BBT: Beneficial Bacteria Treatment for Autism
Study Details
Study Description
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.
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
|
Phase 1/Phase 2 |
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 Design
Arms and Interventions
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
|
Outcome Measures
Primary Outcome Measures
- 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
- 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.
- 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.
- 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.
Eligibility Criteria
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
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Arizona State University
- Northern Arizona University
- University of Arizona
- University of Minnesota
Investigators
- Principal Investigator: James B Adams, PhD, Arizona State University
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- STUDY00001053
Study Results
Participant Flow
Recruitment Details | |
---|---|
Pre-assignment Detail |
Arm/Group Title | Oral Group | Rectal Group |
---|---|---|
Arm/Group Description | This group will receive all treatments orally. The treatments include a combination of Vancomycin, MoviPrep, Prilosec, and human fecal material; processed, frozen. oral Vancomycin: an antibiotic MoviPrep: a bowel cleanse Prilosec: a stomach acid suppressan human fecal material; processed, frozen administered orally: human fecal material; processed, frozen, administered orally | 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 Vancomycin: an antibiotic MoviPrep: a bowel cleanse Prilosec: a stomach acid suppressan human fecal material; processed, frozen; administered orally and rectally: human fecal material; processed, frozen; administered orally and rectally |
Period Title: Overall Study | ||
STARTED | 12 | 6 |
COMPLETED | 12 | 6 |
NOT COMPLETED | 0 | 0 |
Baseline Characteristics
Arm/Group Title | Oral Group | Rectal Group | Total |
---|---|---|---|
Arm/Group Description | This group will receive all treatments orally. The treatments include a combination of Vancomycin, MoviPrep, Prilosec, and human fecal material; processed, frozen. oral Vancomycin: an antibiotic MoviPrep: a bowel cleanse Prilosec: a stomach acid suppressan human fecal material; processed, frozen administered orally: human fecal material; processed, frozen, administered orally | 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 Vancomycin: an antibiotic MoviPrep: a bowel cleanse Prilosec: a stomach acid suppressan human fecal material; processed, frozen; administered orally and rectally: human fecal material; processed, frozen; administered orally and rectally | Total of all reporting groups |
Overall Participants | 12 | 6 | 18 |
Age (Count of Participants) | |||
<=18 years |
12
100%
|
6
100%
|
18
100%
|
Between 18 and 65 years |
0
0%
|
0
0%
|
0
0%
|
>=65 years |
0
0%
|
0
0%
|
0
0%
|
Sex: Female, Male (Count of Participants) | |||
Female |
1
8.3%
|
1
16.7%
|
2
11.1%
|
Male |
11
91.7%
|
5
83.3%
|
16
88.9%
|
Region of Enrollment (participants) [Number] | |||
United States |
12
100%
|
6
100%
|
18
100%
|
Outcome Measures
Title | Gastrointestinal Symptom Responsiveness Scale (GSRS) |
---|---|
Description | 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. |
Time Frame | Baseline and 10 weeks (end of treatment) |
Outcome Measure Data
Analysis Population Description |
---|
We report the data on all participants, regardless of how the initial dose of bacteria was administered (oral or rectal). |
Arm/Group Title | Combined Group |
---|---|
Arm/Group Description | Combination of both groups |
Measure Participants | 18 |
Baseline |
2.74
(0.57)
|
10 weeks |
1.31
(0.45)
|
Statistical Analysis 1
Statistical Analysis Overview | Comparison Group Selection | Combined Group |
---|---|---|
Comments | Wilcoxon signed rank analysis of symptoms at 10 weeks (end of treatment) vs. baseline | |
Type of Statistical Test | Other | |
Comments | ||
Statistical Test of Hypothesis | p-Value | <0.001 |
Comments | no adjustment for multiple hypothesis testing since this was an exploratory study. the p-value listed is the actual result from analysis of the study data. | |
