Microbiota Transfer Therapy for Children With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Autism Spectrum Disorder
- Sponsor
- Arizona State University
- Enrollment
- 50
- Locations
- 1
- Primary Endpoint
- Childhood Autism Rating Scale (CARS)
- Status
- Active, Not Recruiting
- Last Updated
- last year
Overview
Brief Summary
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).
Detailed Description
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. Participant duration will be approximately 6.5 months for treatment, and follow-up at 6, 12 and 18 months after treatment ends. Study duration will be approximately 4 years, including IRB approvals, training staff, recruiting, clinical trial, data analysis, and reporting.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
Childhood Autism Rating Scale (CARS)
Time Frame: 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)
Time Frame: 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.
Secondary Outcomes
- 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))