Trial Outcomes & Findings for Beneficial Bacteria Treatment for Autism (NCT NCT02504554)
NCT ID: NCT02504554
Last Updated: 2025-09-12
Results Overview
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.
COMPLETED
PHASE1/PHASE2
18 participants
Baseline and 10 weeks (end of treatment)
2025-09-12
Participant Flow
Participant milestones
| Measure |
Oral Group
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
|
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.
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
|
|---|---|---|
|
Overall Study
STARTED
|
12
|
6
|
|
Overall Study
COMPLETED
|
12
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Beneficial Bacteria Treatment for Autism
Baseline characteristics by cohort
| Measure |
Oral Group
n=12 Participants
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
|
Rectal Group
n=6 Participants
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
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
12 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
18 Participants
n=27 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=93 Participants
|
5 Participants
n=4 Participants
|
16 Participants
n=27 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=93 Participants
|
6 participants
n=4 Participants
|
18 participants
n=27 Participants
|
PRIMARY outcome
Timeframe: Baseline and 10 weeks (end of treatment)Population: We report the data on all participants, regardless of how the initial dose of bacteria was administered (oral or rectal).
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.
Outcome measures
| Measure |
Combined Group
n=18 Participants
Combination of both groups
|
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.
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
|
|---|---|---|
|
Gastrointestinal Symptom Responsiveness Scale (GSRS)
Baseline
|
2.74 units on a scale
Standard Deviation 0.57
|
—
|
|
Gastrointestinal Symptom Responsiveness Scale (GSRS)
10 weeks
|
1.31 units on a scale
Standard Deviation 0.45
|
—
|
SECONDARY outcome
Timeframe: Baseline and 10 weeks (end of treatment)Population: We report the data on all participants regardless of how the initial dose of bacteria was administered.
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.
Outcome measures
| Measure |
Combined Group
n=18 Participants
Combination of both groups
|
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.
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
|
|---|---|---|
|
Parent Global Impressions-Revised (PGI-R)
|
1.4 units on a scale
Standard Deviation 0.9
|
—
|
SECONDARY outcome
Timeframe: Baseline and 10 weeks (end of treatment)Population: We report the data on 18 participants, regardless of how the initial dose of bacteria was administered.
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)
Outcome measures
| Measure |
Combined Group
n=18 Participants
Combination of both groups
|
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.
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
|
|---|---|---|
|
Blood Safety Markers (Assessment of Blood Chemistry Panel and Complete Blood Count)
|
0 participants
|
—
|
SECONDARY outcome
Timeframe: Baseline and 10 weeks (end of treatment)Population: We report the data on all participants, regardless of how the initial dose of bacteria was administered.
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.
Outcome measures
| Measure |
Combined Group
n=18 Participants
Combination of both groups
|
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.
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
|
|---|---|---|
|
Childhood Autism Rating Scale (CARS)
Baseline
|
39.7 units on a scale
Standard Deviation 5.5
|
—
|
|
Childhood Autism Rating Scale (CARS)
10 weeks
|
34.1 units on a scale
Standard Deviation 4.7
|
—
|
SECONDARY outcome
Timeframe: Baseline and 10 weeks (end of treatment)Population: We report the data on 18 participants, regardless of how the initial dose of bacteria was administered.
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.
Outcome measures
| Measure |
Combined Group
n=18 Participants
Combination of both groups
|
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.
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
|
|---|---|---|
|
Social Responsiveness Scale (SRS)
Baseline
|
116.2 units on a scale
Standard Deviation 24.9
|
—
|
|
Social Responsiveness Scale (SRS)
10 weeks
|
97.8 units on a scale
Standard Deviation 27.9
|
—
|
SECONDARY outcome
Timeframe: baseline and 10 weeksPopulation: Due to a logistical error, this data was not collected
The Short Sensory Profile is an assessment of sensory problems. However, the data on this scale was not collected due to administrative error.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: baseline and 18 weeks (8 weeks after treatment stopped)Population: We report the data on all participants, regardless of how they received the initial dose of bacteria.
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.
Outcome measures
| Measure |
Combined Group
n=18 Participants
Combination of both groups
|
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.
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
|
|---|---|---|
|
Vineland Adaptive Behavior Scale (VABS)
Baseline
|
5.39 years
Standard Deviation 2.48
|
—
|
|
Vineland Adaptive Behavior Scale (VABS)
18 weeks
|
6.84 years
Standard Deviation 2.81
|
—
|
SECONDARY outcome
Timeframe: Baseline and 10 weeks (end of treatment)Population: We report the data on all participants, regardless of how the initial dose of bacteria was administered.
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.
Outcome measures
| Measure |
Combined Group
n=18 Participants
Combination of both groups
|
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.
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
|
|---|---|---|
|
Daily Stool Record (DSR)
Baseline
|
62 percentage of days with abnormal stool
Standard Deviation 22
|
—
|
|
Daily Stool Record (DSR)
10 weeks
|
34 percentage of days with abnormal stool
Standard Deviation 24
|
—
|
Adverse Events
All Groups
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
All Groups
n=18 participants at risk
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.
|
|---|---|
|
Skin and subcutaneous tissue disorders
rash
|
5.6%
1/18 • Number of events 1 • During the 10 weeks of treatment, and the 8 weeks of follow-up (18 weeks total).
|
|
Gastrointestinal disorders
nausea/vomiting
|
5.6%
1/18 • Number of events 1 • During the 10 weeks of treatment, and the 8 weeks of follow-up (18 weeks total).
|
|
Nervous system disorders
hyperactivity and/or irritability
|
66.7%
12/18 • Number of events 12 • During the 10 weeks of treatment, and the 8 weeks of follow-up (18 weeks total).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place