A Proof of Concept Study : Open Label Phase IIa Trial Evaluating the Effect and Safety of Transplantation of Fecal Microbiota in Children With Autism Spectrum Disorders and Gastrointestinal Symptoms
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Score of the Gastrointestinal Symptoms Rating Scale (GSRS)
研究概览
简要总结
Autism Spectrum Disorders (ASD), affect approximately 1% of the general population and are characterized by impairments in social communication associated with repetitive/stereotyped behaviors. Approximately one-third of these patients display gastrointestinal (GI) symptoms, and a growing number of studies suggest abnormalities of gut microbiota in ASD. Gut microbiota and the brain interact through complex pathways. Preliminary evidence in adults with ASD suggests that modulation of the gut microbiota by probiotics and/or prebiotics and more recently by fecal microbiota transplantation could improve GI symptoms but also socio-communication deficit, with persistent improvement at year 2.
详细描述
- Screening :
Screening will be done in ASD units of child psychiatry departments of Robert Debré 2. Information of patients and their families Information will be delivered in the child psychiatry department of Robert Debré hospital. 3. Inclusion Consent will be collected in CIC of Robert Debré hospital during the initial visit by child psychiatrist 7 to 15 days after information from the patient's family. 4. Patient follow-up during trial Initial visit: for parents and patient information. Further visits (W4, W18, W24, M12): treatment administrations will be done at the CIC. Subjects will be evaluated on efficacy and safety and acceptability. Stool, urine and blood samples will be collected.
Phone visits (W11 and M9): Safety and the Acceptability questionnaire (parents) will be evaluated. 5. Products: donor and transplant preparation 6. Same administration schedule in FMT
- D1 to D4: Vancomycin (10mg/kg, 4 times a day). 1st dose in the CIC, the 3 others at home
- D4: Picoprep® treatment at home
- D5: First FMT using rectal enema (100ml = 25g of stool from donor) using Foley probe (rectal administration) in CIC
- D12: Second FMT using rectal enema (100ml = 25g of stool from donor) using Foley probe (rectal administration) in CIC
- D19: Third FMT using rectal enema (100ml = 25g of stool from donor) using Foley probe (rectal administration) in CIC
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 36 Months 至 72 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •ASD diagnosis based on DSM-5 criteria
- •Gastrointestinal symptoms: at least a score of 5 on 1 of the 5 GSRS domains (diarrhea, abdominal pain, constipation, indigestion, reflux) for over a period of 1 year or more
- •Age between 36 and 72 months
- •Consent from all legal guardians
- •Social insurance affiliation
排除标准
- •Severe intellectual deficit (IQ<30) or another uncontrolled comorbidity (e.g. epilepsy)
- •Recent change in ASD global care (cognitive behavioral therapy 6 weeks before inclusion)
- •Actual or past psychotropic medication
- •Contraindications to colon preparation (Picoprep®) and/or to Meopa)
- •Severe or uncontrolled gastrointestinal symptoms (e.g. urgent treatment needed)
- •Undernutrition (age related BMI < 3rd percentile)
- •Inflammatory bowel disease
- •Exclusive diet (gluten- or sugar-free)
- •Rectal enema contraindication
- •Antibiotic or antifungal therapy 3 months before inclusion
- •Probiotics 2 months before inclusion or FMT 12 months before inclusion
- •Vancomycin or Picoprep® or Meopa (including excipients) known allergy or intolerance Experimental therapeutic trial participation 3 months before inclusion
研究组 & 干预措施
Felictia Arm
FMT
干预措施: FMT Protocol (Drug)
结局指标
主要结局
Score of the Gastrointestinal Symptoms Rating Scale (GSRS)
时间窗: Week 18
Assess initial efficacy on gastrointestinal (GI) symptoms of fecal microbiota transplantation (FMT) in children (36 to 72 months) with autism spectrum disorders (ASD (FMT) The Gastrointestinal Symptoms Rating Scale (GSRS) uses 15 items rated on a 7-point scale to assess gastrointestinal symptom severity, with total scores ranging from 15 (no symptoms) to 105 (very severe symptoms). Higher GSRS scores indicate more severe symptoms and a worse gastrointestinal outcome.
次要结局
- Score of Vineland Adaptative Behavior Composite 3 scale(Day 0, Week 4, Week 18, Week 24, Month 12)
- Tolerability: Score of bristol Stool form(Day 0, Week 4, Week 18, Week 24, Month12)
- Score of Pediatric Adverse Event Rating Scale(Day 0, Day 5, Week 4, Week 18, Week 24, Month 12)
- Score of Pediatric Quality of Life Inventory(Day 0, Week 18, Month 12)