Method | Wilcoxon (Mann-Whitney) | |
Comments |
Title | Parent Global Impressions-Revised (PGI-R) |
---|---|
Description | 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. |
Time Frame | Baseline and 10 weeks (end of treatment) |
Outcome Measure Data
Analysis Population Description |
---|
We report the data on all participants regardless of how the initial dose of bacteria was administered. |
Arm/Group Title | Combined Group |
---|---|
Arm/Group Description | Combination of both groups |
Measure Participants | 18 |
Mean (Standard Deviation) [units on a scale] |
1.4
(0.9)
|
Statistical Analysis 1
Statistical Analysis Overview | Comparison Group Selection | Combined Group |
---|---|---|
Comments | ||
Type of Statistical Test | Other | |
Comments | paired 2-sided t-test comparing baseline and 10 weeks (end of treatment) | |
Statistical Test of Hypothesis | p-Value | <0.001 |
Comments | no adjustment for multiple comparisons | |
Method | t-test, 2 sided | |
Comments | paired t-test, 2 sided |
Title | Blood Safety Markers (Assessment of Blood Chemistry Panel and Complete Blood Count) |
---|---|
Description | 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) |
Time Frame | Baseline and 10 weeks (end of treatment) |
Outcome Measure Data
Analysis Population Description |
---|
We report the data on 18 participants, regardless of how the initial dose of bacteria was administered. |
Arm/Group Title | Combined Group |
---|---|
Arm/Group Description | Combination of both groups |
Measure Participants | 18 |
Number [participants] |
0
0%
|
Title | Childhood Autism Rating Scale (CARS) |
---|---|
Description | 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. |
Time Frame | Baseline and 10 weeks (end of treatment) |
Outcome Measure Data
Analysis Population Description |
---|
We report the data on all participants, regardless of how the initial dose of bacteria was administered. |
Arm/Group Title | Combined Group |
---|---|
Arm/Group Description | Combination of both groups |
Measure Participants | 18 |
Baseline |
39.7
(5.5)
|
10 weeks |
34.1
(4.7)
|
Statistical Analysis 1
Statistical Analysis Overview | Comparison Group Selection | Combined Group |
---|---|---|
Comments | Wilcoxon signed-rank test comparing the baseline score vs. the score at 10 weeks (end of treatment). | |
Type of Statistical Test | Other | |
Comments | ||
Statistical Test of Hypothesis | p-Value | <0.001 |
Comments | no adjustment for multiple comparisons The p-value listed is the result for the actual analysis of the data. | |
Method | Wilcoxon (Mann-Whitney) | |
Comments |
Title | Social Responsiveness Scale (SRS) |
---|---|
Description | 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. |
Time Frame | Baseline and 10 weeks (end of treatment) |
Outcome Measure Data
Analysis Population Description |
---|
We report the data on 18 participants, regardless of how the initial dose of bacteria was administered. |
Arm/Group Title | Combined Group |
---|---|
Arm/Group Description | Combination of both groups |
Measure Participants | 18 |
Baseline |
116.2
(24.9)
|
10 weeks |
97.8
(27.9)
|
Statistical Analysis 1
Statistical Analysis Overview | Comparison Group Selection | Combined Group |
---|---|---|
Comments | Wilcoxon signed-rank test comparing scores at baseline vs. 10 weeks (end of treatment) | |
Type of Statistical Test | Other | |
Comments | ||
Statistical Test of Hypothesis | p-Value | <0.001 |
Comments | no adjustment for multiple comparisons the p-value listed is the actual result for the study results. | |
Method | Wilcoxon (Mann-Whitney) | |
Comments |
Title | Short Sensory Profile |
---|---|
Description | The Short Sensory Profile is an assessment of sensory problems. However, the data on this scale was not collected due to administrative error. |
Time Frame | baseline and 10 weeks |
Outcome Measure Data
Analysis Population Description |
---|
Due to a logistical error, this data was not collected |
Arm/Group Title | Oral Group | Rectal Group |
---|---|---|
