Guidance on preparing an investigational new drug application for fecal microbiota transplantation studies

Colleen R. Kelly, Sachin S. Kunde, Alexander Khoruts

Research output: Contribution to journalArticlepeer-review

43 Scopus citations


Fecal microbiota transplantation (FMT) is an effective treatment for Clostridium difficile infections that are refractory to antibiotic therapy. Because of the important roles of the microbiota in the function of the gastrointestinal tract and other aspects of human physiology, there is a growing interest in studying FMT for other clinical indications. The US Food and Drug Administration regulates clinical studies to evaluate the safety and efficacy of FMT. Studies of FMT for recurrent Clostridium difficile infection or other indications could require submission of an investigational new drug application. Most academic physicians and investigators do not have the regulatory experience necessary to undertake this process. We provide guidance to researchers on the preparation and submission of investigational new drug applications to study FMT.

Original languageEnglish (US)
Pages (from-to)283-288
Number of pages6
JournalClinical Gastroenterology and Hepatology
Issue number2
StatePublished - Feb 2014

Bibliographical note

Funding Information:
Conflicts of interest This author discloses the following: Alexander Khoruts has received funding from CIPAC LLC to carry out research on FMT using frozen fecal microbiota and also provided consulting services for CIPAC; conflicts of interest are being managed by the University of Minnesota Conflicts of Interest Program. The remaining authors disclose no conflicts.

Funding Information:
Funding Supported by National Institutes of Health 1R21DK093839-01A1 and R21 AI091907 .


  • CBER
  • FDA


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