Beneficial Bacteria Treatment for Autism

Study Overview
=================
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.

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

Conditions
-----------------
Gastrointestinal Problems, Autism Spectrum Disorders

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


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Children ages 7-17 years Diagnosis of autism per Autism Diagnostic Interview-Revised (ADI-R) 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 No changes in medications, supplements, diet, therapies, or education in last 3 months, and no intention to change them during clinical trial General good physical health aside from gastrointestinal problems Cognitive Ability to Provide Informed Assent Exclusion Criteria: Antibiotics in last 6 months Probiotics in last 3 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

Ages Eligible for Study
-----------------
Minimum Age: 7 Years
Maximum Age: 17 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Oral Group<br>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<br>* an antibiotic<br>Drug: MoviPrep<br>* a bowel cleanse<br>Drug: Prilosec<br>* a stomach acid suppressan<br>Biological: human fecal material; processed, frozen administered orally<br>* human fecal material; processed, frozen, administered orally<br>|
| Experimental: Rectal Group<br>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<br>* an antibiotic<br>Drug: MoviPrep<br>* a bowel cleanse<br>Drug: Prilosec<br>* a stomach acid suppressan<br>Biological: human fecal material; processed, frozen; administered orally and rectally<br>* human fecal material; processed, frozen; administered orally and rectally<br>|

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) | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
children