Beneficial Bacteria Treatment for Autism (BBT)

Treating Gastrointestinal Problems in Children With Autism Using Beneficial Bacteria Treatment (BBT)

This is an open-label clinical trial to investigate a combination therapy for treating gastrointestinal problems in children with autism spectrum disorders.

The combination therapy includes beneficial bacteria.

Study Overview

• Status: Completed
• Conditions: Autism Spectrum Disorders; Gastrointestinal Problems
• Intervention / Treatment: Drug: oral Vancomycin; Drug: MoviPrep; Drug: Prilosec; Biological: human fecal material; processed, frozen administered orally; Biological: human fecal material; processed, frozen; administered orally and rectally

Detailed Description

This is an open-label clinical trial to evaluate the safety, tolerability, and feasibility of a combination therapy to treat gastrointestinal problems in children with autism spectrum disorders.

It involves a combination therapy including beneficial bacteria.

• Study Type: Interventional
• Enrollment (Actual): 18
• Phase: Phase 2; Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 years to 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Children ages 7-17 years
2. Diagnosis of autism per Autism Diagnostic Interview-Revised (ADI-R)
3. Moderate or Severe GI problems ( on the GSRS, a single score of 4 (severe) on any item, or a score of 3 (moderate) on two items, or a score of 2 (mild) or more on any 4 items
4. No changes in medications, supplements, diet, therapies, or education in last 3 months, and no intention to change them during clinical trial
5. General good physical health aside from gastrointestinal problems
6. Cognitive Ability to Provide Informed Assent

Exclusion Criteria:

1. Antibiotics in last 6 months
2. Probiotics in last 3 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Oral Group; This group will receive all treatments orally. The treatments include a combination of Vancomycin, MoviPrep, Prilosec, and human fecal material; processed, frozen.	Drug: oral Vancomycin; an antibiotic; Drug: MoviPrep; a bowel cleanse; Drug: Prilosec; a stomach acid suppressan; Biological: human fecal material; processed, frozen administered orally; human fecal material; processed, frozen, administered orally
EXPERIMENTAL: Rectal Group; This group will receive some treatments rectally and some orally. The treatments include a combination of Vancomycin, MoviPrep, Prilosec, and human fecal material; processed, frozen.	Drug: oral Vancomycin; an antibiotic; Drug: MoviPrep; a bowel cleanse; Drug: Prilosec; a stomach acid suppressan; Biological: human fecal material; processed, frozen; administered orally and rectally; human fecal material; processed, frozen; administered orally and rectally

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Gastrointestinal Symptom Responsiveness Scale (GSRS)	The GSRS is a measure of gastrointestinal symptoms. It includes 15 questions, rated on a Likert scale of 1-7, from no symptoms to severe symptoms, respectively (i.e., higher is worse). We report an average score for all 15 questions, so the possible range for the average score is also 1-7.	Baseline and 10 weeks (end of treatment)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Parent Global Impressions-Revised (PGI-R)	The PGI-R is a rating of change of 18 autism symptoms compared to baseline, with each symptom rated on a Likert scale from -3 (much worse) to zero (no change) to +3 (much better). We report the average change of the 18 symptoms, so the possible range is from -3 to +3.	Baseline and 10 weeks (end of treatment)
Blood Safety Markers (Assessment of Blood Chemistry Panel and Complete Blood Count)	Number of participants who had a clinically significant change in their blood safety markers (comprehensive metabolic panel including kidney/liver function and complete blood count with differential)	Baseline and 10 weeks (end of treatment)
Childhood Autism Rating Scale (CARS)	The CARS is an assessment of 15 autism-related symptoms. Each item is scored on a scale of 1 (no symptoms) to 4 (severe symptoms), and the scores for each symptom are summed to obtain a total score. So, the possible scores range from 15 to 60, with a higher score indicating greater severity.	Baseline and 10 weeks (end of treatment)
Social Responsiveness Scale (SRS)	The SRS is an assessment of social skills based on 65 items, where each item is scored on a range of 0 (no symptoms) to 3 (severe symptoms). The total score is a sum of the individual scores, so the total possible range is from 0 to 195, with a higher score indicating greater severity of symptoms.	Baseline and 10 weeks (end of treatment)
Short Sensory Profile	The Short Sensory Profile is an assessment of sensory problems. However, the data on this scale was not collected due to administrative error.	baseline and 10 weeks
Vineland Adaptive Behavior Scale (VABS)	The VABS is an assessment of adaptive behaviors, and we analyzed it to determine the developmental age of the participants, with possible scores ranging from 0 years to 21 years.	baseline and 18 weeks (8 weeks after treatment stopped)
Daily Stool Record (DSR)	The DSR is a record of the type of stool that a participant had each day, using the Bristol Stool Form which ranges from 1 (very hard) to 7 (liquid). We then analyzed the data as % days of abnormal stools over a 14 day period, where an abnormal stool is defined as an unusually hard stool (types 1-2), unusually soft stool (types 6-7), or no stool that day. So, the possible range of % days of abnormal stools is from 0% to 100%, with a higher score indicating a more severe problem.	Baseline and 10 weeks (end of treatment)

Collaborators and Investigators

• Sponsor: Arizona State University
• Collaborators: University of Minnesota; University of Arizona; Northern Arizona University

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start: July 1, 2014
• Primary Completion (ACTUAL): April 1, 2016
• Study Completion (ACTUAL): April 1, 2016

Study Registration Dates

• First Submitted: March 30, 2015
• First Submitted That Met QC Criteria: July 21, 2015
• First Posted (ESTIMATE): July 22, 2015

Study Record Updates

• Last Update Posted (ACTUAL): December 18, 2019
• Last Update Submitted That Met QC Criteria: November 27, 2019
• Last Verified: November 1, 2019

More Information

Terms related to this study

• Keywords: children
• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Autistic Disorder; Autism Spectrum Disorder; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Enzyme Inhibitors; Gastrointestinal Agents; Anti-Bacterial Agents; Anti-Ulcer Agents; Proton Pump Inhibitors; Vancomycin; Omeprazole
• Other Study ID Numbers: STUDY00001053