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

Phase 2

Active, not recruiting

• Conditions: Gastrointestinal Disorder; Autism Spectrum Disorder
• Interventions: Drug: Vancomycin; Drug: MoviPrep; Biological: Full Spectrum Microbiota

• Registration Number: NCT03408886
• Lead Sponsor: Arizona State University

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.

Study Timeline

• Study Start: 2018-01-04
• Study First Submitted: 2018-01-08
• Study First Submitted QC: 2018-01-23
• Study First Posted: 2018-01-24
• Primary Completion: 2022-04-15
• Status Verified: 2023-01
• Last Update Submitted: 2023-01-06
• Last Update Posted: 2023-01-09
• Study Completion: 2024-12-15

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 55

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Group B	Full Spectrum Microbiota	Group B receives no treatment in Part 1, but does receive treatment in Part 2
Group A	Full Spectrum Microbiota	Group A receives treatment in Part 1 and Part 2
Group A	Vancomycin	Group A receives treatment in Part 1 and Part 2
Group B	MoviPrep	Group B receives no treatment in Part 1, but does receive treatment in Part 2
Group A	MoviPrep	Group A receives treatment in Part 1 and Part 2

Primary Outcome Measures

Name	Time	Method
Change in Childhood Autism Rating Scale (CARS) from baseline to 10 weeks	baseline; month 2.5, 4.5, 10.5, 16.5, 22.5	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

Name	Time	Method
Change in Daily Stool Log (DSL) from baseline to 10 weeks	baseline for 2 weeks; daily for 18 weeks, and 2 weeks at month 10, 16, 22	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	baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; months 10.5, 16.5, 22.5	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	baseline; month 2.5, 4.5, 10.5, 16.5, 22.5	a questionnaire about aberrant behaviors. Scores range from 0 to 174, with higher scores indicating more severe behaviors

Trial Locations

Locations (1)

Arizona State University; 🇺🇸 Tempe, Arizona, United States