Arm/Group Description | This group will receive all treatments orally. The treatments include a combination of Vancomycin, MoviPrep, Prilosec, and human fecal material; processed, frozen. oral Vancomycin: an antibiotic MoviPrep: a bowel cleanse Prilosec: a stomach acid suppressan human fecal material; processed, frozen administered orally: human fecal material; processed, frozen, administered orally | 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 Vancomycin: an antibiotic MoviPrep: a bowel cleanse Prilosec: a stomach acid suppressan human fecal material; processed, frozen; administered orally and rectally: human fecal material; processed, frozen; administered orally and rectally |
Measure Participants | 0 | 0 |
Title | Vineland Adaptive Behavior Scale (VABS) |
---|---|
Description | 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. |
Time Frame | baseline and 18 weeks (8 weeks after treatment stopped) |
Outcome Measure Data
Analysis Population Description |
---|
We report the data on all participants, regardless of how they received the initial dose of bacteria. |
Arm/Group Title | Combined Group |
---|---|
Arm/Group Description | Combination of both groups |
Measure Participants | 18 |
Baseline |
5.39
(2.48)
|
18 weeks |
6.84
(2.81)
|
Statistical Analysis 1
Statistical Analysis Overview | Comparison Group Selection | Combined Group |
---|---|---|
Comments | 2-sided t-test comparing the group at baseline vs. 18 weeks (8 weeks after end of treatment) | |
Type of Statistical Test | Other | |
Comments | ||
Statistical Test of Hypothesis | p-Value | <0.001 |
Comments | no adjustment for multiple comparisons the p-value listed is the actual result for the analysis of the study data | |
Method | t-test, 2 sided | |
Comments |
Title | Daily Stool Record (DSR) |
---|---|
Description | 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. |
Time Frame | Baseline and 10 weeks (end of treatment) |
Outcome Measure Data
Analysis Population Description |
---|
We report the data on all participants, regardless of how the initial dose of bacteria was administered. |
Arm/Group Title | Combined Group |
---|---|
Arm/Group Description | Combination of both groups |
Measure Participants | 18 |
Baseline |
62
(22)
|
10 weeks |
34
(24)
|
Statistical Analysis 1
Statistical Analysis Overview | Comparison Group Selection | Combined Group |
---|---|---|
Comments | Wilcoxon signed rank test was used to compare scores at baseline vs. at 10 weeks (end of treatment) | |
Type of Statistical Test | Other | |
Comments | ||
Statistical Test of Hypothesis | p-Value | 0.002 |
Comments | no adjustment for multiple comparisons | |
Method | Wilcoxon (Mann-Whitney) | |
Comments |
Adverse Events
Time Frame | During the 10 weeks of treatment, and the 8 weeks of follow-up (18 weeks total). | |
---|---|---|
Adverse Event Reporting Description | ||
Arm/Group Title | All Groups | |
Arm/Group Description | We report the data on 18 participants, regardless of how the initial dose of bacteria was administered. The reason is that all but one of the adverse events occurred during the vancomycin phase, which was common to both groups, and occurred before the randomization to receive the microbiota either orally or rectally. The only exception is one participant who had initial nausea/vomiting upon receiving the initial oral dose of microbiota, and they were switched from the oral group to the rectal group, which they tolerated without incident. There were no adverse effects from the microbiota. | |
All Cause Mortality |
||
All Groups | ||
Affected / at Risk (%) | # Events | |
Total | 0/18 (0%) | |
Serious Adverse Events |
||
All Groups | ||
Affected / at Risk (%) | # Events | |
Total | 0/18 (0%) | |
Other (Not Including Serious) Adverse Events |
||
All Groups | ||
Affected / at Risk (%) | # Events | |
Total | 12/18 (66.7%) | |
Gastrointestinal disorders | ||
nausea/vomiting | 1/18 (5.6%) | 1 |
Nervous system disorders | ||
hyperactivity and/or irritability | 12/18 (66.7%) | 12 |
Skin and subcutaneous tissue disorders | ||
rash | 1/18 (5.6%) | 1 |
Limitations/Caveats
More Information
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title | James Adams |
---|---|
Organization | Arizona State University |
Phone | 480 965 3316 |
jim.adams@asu.edu |
- STUDY00001053