MTT for Children With ASD Who Have Gastrointestinal Disorders

NCT04182633 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: NEIRB#: 120190306
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

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).

Conditions

Autism Spectrum Disorder; Gastro-Intestinal Disorder; Constipation; Diarrhea

Keywords

fecal transplant; fecal microbiota transplant; intestinal microbiota transplant; microbiota transplant; autism; autism spectrum disorder

Outcome Measures

Primary Outcomes (2)

• Childhood Autism Rating Scale (CARS)

  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.

  change in score from baseline to 14 weeks (end of treatment)

• Gastrointestinal Symptom Severity Scale (GSRS)

  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.

  change in score from baseline to 14 weeks (end of treatment)

Secondary Outcomes (3)

• 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)

Study Arms (2)

Group A - Treatment Group

EXPERIMENTAL

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

Combination Product: vancomycin, Miralax, intestinal microbiota

Group B - Control Group (Miralax only for 1 day)

PLACEBO COMPARATOR

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

Combination Product: placebo vancomycin, Miralax, placebo intestinal microbiota

Interventions

vancomycin, Miralax, intestinal microbiota COMBINATION_PRODUCT

vancomycin for 14 days, then Miralax for 1 day, then intestinal microbiota (high dose for 2 days, then maintenance dose for 12 weeks)

Group A - Treatment Group

placebo vancomycin, Miralax, placebo intestinal microbiota COMBINATION_PRODUCT

placebo vancomycin for 14 days, then Miralax for 1 day, then placebo intestinal microbiota (high dose for 2 days, then maintenance dose for 12 weeks)

Group B - Control Group (Miralax only for 1 day)

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Arizona State University: lead
• University of Minnesota: collaborator

Study Sites (1)

Arizona State University

Tempe, Arizona, 85287, United States

Location

Related Links

• The web page for the research group leading this study

MeSH Terms

Conditions

Autism Spectrum Disorder; Digestive System Diseases; Constipation; Diarrhea; Autistic Disorder

Interventions

Vancomycin; polyethylene glycol 3350; Gastrointestinal Microbiome

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Glycopeptides; Glycoconjugates; Carbohydrates; Peptides; Amino Acids, Peptides, and Proteins; Microbiota; Microbiological Phenomena; Biota; Biodiversity; Ecosystem; Environment; Ecological and Environmental Phenomena; Biological Phenomena; Environment and Public Health

Study Officials

• James B Adams, PhD

  Arizona State University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Randomized, double-blind, placebo-controlled study.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This is a randomized, double-blind, placebo-controlled study.

Study Record Dates

• First Submitted: November 27, 2019
• First Posted: December 2, 2019
• Study Start: November 15, 2019
• Primary Completion: February 28, 2025
• Study Completion: December 30, 2025
• Last Updated: April 25, 2025

Record last verified: 2025-04

Locations

US (1)