Fecal Microbiota Transplantation in Pediatric Patients (FMT)

• ClinicalTrials.gov Identifier: NCT02108821
• Recruitment Status: Completed
• First Posted: April 9, 2014
• Results First Posted: February 4, 2021
• Last Update Posted: February 4, 2021
• Sponsor: Children's Mercy Hospital Kansas City
• Collaborators: University of Pittsburgh; Stanford University
• Information provided by (Responsible Party): Children's Mercy Hospital Kansas City

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Conditions: Inflammatory Bowel Diseases (IBD); Crohn's Disease (CD); Ulcerative Colitis (UC)
• Intervention: Biological: Fecal Microbiota Transplantation (FMT)
• Enrollment: 23

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Fecal Microbiome Transplantation-open Label
Arm/Group Description	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
Period Title: Overall Study
Started [1]	23
Completed [2]	23
Not Completed	0
[1] March 12, 2014; [2] September 30th, 2016

Baseline Characteristics

Arm/Group Title		Fecal Microbiome Transplantation
Arm/Group Description		Use of Fecal Microbiome Transplantation for participants with IBD.
Overall Number of Baseline Participants		23
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	23 participants
	<=18 years	19 82.6%
	Between 18 and 65 years	4 17.4%
	>=65 years	0 0.0%
Age, Continuous; Median (Full Range); Unit of measure: Years
	Number Analyzed	23 participants
		10; (8 to 21)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	23 participants
	Female	11 47.8%
	Male	12 52.2%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	23 participants
	American Indian or Alaska Native	0 0.0%
	Asian	3 13.0%
	Native Hawaiian or Other Pacific Islander	0 0.0%
	Black or African American	0 0.0%
	White	20 87.0%
	More than one race	0 0.0%
	Unknown or Not Reported	0 0.0%
Region of Enrollment [1]; Measure Type: Count of Participants; Unit of measure: Participants
United States	Number Analyzed	23 participants
		23 100.0%
		[1] Measure Description: Outpatient Gastroenterology clinic Children's Hospital of Pittsburgh. Pittsburgh. Pennsylvania. USA

Outcome Measures

1. Primary Outcome

Title	Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term
Description	Adverse events recorded to determine safety of Fecal Microbiome Transplant in the treatment of children with IBD.
Time Frame	6 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Overall Study
Arm/Group Description:	Overall Study Events
Overall Number of Participants Analyzed	21
Measure Type: Number; Unit of Measure: Events
Moderate Abdominal Pain	11
Diarrhea	5
Flatulence and Bloating	5
Emesis	3
Bloody Stools	2
Nausea	2
Fever	1

2. Secondary Outcome

Title	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.
Description	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.
Time Frame	Baseline, Day 30, Day 180

Outcome Measure Data

Analysis Population Description

Only participants that responded to treatment

Arm/Group Title	Fecal Microbiome Transplantation Ulcerative Colitis/Indeterminate Colitis	Fecal Microbiome Transplantation With Crohn's Disease
Arm/Group Description:	Use of Fecal Microbiome Transplantation for participants with Ulcerative Colitis/Indeterminate Colitis.	Use of Fecal Microbiome Transplantation for participants with Crohn's Disease.
Overall Number of Participants Analyzed	3	3
Mean (95% Confidence Interval); Unit of Measure: score on a scale
Baseline	40; (38.04 to 41.96)	11.6; (9.64 to 13.56)
Day 30 in responders	11.6; (9.64 to 13.56)	1.6; (0 to 3.56)
Day 180 in responders	21.6; (19.64 to 23.56)	0; (0 to 0)

Adverse Events

Time Frame	6 months
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Fecal Microbiota Transplantation
Arm/Group Description	Use of Fecal Microbiota Transplantation for participants with IBD.
All-Cause Mortality
	Fecal Microbiota Transplantation
	Affected / at Risk (%)
Total	0/23 (0.00%)
Serious Adverse Events
	Fecal Microbiota Transplantation
	Affected / at Risk (%)
Total	0/23 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	4%
	Fecal Microbiota Transplantation
	Affected / at Risk (%)
Total	12/23 (52.17%)
Gastrointestinal disorders
Abdominal Pain †	11/23 (47.83%)
Diarrhea †	5/23 (21.74%)
Flatulence and bloating †	5/23 (21.74%)
Emesis †	3/23 (13.04%)
Bloody stools †	2/23 (8.70%)
Nausea †	2/23 (8.70%)
General disorders
Fever †	1/23 (4.35%)
† Indicates events were collected by systematic assessment

Limitations and Caveats

small number of subjects, open label trial, fecal calprotectin not collected in every subject so response measurement was subjective

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

• Name/Title: Alka Goyal MD
• Organization: Children's Mercy Hospital, Kansas City
• Phone: 816-302-3069
• EMail: agoyal4@stanford.edu

• Responsible Party: Children's Mercy Hospital Kansas City
• ClinicalTrials.gov Identifier: NCT02108821
• Other Study ID Numbers: PRO13030081
• First Submitted: March 21, 2014
• First Posted: April 9, 2014
• Results First Submitted: June 16, 2018
• Results First Posted: February 4, 2021
• Last Update Posted: February 4, 2021