Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

MTT for Children With ASD Who Have Gastrointestinal Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04182633
Recruitment Status : Recruiting
First Posted : December 2, 2019
Last Update Posted : December 3, 2019
Sponsor:
Collaborators:
Arizona State University Foundation
University of Minnesota
Finch Therapeutics
Information provided by (Responsible Party):
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).

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Gastro-Intestinal Disorder Constipation Diarrhea Combination Product: vancomycin, Miralax, intestinal microbiota Combination Product: placebo vancomycin, Miralax, placebo intestinal microbiota Phase 2

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: This is a randomized, double-blind, placebo-controlled study.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Randomized, double-blind, placebo-controlled study.
Primary Purpose: Treatment
Official Title: Microbiota Transfer Therapy for Children With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
Actual Study Start Date : November 15, 2019
Estimated Primary Completion Date : June 30, 2021
Estimated Study Completion Date : June 30, 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Group A - Treatment Group
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
vancomycin for 14 days, then Miralax for 1 day, then intestinal microbiota (high dose for 2 days, then maintenance dose for 12 weeks)

Placebo Comparator: Group B - Control Group (Miralax only for 1 day)
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
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 Measures :
  1. 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.

  2. 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 Outcome Measures :
  1. Daily Stool Record [ Time Frame: 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.

  2. Social Responsiveness Scale 2 [ Time Frame: 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.

  3. Aberrant Behavior Checklist [ Time Frame: 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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04182633


Contacts
Layout table for location contacts
Contact: James B Adams, PhD 480 965 3316 jim.adams@asu.edu

Locations
Layout table for location information
United States, Arizona
Arizona State University Recruiting
Tempe, Arizona, United States, 85287
Contact: James B Adams, PhD    480-965-3316    jim.adams@asu.edu   
Sponsors and Collaborators
Arizona State University
Arizona State University Foundation
University of Minnesota
Finch Therapeutics
Investigators
Layout table for investigator information
Principal Investigator: James B Adams, PhD Arizona State University
Additional Information:
Layout table for additonal information
Responsible Party: Arizona State University
ClinicalTrials.gov Identifier: NCT04182633    
Other Study ID Numbers: NEIRB#: 120190306
First Posted: December 2, 2019    Key Record Dates
Last Update Posted: December 3, 2019
Last Verified: December 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Arizona State University:
fecal transplant
fecal microbiota transplant
intestinal microbiota transplant
microbiota transplant
autism
autism spectrum disorder
Additional relevant MeSH terms:
Layout table for MeSH terms
Digestive System Diseases
Gastrointestinal Diseases
Intestinal Diseases
Disease
Constipation
Diarrhea
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
Neurodevelopmental Disorders
Mental Disorders
Signs and Symptoms, Digestive
Vancomycin
Polyethylene glycol 3350
Anti-Bacterial Agents
Anti-Infective Agents
Laxatives
Gastrointestinal Agents