Efficacy and Safety of Metformin in the Treatment of Fragile X Syndrome
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 37
- 试验地点
- 2
- 主要终点
- Change of scores of Aberrant Behavior Checklist (ABC)
研究概览
简要总结
This study is a controlled trial of metformin in children with fragile X syndrome(FXS). The age of FXS children range from 2 to 16 years old. Participants will be randomized in a double-blind design to either drug or placebo for 6-month period. The primary objectives are to assess metformin in treatment of behavior problems, cognitive and language with fragile X syndrome.
详细描述
This is a single-center study at the Children's Hospital of Fudan University for FXS patients aged 2 to 16 years inclusive. It is a randomized, double-blind, placebo-controlled trial of metformin.
Studies showed that there were pathogenically over activates of mTOR and MAPK/ERK pathways in FXS. Metformin, a guanidine derivative ,has been shown to reduce mTORC1 pathway activity in an AMPK-dependent manner and has also been shown to reduce MAPK pathway activity. Metformin treatments for FXS have been reported in animal experiments, some open label trials of metformin in FXS patients had been reported. Therefore, metformin has potential to rescue symptoms in children with FXS.
In this study, researchers hope to investigate the improvement effect of metformin on FXS symptoms such as behavior problems, cognition, language.
The intervention period is 6 months, follow-up visit at 1 year. The researchers will also assess the side effects of the study medication throughout the trial.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 2 Years 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetic testing confirms the diagnosis of FXS
- •Participate in the study with the informed consent of the guardian
- •BMI>the 3rd percentile
- •Not taking more than 2 therapeutic drugs
- •Able to receive regular follow-up visits
排除标准
- •Malnutrition
- •Primary heart disease
- •Severe infection or acute clinical illness
- •Gastrointestinal, renal, or hepatic disease
- •Previous history of lactic acidosis
- •previous use of metformin intolerant
- •Use of angiotensin converting enzyme inhibitors, use of anticoagulants, vitamin B12 deficiency, alcohol consumption
- •Unstable systemic diseases other than FXS
- •Changes in clinical medication
研究组 & 干预措施
Metformin group
The patients will be obtain Metformin starting from 50mg everyday to 1-2g per day for 6 months.
干预措施: Metformin (Drug)
Placebo group
The patients will be obtain starch tablets starting from 50mg everyday to 1-2g per day for 6 months.
干预措施: Placebo (Drug)
结局指标
主要结局
Change of scores of Aberrant Behavior Checklist (ABC)
时间窗: From baseline to the 6th month
Difference on the scores of the ABC from baseline to the 6th month. ABC is a 58-item behavior scale ranked from 0("not a problem") to 3("severe problem") being the most severe for each item. 5 subscales include irritability, lethargy, stereotypy , hyperactivity and inappropriate speech. The same parent of patient will complete the checklist from baseline to 6th month. The global score changes of ABC will be measured as the primary outcome.
次要结局
未报告次要终点