A Randomized Controlled Trial of Efficacy and Safety of Fecal Microbiota Transplant for Preventing Recurrent Clostridioides difficile Infection

Clin Infect Dis. 2025 Feb 5;80(1):52-60. doi: 10.1093/cid/ciae467.

Abstract

Background: Clostridioides difficile infection (CDI) is the most common cause of healthcare-associated infections in US hospitals, with 15%-30% of patients experiencing recurrence. The aim of our randomized, double-blind clinical trial was to assess the efficacy of capsule-delivered fecal microbiota transplant (FMT) versus placebo in reducing recurrent diarrhea and CDI recurrence. The secondary aim was FMT safety assessment.

Methods: Between 2018 and 2022, Veterans across the Veterans Health Administration system with recurrent CDI who responded to antibiotic treatment were randomized in a 1:1 ratio to oral FMT or placebo capsules. Randomization was stratified by number of prior CDI recurrences (1 or ≥2). The primary endpoint was clinical recurrence by day 56, defined as >3 unformed stools daily for ≥2 days with or without laboratory confirmation of C. difficile, or death within 56 days.

Results: The study was stopped due to futility after meeting prespecified criteria. Of 153 participants (76 FMT, 77 placebo) with an average age of 66.5 years, 25 participants (32.9%) in the FMT arm and 23 (29.9%) in the placebo arm experienced the primary endpoint of diarrhea and possible or definite CDI recurrence or death within 56 days of capsule administration (absolute difference, 3.0% [95% confidence interval, -11.7% to 17.7%]). Stratification by number of recurrences revealed no statistically significant differences. There were no clinically important differences in adverse events.

Conclusions: FMT therapy versus placebo did not reduce CDI recurrence or death at 56 days. There were no meaningful differences in adverse events between treatment groups.

Clinical trials registration: NCT03005379.

Keywords: Clostridioides difficile; diarrhea; fecal microbiota transplantation; placebos; randomized controlled trial.

Publication types

  • Randomized Controlled Trial
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Aged
  • Clostridioides difficile
  • Clostridium Infections* / prevention & control
  • Clostridium Infections* / therapy
  • Diarrhea / microbiology
  • Diarrhea / therapy
  • Double-Blind Method
  • Fecal Microbiota Transplantation* / adverse effects
  • Fecal Microbiota Transplantation* / methods
  • Feces / microbiology
  • Female
  • Humans
  • Male
  • Middle Aged
  • Recurrence
  • Secondary Prevention
  • Treatment Outcome

Associated data

  • ClinicalTrials.gov/NCT03005379