MTT for Children With ASD Who Have Gastrointestinal Disorders

Phase 2

Active, not recruiting

• Conditions: Autism Spectrum Disorder; Constipation; Diarrhea; Gastro-Intestinal Disorder
• Interventions: Combination Product: vancomycin, Miralax, intestinal microbiota; Combination Product: placebo vancomycin, Miralax, placebo intestinal microbiota

• Registration Number: NCT04182633
• Lead Sponsor: Arizona State University

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.

Study Timeline

• Study Start: 2019-11-15
• Study First Submitted: 2019-11-27
• Study First Submitted QC: 2019-11-27
• Study First Posted: 2019-12-02
• Primary Completion: 2025-02-28
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-23
• Last Update Posted: 2025-04-25
• Study Completion: 2025-12-30

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Group A - Treatment Group	vancomycin, Miralax, intestinal microbiota	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
Group B - Control Group (Miralax only for 1 day)	placebo vancomycin, Miralax, placebo intestinal microbiota	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

Primary Outcome Measures

Name	Time	Method
Childhood Autism Rating Scale (CARS)	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)	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 Outcome Measures

Name	Time	Method
Daily Stool Record	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 bowel movements including Bristol Stool Form scale. It is rated as the % of days with an abnormal report (abnormal stool, no stool, or the use of a gastrointestinal treatment). A higher percentage indicates worse symptoms.
Social Responsiveness Scale 2	change in score from baseline to 14 weeks (end of treatment)	A questionnaire about social skills, where higher scores suggest more severe autism. Scores range from 0 to 195, with higher scores indicating more severe problems.
Aberrant Behavior Checklist	change in score from baseline to 14 weeks (end of treatment)	A questionnaire about aberrant behaviors. Scores range from 0 to 174, with higher scores indicating more severe behaviors

Trial Locations

Locations (1)

Arizona State University; 🇺🇸 Tempe, Arizona, United States