Protocol for a pilot randomised, double-blind, placebo-controlled trial for assessing the feasibility and efficacy of faecal microbiota transplantation in adolescents with refractory irritable bowel syndrome: FAIS Trial

Judith Zeevenhooven, Clara Marieke Andrea de Bruijn, Arine Vlieger, Max Nieuwdorp, Marc Alexander Benninga, Judith Zeevenhooven, Clara Marieke Andrea de Bruijn, Arine Vlieger, Max Nieuwdorp, Marc Alexander Benninga

Abstract

Background: Irritable bowel syndrome (IBS) is a common chronic medical condition, in both children and adults. Despite the availability of effective (non)pharmacological treatments, symptoms persist in a significant amount of patients with IBS. Faecal microbiota transplantation (FMT) may be an effective alternative treatment in adolescents with refractory IBS through manipulation of the intestinal microbiota.

Methods and analysis: This randomised, placebo-controlled single-centre pilot study will assess feasibility and efficacy of FMT in 30 adolescents (16-21 years) with refractory IBS. Patients will be randomly allocated (1:1) to receive two allogeneic (healthy donor) or two autologous (own) faecal infusions at baseline and after 6 weeks. Primary outcomes will assess feasibility, including patient and donor recruitment, adherence and incidence rates of adverse events. To evaluate clinical efficacy, secondary outcomes will include the proportion of patients with at least >50% reduction of their abdominal pain intensity and frequency 12 weeks after the first FMT, and after 6-month and 12-month follow-up. Other outcomes comprise changes in faecal gut microbiota composition, quality of life, depression and anxiety, school or work absenteeism and adequate relief, measured directly after FMTs and after 6 and 12 months of follow-up.

Discussion: This randomised controlled trial will investigate the feasibility and effectiveness of repetitive FMTs in adolescents with refractory IBS.

Ethics and dissemination: The study is approved by the Medical Research Ethics Committees AMC (MEC-AMC) in the Netherlands.

Trial registration number: NCT03074227.

Keywords: gastroenterology.

Conflict of interest statement

Competing interests: MN is in the scientific advisory board of Caelus Health and Kaleido BioSciences; however, none of these are directly related to the current manuscript.

© Author(s) (or their employer(s)) 2020. Re-use permitted under CC BY. Published by BMJ.

Figures

Figure 1
Figure 1
Trial design. After adolescents sign the informed consent form (T-2), patients complete the baseline pain diary, the Irritable Bowel Syndrome Severity Scoring System (IBS-SSS) and deliver stool samples and blood samples for eligibility screening. At T0, adolescents are randomised in the allogeneic or autologous faecal microbiota transplantation (FMT) group.

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