Microbiota Transfer Therapy for Children With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Childhood Autism Rating Scale (CARS)
研究概览
简要总结
The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating children with Autism Spectrum Disorder (ASD) and gastrointestinal problems (primarily constipation and/or diarrhea). MTT involves a combination of 10 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by a bowel cleanse, followed by 12 weeks of Fecal Microbiota (FM).
详细描述
For children ages 5-17 years with Autism Spectrum Disorder and gastrointestinal problems, a Phase 2 clinical trial will evaluate the safety, tolerability, and efficacy of MTT. The three parts of this trial are described below.
Part 1: Randomized Trial The trial will begin with a randomized, double-blind, placebo-controlled trial which will include a 2-week treatment with oral vancomycin (Group A) or placebo (Group B), then 1 day of Miralax to cleanse the bowel of vancomycin and bacteria/feces (both groups), followed by oral administration of FM (Group A) or placebo (Group B). An initial high dose of FM (or placebo) for two days will be followed by a lower maintenance dose of FM or placebo for 12 weeks.
Part 2: Extension (Group A) and Treatment (Group B)
- For Group A there will be 12-week observation period
- For Group B, there will be treatment similar to that received by the treatment group in Part 1 (partial cross-over, open-label), except that they will not receive vancomycin. So, they will be treated with Miralax followed by an initial high dose of FM for 2 days, and then a maintenance dose of FM for 12 weeks. The purpose of this arm is to determine if vancomycin is necessary or not.
Part 3: Follow-up There will be a follow-up evaluation at 6, 12, and 18 months after treatment ends, to determine long-term safety and efficacy.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Randomized, double-blind, placebo-controlled study.
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
- •for Children with ASD
- •Child aged 5-17 years
- •Diagnosis of autism per the Childhood Autism Rating Scale 2 (CARS-2) and either the Autism Diagnostic Interview-Revised (ADI-R) or the Autism Diagnostic Observation Schedule 2 (ADOS 2).
- •GI disorder as defined below that has lasted for at least 3 years.
- •No changes in medications, supplements, diet, therapies, or education in last 3 months, and no intention to change them during the clinical trial.
- •General good physical health aside from gastrointestinal problems
- •Ability to swallow pills (without chewing)
排除标准
- •for Children with ASD
- •Antibiotics in last 3 months (does not apply to topical antibiotics)
- •Probiotics in last 2 months, or fecal transplant in last 12 months
- •Single-gene disorder (Fragile X, etc.)
- •Major brain malformation
- •Tube feeding
- •Severe gastrointestinal problems that require immediate treatment (life-threatening)
- •Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions
- •Severely underweight/malnourished (per physician clinical judgement)
- •Unstable, poor health; seizure disorder that is not responsive to treatment or not on stable management or complex type; or other health conditions that would significantly increase their risk of adverse effects (per physician clinical judgement)
- •Recent or scheduled surgeries
- •Current participation in other clinical trials
- •Females who are pregnant or who are sexually active without effective birth control.
- •Allergy or intolerance to vancomycin or Miralax
- •Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel and Complete Blood Count with Differential.
- •Psychotropic medication daily use - Current or within past 2 months - which are known to interfere in gastrointestinal function
- •Evidence of significant impairment of immune system, or taking medications that can compromise the immune system, and thus increase risk if exposed to multiple-drug resistant bacteria.
研究组 & 干预措施
Group A - Treatment Group
This group will receive vancomycin for 14 days, then Miralax for 1 day, then intestinal microbiota for 2 days at high dose, then intestinal microbiota for 12 weeks at a maintenance dose
干预措施: vancomycin, Miralax, intestinal microbiota (Combination Product)
Group B - Control Group (Miralax only for 1 day)
This group will receive placebo vancomycin for 14 days, then Miralax for 1 day, then placebo intestinal microbiota for 2 days at high dose, then placebo intestinal microbiota for 12 weeks at a maintenance dose
干预措施: placebo vancomycin, Miralax, placebo intestinal microbiota (Combination Product)
结局指标
主要结局
Childhood Autism Rating Scale (CARS)
时间窗: change in score from baseline to 14 weeks (end of treatment)
An evaluation by a trained evaluator of autism symptoms. The range is 15-60, and scores in the range of 27-30 or higher are indicative of autism.
Gastrointestinal Symptom Severity Scale (GSRS)
时间窗: change in score from baseline to 14 weeks (end of treatment)
A 15-item questionnaire for evaluation of GI symptoms, with each item rated on a scale of 1 (no symptoms) to 7 (severe symptoms), with an average score of 1-7.
次要结局
- Daily Stool Record(change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)])
- Social Responsiveness Scale 2(change in score from baseline to 14 weeks (end of treatment))
- Aberrant Behavior Checklist(change in score from baseline to 14 weeks (end of treatment))