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MTT for Children With ASD Who Have Gastrointestinal Disorders

Primary Purpose

Autism Spectrum Disorder, Gastro-Intestinal Disorder, Constipation

Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
vancomycin, Miralax, intestinal microbiota
placebo vancomycin, Miralax, placebo intestinal microbiota
Sponsored by
Arizona State University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autism Spectrum Disorder focused on measuring fecal transplant, fecal microbiota transplant, intestinal microbiota transplant, microbiota transplant, autism, autism spectrum disorder

Eligibility Criteria

5 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria for Children with ASD

  1. Child aged 5-17 years
  2. 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).
  3. GI disorder as defined below that has lasted for at least 3 years.
  4. No changes in medications, supplements, diet, therapies, or education in last 3 months, and no intention to change them during the clinical trial.
  5. General good physical health aside from gastrointestinal problems
  6. Ability to swallow pills (without chewing)

Exclusion Criteria for Children with ASD

  1. Antibiotics in last 3 months (does not apply to topical antibiotics)
  2. Probiotics in last 2 months, or fecal transplant in last 12 months
  3. Single-gene disorder (Fragile X, etc.)
  4. Major brain malformation
  5. Tube feeding
  6. Severe gastrointestinal problems that require immediate treatment (life-threatening)
  7. Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions
  8. Severely underweight/malnourished (per physician clinical judgement)
  9. 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)
  10. Recent or scheduled surgeries
  11. Current participation in other clinical trials
  12. Females who are pregnant or who are sexually active without effective birth control.
  13. Allergy or intolerance to vancomycin or Miralax
  14. Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel and Complete Blood Count with Differential.
  15. Psychotropic medication daily use - Current or within past 2 months - which are known to interfere in gastrointestinal function
  16. 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.

Sites / Locations

  • Arizona State University

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Group A - Treatment Group

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

Arm Description

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

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

Outcomes

Primary Outcome Measures

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.
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.

Secondary Outcome Measures

Daily Stool Record
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
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
A questionnaire about aberrant behaviors. Scores range from 0 to 174, with higher scores indicating more severe behaviors

Full Information

First Posted
November 27, 2019
Last Updated
January 6, 2023
Sponsor
Arizona State University
Collaborators
University of Minnesota, Finch Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT04182633
Brief Title
MTT for Children With ASD Who Have Gastrointestinal Disorders
Official Title
Microbiota Transfer Therapy for Children With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
November 15, 2019 (Actual)
Primary Completion Date
August 1, 2023 (Anticipated)
Study Completion Date
December 30, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Arizona State University
Collaborators
University of Minnesota, Finch Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism Spectrum Disorder, Gastro-Intestinal Disorder, Constipation, Diarrhea
Keywords
fecal transplant, fecal microbiota transplant, intestinal microbiota transplant, microbiota transplant, autism, autism spectrum disorder

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
This is a randomized, double-blind, placebo-controlled study.
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
Randomized, double-blind, placebo-controlled study.
Allocation
Randomized
Enrollment
50 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Group A - Treatment Group
Arm Type
Experimental
Arm Description
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
Arm Title
Group B - Control Group (Miralax only for 1 day)
Arm Type
Placebo Comparator
Arm Description
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
Intervention Type
Combination Product
Intervention Name(s)
vancomycin, Miralax, intestinal microbiota
Intervention Description
vancomycin for 14 days, then Miralax for 1 day, then intestinal microbiota (high dose for 2 days, then maintenance dose for 12 weeks)
Intervention Type
Combination Product
Intervention Name(s)
placebo vancomycin, Miralax, placebo intestinal microbiota
Intervention Description
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)
Primary Outcome Measure Information:
Title
Childhood Autism Rating Scale (CARS)
Description
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.
Time Frame
change in score from baseline to 14 weeks (end of treatment)
Title
Gastrointestinal Symptom Severity Scale (GSRS)
Description
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.
Time Frame
change in score from baseline to 14 weeks (end of treatment)
Secondary Outcome Measure Information:
Title
Daily Stool Record
Description
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.
Time Frame
change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)]
Title
Social Responsiveness Scale 2
Description
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.
Time Frame
change in score from baseline to 14 weeks (end of treatment)
Title
Aberrant Behavior Checklist
Description
A questionnaire about aberrant behaviors. Scores range from 0 to 174, with higher scores indicating more severe behaviors
Time Frame
change in score from baseline to 14 weeks (end of treatment)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria for Children with ASD 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) Exclusion Criteria for Children with ASD 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 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
James B Adams, PhD
Organizational Affiliation
Arizona State University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Arizona State University
City
Tempe
State/Province
Arizona
ZIP/Postal Code
85287
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Undecided
Links:
URL
http://autism.asu.edu
Description
The web page for the research group leading this study

Learn more about this trial

MTT for Children With ASD Who Have Gastrointestinal Disorders

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