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Microbiota Transfer Therapy for Adults With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders (MTT-ASD)

Primary Purpose

Autism Spectrum Disorder, Gastrointestinal Disorder

Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Vancomycin
MoviPrep
Full Spectrum 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, microbiota transplant, vancomycin, moviprep

Eligibility Criteria

18 Years - 60 Years (Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Adult aged 18-60 years
  2. Diagnosis of autism per both the Autism Diagnostic Interview - Revised (ADI-R) and the Childhood Autism Rating Scale 2 (CARS-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. Neurotypical adult observer (such as parent, guardian, or sibling) who observes adult for at least 4 hours/week who can serve as an Evaluator to complete questionnaires on their symptoms with the assistance of the Participant as much as they are able.
  7. Ability to swallow pills (without chewing)

Exclusion Criteria:

  1. Antibiotics in last 3 months
  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
  9. Recent or scheduled surgeries
  10. Current participation in other clinical trials
  11. Females who are pregnant or who are sexually active without effective birth control. We will conduct a urine pregnancy test on all female participants as part of the screening and at each clinical visit.
  12. Allergy or intolerance to vancomycin or MoviPrep

  13. Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel and Complete Blood Count with Differential.

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Sites / Locations

  • Arizona State University

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Other

Arm Label

Group A

Group B

Arm Description

Group A receives treatment in Part 1 and Part 2

Group B receives no treatment in Part 1, but does receive treatment in Part 2

Outcomes

Primary Outcome Measures

Change in Childhood Autism Rating Scale (CARS) from baseline to 10 weeks
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.

Secondary Outcome Measures

Change in Daily Stool Log (DSL) from baseline to 10 weeks
The DSL is a report of the number and type of stools over 14 days. Each stool is rated on a scale of 1-7, where 1=very hard, 4=normal, 7=very soft/liquid. The DSL is scored by the number of days of an abnormal stool (type 1-2 or 6-7) or no stool.
Change in Social Responsiveness Scale - 2 from baseline to 10 weeks 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.
Change in Aberrant Behavior Checklist from baseline to 10 weeks
a questionnaire about aberrant behaviors. Scores range from 0 to 174, with higher scores indicating more severe behaviors

Full Information

First Posted
January 8, 2018
Last Updated
January 6, 2023
Sponsor
Arizona State University
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1. Study Identification

Unique Protocol Identification Number
NCT03408886
Brief Title
Microbiota Transfer Therapy for Adults With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
Acronym
MTT-ASD
Official Title
Microbiota Transfer Therapy for Adults 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
January 4, 2018 (Actual)
Primary Completion Date
April 15, 2022 (Actual)
Study Completion Date
December 15, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Arizona State University

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
This is a clinical trial of Microbiota Transplant Therapy (MTT) for adults with autism spectrum disorders (ASD) who have gastrointestinal problems. Previous research has shown that individuals with ASD have a low diversity of gut bacteria, and low diversity is generally associated with poor gastrointestinal (GI) health. We previously found that MTT therapy for children with ASD and GI symptoms was helpful in reducing their GI symptoms, reducing their ASD symptoms, and increasing their diversity of gut bacteria. This clinical trial will investigate the hypothesis that MTT therapy will be helpful for adults with ASD who have GI symptoms.
Detailed Description
For adults ages 18-60 years with ASD and gastrointestinal problems, the investigators propose a Phase 2 clinical trial to evaluate the safety and efficacy of MTT. The study will also determine if longer treatment is beneficial, and to conduct a longer observation after treatment stops to determine long-term safety and efficacy. The three parts of this trial are described below. Part 1: Placebo-Controlled Treatment The trial will begin with a randomized, double-blind, placebo-controlled trial which will include a 2-week treatment with oral vancomycin (or placebo), then 1 day of Moviprep to cleanse the bowel of vancomycin and bacteria/feces (all participants, since its bowel-emptying effect cannot be blinded), followed by oral administration of Full Spectrum Microbiota (FSM) or placebo. An initial high dose of FSM (or placebo) for two days will be followed by a lower maintenance dose of FSM (or placebo) for 8 weeks. Part 2 Extension and Cross-Over For the treatment group from Part 1, there will be an 8-week extension of the maintenance dose, to determine if longer treatment has additional benefits. For the placebo group from Part 1, they will receive MoviPrep, an initial high dose of FSM for 2 days, and then a lower dose of FSM for 8 weeks (similar to the treatment group in Part 1, but without the vancomycin). This will help us determine if pre-treatment with vancomycin is needed or not. Part 3: Follow-up There will be follow-up evaluations at 6, 12, and 18 months after treatment is stopped, to assess long-term efficacy and possible adverse effects.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism Spectrum Disorder, Gastrointestinal Disorder
Keywords
fecal transplant, fecal microbiota transplant, microbiota transplant, vancomycin, moviprep

