Clinical trial NCT03408886: Microbiota Transfer Therapy for Adults With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
Official title: Microbiota Transfer Therapy for Adults With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Gastrointestinal Disorder
Keywords: fecal transplant
fecal microbiota transplant
microbiota transplant
vancomycin
moviprep
Lead sponsor: Arizona State University
Sponsor class: OTHER
First posted: 2018-01-24
Enrollment: 55
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention 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.
Primary purpose: TREATMENT

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.

Eligibility criteria: 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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Primary outcomes:
measure: Change in Childhood Autism Rating Scale (CARS) from baseline to 10 weeks
timeFrame: baseline; month 2.5, 4.5, 10.5, 16.5, 22.5
Secondary outcomes:
measure: Change in Daily Stool Log (DSL) from baseline to 10 weeks
timeFrame: baseline for 2 weeks; daily for 18 weeks, and 2 weeks at month 10, 16, 22
measure: Change in Social Responsiveness Scale - 2 from baseline to 10 weeks of treatment
timeFrame: baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; months 10.5, 16.5, 22.5
measure: Change in Aberrant Behavior Checklist from baseline to 10 weeks
timeFrame: baseline; month 2.5, 4.5, 10.5, 16.5, 22.5
Other outcomes:
measure: Change in Gastrointestinal Stool and Symptom Questionnaire for Autism, from baseline to 10 weeks
timeFrame: baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; month 10.5, 16.5, 22.5
measure: Change in Microbiome composition from baseline to 10 weeks
timeFrame: baseline; month 1, 2.5, 3.5, 4.5, 10.5, 16.5, 22.5
measure: Change in the Vineland Adaptive Behavior Scale - II, from baseline to 10 weeks
timeFrame: baseline; month 2.5, 4.5, 22.5
measure: Change in Ohio State University Clinical Impressions Scale, from baseline 10 weeks
timeFrame: baseline; month 2.5, 4.5
measure: Change in Parent Global Impressions, from baseline to 10 weeks
timeFrame: baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; month 10.5, 16.5, 22.5
Locations:
city: Tempe
country: United States
facility: Arizona State University
state: Arizona
Study officials:
affiliation: Arizona State University
name: James B Adams, PhD
role: STUDY_DIRECTOR
References:
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.