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

NCT03408886 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: MTT-Adults-1Wi1XWH-16-1-0492
• Study Type: interventional
• Target: 55
• Locations: 1 country
• Sites: 1

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.

Conditions

Autism Spectrum Disorder; Gastrointestinal Disorder

Keywords

fecal transplant; fecal microbiota transplant; microbiota transplant; vancomycin; moviprep

Outcome Measures

Primary Outcomes (1)

• 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.

  baseline; month 2.5, 4.5, 10.5, 16.5, 22.5

Secondary Outcomes (3)

• 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

• 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

• Change in Aberrant Behavior Checklist from baseline to 10 weeks

  baseline; month 2.5, 4.5, 10.5, 16.5, 22.5

Other Outcomes (5)

• Change in Gastrointestinal Stool and Symptom Questionnaire for Autism, from baseline to 10 weeks

  baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; month 10.5, 16.5, 22.5

• Change in Microbiome composition from baseline to 10 weeks

  baseline; month 1, 2.5, 3.5, 4.5, 10.5, 16.5, 22.5

• Change in the Vineland Adaptive Behavior Scale - II, from baseline to 10 weeks

  baseline; month 2.5, 4.5, 22.5

Study Arms (2)

Group A

EXPERIMENTAL

Group A receives treatment in Part 1 and Part 2

Drug: Vancomycin; Drug: MoviPrep; Biological: Full Spectrum Microbiota

Group B

OTHER

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

Drug: MoviPrep; Biological: Full Spectrum Microbiota

Interventions

Vancomycin DRUG

Oral vancomycin is administered to reduce pathogenic bacteria.

Group A

MoviPrep DRUG

MoviPrep is given at the end of vancomycin therapy to remove the vancomycin and remaining bacteria prior to administering Full-Spectrum Microbiota

Group A; Group B

Full Spectrum Microbiota BIOLOGICAL

Gut bacteria from healthy human donors are administered orally in a pill form

Also known as: fecal microbiota transplant

Group A; Group B

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Arizona State University: lead

Study Sites (1)

Arizona State University

Tempe, Arizona, 85287, United States

Location

Related Publications (1)

• 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.

  PMID: 28122648 BACKGROUND

Related Links

• link to more information about the research group

MeSH Terms

Conditions

Autism Spectrum Disorder; Gastrointestinal Diseases

Interventions

Vancomycin; MoviPrep; Fecal Microbiota Transplantation

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Glycopeptides; Glycoconjugates; Carbohydrates; Peptides; Amino Acids, Peptides, and Proteins; Biological Therapy; Therapeutics

Study Officials

• James B Adams, PhD

  Arizona State University

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: January 8, 2018
• First Posted: January 24, 2018
• Study Start: January 4, 2018
• Primary Completion: April 15, 2022
• Study Completion: December 15, 2024
• Last Updated: January 9, 2023

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)