Microbiota Transfer Therapy for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Daily Stool Record (DSR(
研究概览
简要总结
The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating patients with Pitt Hopkins Syndrome (PTHS) and gastrointestinal problems similar to Irritable Bowel Syndrome (IBS). 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 PTHS and gastrointestinal problems, a Phase 2 clinical trial will evaluate the safety, tolerability, and efficacy of MTT.
Randomized, double-blind, placebo-Controlled Treatment (14 weeks)
The trial will be a randomized, double-blind, placebo-controlled trial which will include a 10-day treatment with oral vancomycin (or placebo), then 1 day of magnesium citrate to cleanse the bowel of vancomycin and bacteria/feces (all participants, since its bowel-emptying effect cannot be blinded), followed by oral administration of FM (or placebo). 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.
Group A: real treatment Group B: placebo vancomycin, real magnesium citrate, placebo FM
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
Part 1 is double-blind Parts 2 and 3 are single-blind (outcomes assessor is blinded)
入排标准
- 年龄范围
- 7 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children ages 7-17 years with Pitt Hopkins Syndrome (verified by genetic testing)
- •GI disorder as defined below that has lasted for at least 2 years.
- •No changes in medications, supplements, diet, or therapies in last 2 months, and no intention to change them during the Parts 1 and 2 of the clinical trial.
- •Ability to swallow pills (without chewing)
- •Review of last two years of medical records by the study physician.
排除标准
- •Antibiotics in last 3 months
- •Probiotics in last 2 months, or fecal transplant in last 12 months
- •Tube feeding
- •Severe gastrointestinal problems that require immediate treatment (life-threatening)
- •Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions
- •Unstable, poor health (based on study physician's opinion)
- •Recent or scheduled surgeries
- •Current participation in other clinical trials
- •Females who are pregnant or who are at risk of pregnancy and sexually active without effective birth control.
- •Allergy or intolerance to vancomycin or magnesium citrate
- •Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel, and Complete Blood Count with Differential.
- •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.
结局指标
主要结局
Daily Stool Record (DSR(
时间窗: change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)
The DSR is a daily record of their bowel movements including Bristol Stool Form scale. It is rated as the number of days (out of 14 days) with an abnormal report (abnormal stool, no stool, or the use of a gastrointestinal treatment). A higher percentage indicates worse symptoms.
Safety Measures
时间窗: weeks 0-14
number of adverse events and serious adverse events likely associated with treatment
次要结局
- CGI for GI Disorders(change in score between baseline and week 14)
- CGI for PTHS Symptoms(change in score between baseline and week 14)
- GSRS(change in score between baseline and week 14)
- PGI-PTHS(change in score between baseline and week 14)
- FLACC(change in score between baseline and week 14)