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Updated: Aug 9, 2026
Fecal Microbiota Transplantation via Colonoscopy for Recurrent C. difficile Infection
Published on: December 8, 2014
Protocol for a randomised, placebo-controlled pilot study for assessing feasibility and efficacy of faecal microbiota
Nikhil Pai1, Jelena Popov1,2
1Division of Pediatric Gastroenterology & Nutrition, Department of Pediatrics, McMaster University, Hamilton, Canada.
Insights
This study investigates fecal microbiota transplantation (FMT) for pediatric ulcerative colitis (UC). It assesses the feasibility and patient outcomes of FMT in children with UC, offering a potential new therapy.
Area of Science:
- Gastroenterology
- Microbiology
- Pediatric Inflammatory Bowel Disease
Background:
- Ulcerative colitis (UC) is a chronic inflammatory condition with increasing prevalence in children.
- Current treatments for pediatric UC can have side effects, necessitating novel therapeutic approaches.
- Fecal microbiota transplantation (FMT) shows promise as a potential treatment for inflammatory conditions.
Purpose of the Study:
- To evaluate the feasibility and patient outcomes of FMT in pediatric patients with ulcerative colitis.
- To establish safety and efficacy parameters for FMT in this population.
- To explore FMT as a novel therapeutic option for pediatric IBD.
Main Methods:
- A phase Ib, randomized, single-blinded, placebo-controlled trial involving 50 pediatric patients.
- Patients receive either normal saline control or active FMT via enema twice weekly for 6 weeks.
- Feasibility assessed by eligibility, recruitment, participation, sample collection, hospitalizations, and dropout rates. Efficacy measured by clinical disease scores (PUCAI) and inflammatory markers.
Main Results:
- Data on feasibility outcomes including patient eligibility, recruitment, and dropout rates were collected.
- Clinical disease activity was monitored using the Pediatric Ulcerative Colitis Activity Index (PUCAI).
- Inflammatory markers and microbiome composition were analyzed from collected samples.
Conclusions:
- The study provides initial data on the feasibility of conducting FMT trials in pediatric UC.
- Findings will inform future, larger-scale randomized controlled trials for FMT in pediatric IBD.
- FMT represents a potential new treatment avenue for children with ulcerative colitis.
Introduction:
Ulcerative colitis (UC) is a chronic, relapsing condition characterised by colonic inflammation. Increasing prevalence in early-age diagnosis provides opportunities for additional complications in later life as a result of prolonged exposure to inflammatory and therapeutic insults, necessitating novel avenues for therapeutics which may result in fewer side effects. Faecal microbiota transplantation (FMT) has previously demonstrated potential therapeutic benefit in an adult randomised-controlled trial and several recurrent Clostridium difficile infection studies. This phase Ib pilot will be the first randomised, single-blinded, placebo-controlled trial to assess feasibility and patient outcomes in a paediatric inflammatory bowel disease (IBD) population.
Methods And Analysis:
Fifty patients will be randomised 1:1 to receive normal saline control or active sample. Enema administrations will be performed two times per week for 6 weeks, followed at a 6-month follow-up period. Feasibility outcomes will include measures of patient eligibility, recruitment, willingness to participate, samples collections, hospitalizations and drop-out rate. Improvements in disease symptoms will determine the efficacy of treatment. Clinical disease scores will be taken throughout the study period using the Paediatric Ulcerative Colitis Activity Index (PUCAI). Monitoring of inflammatory markers in blood and stool will be performed at regular intervals. Microbiome analysis will be conducted on stool samples collected throughout the trials period. Imaging and endoscopic surveillance will be conducted if clinically necessary.
Ethics And Dissemination:
Ethics was obtained from local hospital research ethics boards across all three sites. Health Canada and FDA approval was obtained for the use of an Investigatory New Drug product. Results from this trial will be presented in international conferences and published in peer-review journals.
Trial Registration Number:
Trial registration number: NCT02487238; preresults.
