Trial Outcomes & Findings for Fecal Microbiota Transplantation in Pediatric Patients (NCT NCT02108821)

NCT ID: NCT02108821

Last Updated: 2021-02-04

Results Overview

Adverse events recorded to determine safety of Fecal Microbiome Transplant in the treatment of children with IBD.

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 23 participants
• Primary outcome timeframe: 6 months
• Results posted on: 2021-02-04

Participant Flow

Participant milestones

Measure	Fecal Microbiome Transplantation-open Label Fecal Microbiome Transplantation will be done at the time of EGD and colonoscopy. A parent or sibling or a healthy relative will be tested for several infections like hepatitis, H. Pylori, HIV, syphilis, ova and parasites, culture and C.diff. They will fill out a donor questionnaire used for blood donors prior to the sample collection. After eligibility criteria have been met, appropriate consent has been obtained, and the screening labs have been assessed, the fecal transplant procedure will take place in the procedure center at Children's Hospital of Pittsburgh. Fresh stool sample will be obtained from the donor. The fecal sample will be prepared for transplantation in a designated area in the procedure center. Frequency: once. Duration: Approximately 1 hour Fecal Microbiota Transplantation (FMT): The process of fecal microbiota transplantation (FMT), where fecal bacteria from a healthy individual is transferred into a recipient as an alternative therapy for individuals affected
Overall Study STARTED	23
Overall Study COMPLETED	23
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Fecal Microbiota Transplantation in Pediatric Patients

Baseline characteristics by cohort

Measure	Fecal Microbiome Transplantation n=23 Participants Use of Fecal Microbiome Transplantation for participants with IBD.
Age, Categorical <=18 years	19 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	10 years n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	20 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	23 Participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Adverse events recorded to determine safety of Fecal Microbiome Transplant in the treatment of children with IBD.

Outcome measures

Measure	Overall Study n=21 Participants Overall Study Events	Fecal Microbiome Transplantation With Crohn's Disease Use of Fecal Microbiome Transplantation for participants with Crohn's Disease.
Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term Moderate Abdominal Pain	11 Events	—
Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term Diarrhea	5 Events	—
Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term Flatulence and Bloating	5 Events	—
Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term Emesis	3 Events	—
Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term Bloody Stools	2 Events	—
Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term Nausea	2 Events	—
Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term Fever	1 Events	—

SECONDARY outcome

Timeframe: Baseline, Day 30, Day 180

Population: Only participants that responded to treatment

Efficacy outcomes scored via Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI) analysis.

Scored at baseline, day 30, and at day 180 for responders.

The PUCAI score range is 0-85, where the higher the number the more severe the disease (A score of 65 and up is considered severe, a score of 35-64 is considered moderate, and a score of 10-34 is considered mild). The PUCAI is the scoring system used for patients with ulcerative colitis/indeterminate colitis.

The PCDAI score range is 0-100, where the higher the number the more severe the disease (A score of greater than 30 is considered moderate to severe, a score of 11-30 is considered mild, and a score of 10 or less is considered inactive disease). The PCDAI is the scoring system used for patients with crohn's disease.

Outcome measures

Measure	Overall Study n=3 Participants Overall Study Events	Fecal Microbiome Transplantation With Crohn's Disease n=3 Participants Use of Fecal Microbiome Transplantation for participants with Crohn's Disease.
Examine the Efficacy of Fecal Microbiome Transplant in the Treatment of Children With IBD Using the Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI) Analysis. Baseline	40 score on a scale Interval 38.04 to 41.96	11.6 score on a scale Interval 9.64 to 13.56
Examine the Efficacy of Fecal Microbiome Transplant in the Treatment of Children With IBD Using the Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI) Analysis. Day 30 in responders	11.6 score on a scale Interval 9.64 to 13.56	1.6 score on a scale Interval 0.0 to 3.56
Examine the Efficacy of Fecal Microbiome Transplant in the Treatment of Children With IBD Using the Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI) Analysis. Day 180 in responders	21.6 score on a scale Interval 19.64 to 23.56	0 score on a scale Interval 0.0 to 0.0

Adverse Events

Fecal Microbiota Transplantation

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Fecal Microbiota Transplantation n=23 participants at risk Use of Fecal Microbiota Transplantation for participants with IBD.
Gastrointestinal disorders Abdominal Pain	47.8% 11/23 • 6 months
Gastrointestinal disorders Diarrhea	21.7% 5/23 • 6 months
Gastrointestinal disorders Flatulence and bloating	21.7% 5/23 • 6 months
Gastrointestinal disorders Emesis	13.0% 3/23 • 6 months
Gastrointestinal disorders Bloody stools	8.7% 2/23 • 6 months
Gastrointestinal disorders Nausea	8.7% 2/23 • 6 months
General disorders Fever	4.3% 1/23 • 6 months

Additional Information

Alka Goyal MD

Children's Mercy Hospital, Kansas City

Phone: 816-302-3069

Email: agoyal4@stanford.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place