MTT for Children With ASD Who Have Gastrointestinal Disorders
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|ClinicalTrials.gov Identifier: NCT04182633|
Recruitment Status : Recruiting
First Posted : December 2, 2019
Last Update Posted : December 30, 2021
|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|
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 Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||70 participants|
|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.|
|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 :||February 28, 2023|
|Estimated Study Completion Date :||December 30, 2024|
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)
- 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.
- 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.
- 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.
- 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
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
|Contact: James B Adams, PhD||480 965 firstname.lastname@example.org|
|United States, Arizona|
|Arizona State University||Recruiting|
|Tempe, Arizona, United States, 85287|
|Contact: James B Adams, PhD 480-965-3316 email@example.com|
|Principal Investigator:||James B Adams, PhD||Arizona State University|