Beneficial Bacteria Treatment for Autism

Phase 1

Completed

• Conditions: Gastrointestinal Problems; Autism Spectrum Disorders
• Interventions: 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

• Registration Number: NCT02504554
• Lead Sponsor: Arizona State University

Brief Summary

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.

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 Timeline

• Study Start: 2014-07
• Study First Submitted: 2015-03-30
• Study First Submitted QC: 2015-07-21
• Study First Posted: 2015-07-22
• Primary Completion: 2016-04
• Study Completion: 2016-04
• Results First Submitted: 2018-02-09
• Status Verified: 2019-11
• Results First Submitted QC: 2019-11-27
• Last Update Submitted: 2019-11-27
• Results First Posted: 2019-12-18
• Last Update Posted: 2019-12-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 18

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Oral Group	MoviPrep	This group will receive all treatments orally. The treatments include a combination of Vancomycin, MoviPrep, Prilosec, and human fecal material; processed, frozen.
Oral Group	Prilosec	This group will receive all treatments orally. The treatments include a combination of Vancomycin, MoviPrep, Prilosec, and human fecal material; processed, frozen.
Oral Group	human fecal material; processed, frozen administered orally	This group will receive all treatments orally. The treatments include a combination of Vancomycin, MoviPrep, Prilosec, and human fecal material; processed, frozen.
Rectal Group	oral Vancomycin	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.
Rectal Group	MoviPrep	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.
Oral Group	oral Vancomycin	This group will receive all treatments orally. The treatments include a combination of Vancomycin, MoviPrep, Prilosec, and human fecal material; processed, frozen.
Rectal Group	Prilosec	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.
Rectal Group	human fecal material; processed, frozen; administered orally and rectally	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.

Primary Outcome Measures

Name	Time	Method
Gastrointestinal Symptom Responsiveness Scale (GSRS)	Baseline and 10 weeks (end of treatment)	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.

Secondary Outcome Measures

Name	Time	Method
Short Sensory Profile	baseline and 10 weeks	The Short Sensory Profile is an assessment of sensory problems.; However, the data on this scale was not collected due to administrative error.
Parent Global Impressions-Revised (PGI-R)	Baseline and 10 weeks (end of treatment)	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.
Blood Safety Markers (Assessment of Blood Chemistry Panel and Complete Blood Count)	Baseline and 10 weeks (end of treatment)	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)
Childhood Autism Rating Scale (CARS)	Baseline and 10 weeks (end of treatment)	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.
Social Responsiveness Scale (SRS)	Baseline and 10 weeks (end of treatment)	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.
Vineland Adaptive Behavior Scale (VABS)	baseline and 18 weeks (8 weeks after treatment stopped)	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.
Daily Stool Record (DSR)	Baseline and 10 weeks (end of treatment)	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.