Trial Outcomes & Findings for Bacteriotherapy in Pediatric Inflammatory Bowel Disease (NCT NCT01757964)
NCT ID: NCT01757964
Last Updated: 2017-05-30
Results Overview
The primary outcome measure is based on estimating the responder rate. This is defined as the proportion of patients with response to therapy by a drop of 10 or more points in PUCAI/PCDAI scoring. PUCAI/PCDAI are validated activity indexes for pediatric Ulcerative colitis and Crohn's disease, respectively. PUCAI scoring ranges from 0 to 85, with disease remission less than 10, mild disease activity between 10 - 35, moderate disease activity from 35 - 65, and severe disease activity above 65. PCDAI scoring ranges from 0 to 100; with remission being less than 10, mild disease from 10 to 30, and moderate to severe disease greater than 30.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
13 participants
Primary outcome timeframe
2 weeks
Results posted on
2017-05-30
Participant Flow
Participant milestones
| Measure |
Bacteriotherapy: Crohn's Disease
Initial evaluation: Study subject recipient had laboratory tests Stool Transplantation: Study subject recipients received premedication prior to fecal transplant, which included rifaximin. Study subject recipients also receive Omeprazole (1mg/kg orally) on the day before and morning of procedure. Transplant recipient also MiraLAX for 2 days prior to FMT. For the FMT, a nasogastric (NG) tube was placed. Approximately 30g of donor stool was mixed with 100ml of normal saline and blenderized until a homogenous texture was achieved Post Transplantation follow-up: Study subject recipients were called 2 days after transplantation. Study subject recipients had clinical follow-up at 2 weeks, 6 weeks and 12 weeks. Standardized questionnaires, PUCAI, were completed during each study visit.
|
Bacteriotherapy: Ulcerative Colitis
Initial evaluation: Study subject recipient had laboratory tests Stool Transplantation: Study subject recipients received premedication prior to fecal transplant, which included rifaximin. Study subject recipients also receive Omeprazole (1mg/kg orally) on the day before and morning of procedure. Transplant recipient also MiraLAX for 2 days prior to FMT. For the FMT, a nasogastric (NG) tube was placed. Approximately 30g of donor stool was mixed with 100ml of normal saline and blenderized until a homogenous texture was achieved Post Transplantation follow-up: Study subject recipients were called 2 days after transplantation. Study subject recipients had clinical follow-up at 2 weeks, 6 weeks and 12 weeks. Standardized questionnaires, PUCAI, were completed during each study visit.
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
4
|
|
Overall Study
COMPLETED
|
9
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Bacteriotherapy in Pediatric Inflammatory Bowel Disease
Baseline characteristics by cohort
| Measure |
Bacteriotherapy: Crohn's Disease
n=9 Participants
Initial evaluation: Study subject recipient had laboratory tests Stool Transplantation: Study subject recipients received premedication prior to fecal transplant, which included rifaximin. Study subject recipients also receive Omeprazole (1mg/kg orally) on the day before and morning of procedure. Transplant recipient also MiraLAX for 2 days prior to FMT. For the FMT, a nasogastric (NG) tube was placed. Approximately 30g of donor stool was mixed with 100ml of normal saline and blenderized until a homogenous texture was achieved Post Transplantation follow-up: Study subject recipients were called 2 days after transplantation. Study subject recipients had clinical follow-up at 2 weeks, 6 weeks and 12 weeks. Standardized questionnaires, PUCAI, were completed during each study visit.
|
Bacteriotherapy: Ulcerative Colitis
n=4 Participants
Initial evaluation: Study subject recipient had laboratory tests Stool Transplantation: Study subject recipients received premedication prior to fecal transplant, which included rifaximin. Study subject recipients also receive Omeprazole (1mg/kg orally) on the day before and morning of procedure. Transplant recipient also MiraLAX for 2 days prior to FMT. For the FMT, a nasogastric (NG) tube was placed. Approximately 30g of donor stool was mixed with 100ml of normal saline and blenderized until a homogenous texture was achieved Post Transplantation follow-up: Study subject recipients were called 2 days after transplantation. Study subject recipients had clinical follow-up at 2 weeks, 6 weeks and 12 weeks. Standardized questionnaires, PUCAI, were completed during each study visit.
|
Total
n=13 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 weeksThe primary outcome measure is based on estimating the responder rate. This is defined as the proportion of patients with response to therapy by a drop of 10 or more points in PUCAI/PCDAI scoring. PUCAI/PCDAI are validated activity indexes for pediatric Ulcerative colitis and Crohn's disease, respectively. PUCAI scoring ranges from 0 to 85, with disease remission less than 10, mild disease activity between 10 - 35, moderate disease activity from 35 - 65, and severe disease activity above 65. PCDAI scoring ranges from 0 to 100; with remission being less than 10, mild disease from 10 to 30, and moderate to severe disease greater than 30.
Outcome measures
| Measure |
Bacteriotherapy
n=13 Participants
Study stool recipient's will receive approximately 30 grams of processed donor stool through a tube into their stomach for the transplant.
Bacteriotherapy
|
|---|---|
|
Number of Participants Who Responded to Stool Translplantation By 2 Weeks as Determined by Pediatric Ulcerative Colitis Activity Index (PUCAI)/Pediatric Crohn's Disease Activity Index (PCDAI) Scoring
|
13 participants
|
Adverse Events
Bacteriotherapy
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Bacteriotherapy
n=13 participants at risk
Initial evaluation: Study subject recipient had laboratory tests Stool Transplantation: Study subject recipients received premedication prior to fecal transplant, which included rifaximin. Study subject recipients also receive Omeprazole (1mg/kg orally) on the day before and morning of procedure. Transplant recipient also MiraLAX for 2 days prior to FMT. For the FMT, a nasogastric (NG) tube was placed. Approximately 30g of donor stool was mixed with 100ml of normal saline and blenderized until a homogenous texture was achieved Post Transplantation follow-up: Study subject recipients were called 2 days after transplantation. Study subject recipients had clinical follow-up at 2 weeks, 6 weeks and 12 weeks. Standardized questionnaires, PUCAI, were completed during each study visit.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
38.5%
5/13 • Number of events 5
|
|
Gastrointestinal disorders
Mild bloating
|
46.2%
6/13 • Number of events 6
|
|
Gastrointestinal disorders
Diarrhea
|
30.8%
4/13 • Number of events 4
|
|
Gastrointestinal disorders
Flatulence
|
15.4%
2/13 • Number of events 2
|
|
Ear and labyrinth disorders
Rhinorrhea
|
7.7%
1/13 • Number of events 1
|
|
Gastrointestinal disorders
Sore throat
|
15.4%
2/13 • Number of events 2
|
|
General disorders
Nasal congestion
|
7.7%
1/13 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
15.4%
2/13 • Number of events 2
|
Additional Information
David Suskind MD Professor of Pediatrics
University of Washington
Phone: 206-987-2521
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place