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

Tracking Information

• First Submitted Date: March 21, 2014
• First Posted Date: April 9, 2014
• Results First Submitted Date: June 16, 2018
• Results First Posted Date: February 4, 2021
• Last Update Posted Date: February 4, 2021
• Actual Study Start Date: March 2014
• Actual Primary Completion Date: September 30, 2016 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: February 2, 2021)

Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term [ Time Frame: 6 months ]

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

Original Primary Outcome Measures (submitted: April 4, 2014)

Primary Objective: Study the safety of Fecal Microbiome Transplantation in patients with Inflammatory Bowel Diseases by assessing change in fecal microbiome. [ Time Frame: 1 week, 1 month, and 6 months post Fecal Microbiome Transplantation. ]

Correlate safety and patient outcomes with fecal microbiome prior to and after Fecal Microbiome Transplantation.

Current Secondary Outcome Measures (submitted: February 2, 2021)

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. [ Time Frame: Baseline, Day 30, Day 180 ]

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.

Original Secondary Outcome Measures (submitted: April 4, 2014)

• 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, and assessing the change. [ Time Frame: 1 week, 1 month, and 6 months post Fecal Microbiome Transplant. ]
  Examine the efficacy of Fecal Microbiome Transplantation in the treatment of children with IBD using the Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI) analysis, and assessing the change in the PUCAI or the PCDAI analysis.
• Correlate the patient outcomes with the fecal microbiome prior to, and after Fecal Microbiome Transplantation. [ Time Frame: 1 week, 1 month, and 6 months post Fecal Microbiome Transplantation. ]
  Correlate the patient outcomes with the fecal microbiome prior to, and after Fecal Microbiome Transplantation.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Fecal Microbiota Transplantation in Pediatric Patients
• Official Title: A Study of Fecal Microbiota Transplantation in Pediatric Patients With Relapsed Inflammatory Bowel Disease.
• Brief Summary: A disturbance in the diversity of gut bacterial composition could be linked to several immune mediated diseases including inflammatory bowel diseases (IBD). IBD can be classified into Crohn's Disease (CD) and Ulcerative Colitis (UC). Both these diseases occur from abnormal immune reaction to resident gut bacteria.The process of fecal microbiota transplantation (FMT) where fecal bacteria from a healthy individual is transferred into a recipient, has recently received attention as an alternative therapy for individuals affected with these life-altering diseases. In this study, the investigators will perform fecal transplantation on the subjects meeting inclusion criteria, to determine the efficacy and safety of this therapy in subjects with IBD (CD and UC) who are not responding to first line therapy, and are in a flare.
• Detailed Description: 50 subjects (25 subjects with Crohn's Disease and 25 subjects with Ulcerative Colitis) who are 2 to 22 years of age will be enrolled in the trial over 3 years. The fecal donors, preferably a parent or sibling, will be extensively screened for infectious diseases prior to providing stool for the transplant. Patients who are failing primary therapy, are in a flare, and require restaging of their IBD by an endoscopy and colonoscopy will be approached for the study. Standard of care endoscopy and colonoscopy will be performed on each subject and 2 additional biopsies will be taken for analysis. Microbiota analysis will also be performed on both the donor and recipient stool sample prior to transplantation, and on the recipient sample at 1 week, 1 month, and 6 months post transplantation. The primary objective will be to study the safety of FMT in all enrolled subjects. The study will also correlate efficacy and patient outcomes with the fecal microbiome prior to, and after FMT. The secondary objectives are to examine the efficacy of FMT in the treatment of children with IBD using the Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI) analysis. Correlate the patient outcomes with the fecal microbiome prior to, and after FMT.
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Inflammatory Bowel Diseases (IBD)
• Crohn's Disease (CD)
• Ulcerative Colitis (UC)

Intervention

Biological: 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 with these life-altering diseases: Crohn's Disease (CD), and/or Ulcerative Colitis (UC).

Other Name: Fecal Transplantation

Study Arms

Experimental: Fecal Microbiome Transplantation

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

Intervention: Biological: Fecal Microbiota Transplantation (FMT)

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: April 1, 2016): 23
• Original Estimated Enrollment (submitted: April 4, 2014): 50
• Actual Study Completion Date: September 30, 2016
• Actual Primary Completion Date: September 30, 2016 (Final data collection date for primary outcome measure)

Eligibility Criteria

STUDY SUBJECT INCLUSION CRITERIA:

Current IBD patients who have:

• UC patients with a flare due to failure of current therapy and have to undergo esophagogastroduodenoscopy (EGD) and colonoscopy for restaging the disease and escalation of therapy.
• CD patients with ileo colonic or colonic disease who require an EGD and colonoscopy for disease assessment due to a flare or poor control.
• The ability to safely undergo colonoscopy (physical status classification used by the American Society of Anesthesiologists).
• PUCAI score less than sixty five.
• PCDAI score less than forty.

STUDY SUBJECTS EXCLUSION CRITERIA:

• Patients with Crohn's disease: complications like an abscess, phlegmon, stricture, small bowel obstruction, perforation, internal or external fistulization or infection as causes for flare up before being deemed eligible for recruitment to the study. We will check for these complications if a recent study has not been done.
• Severe immunosuppression: Biologicals with concomitant steroids (>30 mg/day).
• Central Line.
• Pressor or ventilatory support.
• On antibiotics.
• Patients with Crohn's disease found to have complications like an abscess, phlegmon, stricture, small bowel obstruction, perforation, internal or external fistulization or infection.
• Not willing to consent or follow guidelines throughout research trial.
• Screening labs in either donor or recipient reveal problems with performing fecal microbiome transplantation because inclusion requirements are no longer met.
• Physician discretion.
• Participant request.

DONOR EXCLUSION CRITERIA:

• A history of antibiotic treatment during the 3 months preceding donation.
• A history of intrinsic gastrointestinal illnesses.
• A history of autoimmune or atopic illness or modulating therapy.
• A history of chronic pain syndromes, or neurologic or neurodevelopmental disorders.
• Metabolic syndrome or malnutrition or obesity.
• A history of exposure to infectious agents.
• Diarrhea or other symptoms of an intestinal infection within two weeks prior to, or on the day of, stool donation.
• A history of malignant illnesses or ongoing oncologic therapy.
• Weight less than 15 kgs.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years to 22 Years (Child, Adult)
• Accepts Healthy Volunteers: Yes
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02108821
• Other Study ID Numbers: PRO13030081
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Children's Mercy Hospital Kansas City
• Original Responsible Party: Alka Goyal, MD, University of Pittsburgh, Associate Professor of Pediatrics, Medical Director Infusion Center
• Current Study Sponsor: Children's Mercy Hospital Kansas City
• Original Study Sponsor: University of Pittsburgh

Collaborators

• University of Pittsburgh
• Stanford University

Investigators

• Principal Investigator: Alka Goyal, MD; Stanford University

• PRS Account: Children's Mercy Hospital Kansas City
• Verification Date: February 2021