers indicate more relative gamma power pre-dose.
次要结局
- Clinical Global Impressions-Improvement(Pre-dose, 3-hour post-dose)
- Clinical Global Impressions-Improvement-Caregiver(Pre-dose, 3-hour post-dose)
- Visual Analog Scale - Caregiver(Pre-dose, 3-hour post-dose)