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
There are two groups. In Part 1, group A receives treatment and group B receives placebo. In Part 2, group A continues to receive treatment, and group B is switched to treatment.
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
In Part 1, everyone is blinded except for the research pharmacist. In Part 2, the participants and study coordinator are unblinded, but the professional evaluators are blinded.
Allocation
Randomized
Enrollment
55 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Group A
Arm Type
Experimental
Arm Description
Group A receives treatment in Part 1 and Part 2
Arm Title
Group B
Arm Type
Other
Arm Description
Group B receives no treatment in Part 1, but does receive treatment in Part 2
Intervention Type
Drug
Intervention Name(s)
Vancomycin
Intervention Description
Oral vancomycin is administered to reduce pathogenic bacteria.
Intervention Type
Drug
Intervention Name(s)
MoviPrep
Intervention Description
MoviPrep is given at the end of vancomycin therapy to remove the vancomycin and remaining bacteria prior to administering Full-Spectrum Microbiota
Intervention Type
Biological
Intervention Name(s)
Full Spectrum Microbiota
Other Intervention Name(s)
fecal microbiota transplant
Intervention Description
Gut bacteria from healthy human donors are administered orally in a pill form
Primary Outcome Measure Information:
Title
Change in Childhood Autism Rating Scale (CARS) from baseline to 10 weeks
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
baseline; month 2.5, 4.5, 10.5, 16.5, 22.5
Secondary Outcome Measure Information:
Title
Change in Daily Stool Log (DSL) from baseline to 10 weeks
Description
The DSL is a report of the number and type of stools over 14 days. Each stool is rated on a scale of 1-7, where 1=very hard, 4=normal, 7=very soft/liquid. The DSL is scored by the number of days of an abnormal stool (type 1-2 or 6-7) or no stool.
Time Frame
baseline for 2 weeks; daily for 18 weeks, and 2 weeks at month 10, 16, 22
Title
Change in Social Responsiveness Scale - 2 from baseline to 10 weeks of treatment
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
baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; months 10.5, 16.5, 22.5
Title
Change in Aberrant Behavior Checklist from baseline to 10 weeks
Description
a questionnaire about aberrant behaviors. Scores range from 0 to 174, with higher scores indicating more severe behaviors
Time Frame
baseline; month 2.5, 4.5, 10.5, 16.5, 22.5
Other Pre-specified Outcome Measures:
Title
Change in Gastrointestinal Stool and Symptom Questionnaire for Autism, from baseline to 10 weeks
Description
a questionnaire about GI-related symptoms.
Time Frame
baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; month 10.5, 16.5, 22.5
Title
Change in Microbiome composition from baseline to 10 weeks
Description
analysis of the bacterial composition of stool samples, at the species, genus, and phylum level
Time Frame
baseline; month 1, 2.5, 3.5, 4.5, 10.5, 16.5, 22.5
Title
Change in the Vineland Adaptive Behavior Scale - II, from baseline to 10 weeks
Description
questionnaire about adaptive behaviors; it yields the developmental age of the participant
Time Frame
baseline; month 2.5, 4.5, 22.5
Title
Change in Ohio State University Clinical Impressions Scale, from baseline 10 weeks
Description
assessment of autism symptoms by a clinician. It rates 10 symptoms on a scale of 1-7, with higher scores indicating worse symptoms.
Time Frame
baseline; month 2.5, 4.5
Title
Change in Parent Global Impressions, from baseline to 10 weeks
Description
Assessment of autism symptoms. It rates 20 symptoms on a scale of 1-7, with higher scores indicating worse symptoms
Time Frame
baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; month 10.5, 16.5, 22.5

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Adult aged 18-60 years Diagnosis of autism per both the Autism Diagnostic Interview - Revised (ADI-R) and the Childhood Autism Rating Scale 2 (CARS-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 Neurotypical adult observer (such as parent, guardian, or sibling) who observes adult for at least 4 hours/week who can serve as an Evaluator to complete questionnaires on their symptoms with the assistance of the Participant as much as they are able. Ability to swallow pills (without chewing) Exclusion Criteria: Antibiotics in last 3 months 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 Recent or scheduled surgeries Current participation in other clinical trials Females who are pregnant or who are sexually active without effective birth control. We will conduct a urine pregnancy test on all female participants as part of the screening and at each clinical visit. Allergy or intolerance to vancomycin or MoviPrep Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel and Complete Blood Count with Differential. -
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
James B Adams, PhD
Organizational Affiliation
Arizona State University
Official's Role
Study Director
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
No
Citations:
PubMed Identifier
28122648
Citation
Kang DW, Adams JB, Gregory AC, Borody T, Chittick L, Fasano A, Khoruts A, Geis E, Maldonado J, McDonough-Means S, Pollard EL, Roux S, Sadowsky MJ, Lipson KS, Sullivan MB, Caporaso JG, Krajmalnik-Brown R. Microbiota Transfer Therapy alters gut ecosystem and improves gastrointestinal and autism symptoms: an open-label study. Microbiome. 2017 Jan 23;5(1):10. doi: 10.1186/s40168-016-0225-7.
Results Reference
background
Links:
URL
http://autism.asu.edu
Description
link to more information about the research group

Learn more about this trial

Microbiota Transfer Therapy for Adults With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders

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