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Comment & Response |

Fecal Microbiota Transplantation for Clostridium difficile Infection—Reply

Ilan Youngster, MD, MMSc1; Elizabeth L. Hohmann, MD2
[+] Author Affiliations
1Division of Infectious Diseases, Boston Children’s Hospital, Boston, Massachusetts
2Division of Infectious Diseases, Massachusetts General Hospital, Boston
JAMA. 2015;313(7):726. doi:10.1001/jama.2014.18619.
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In Reply: Dr Armstrong and colleagues are concerned that we have compromised a phase 1 study design by incorporating a sample size calculation and by using historical data as a statistical comparator. Traditional phase 1 investigations, or first-in-human studies, are used to determine the dose and schedule of an investigational therapeutic while assessing for potential toxic effects.1

We believe that evaluating a new route of administering a commonly used biological treatment like FMT does not fall under the definition of a formal phase 1 study. Moreover, with the exception of agents like anticancer medications, phase 1 studies are usually conducted in healthy volunteers. Therefore, we considered our trial a pilot study, also commonly known as a feasibility study. Although there is significant controversy about the requirements for pilot studies, a common definition used in the literature is “an experimental use of a treatment in a small group of patients to learn if it will be effective and safe on a broad scale.”2,3 Therefore, in addition to safety and feasibility, if possible, such studies should evaluate some notion of efficacy.

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February 17, 2015
Matthew J. Armstrong, MRCP, PhD; Shrikanth Pathmakanthan, FRCP, PhD; Tariq H. Iqbal, FRCP, PhD
1Gastroenterology Department, Queen Elizabeth University Hospital Birmingham, Birmingham, England
JAMA. 2015;313(7):725-726. doi:10.1001/jama.2014.18617.
November 5, 2014
Ilan Youngster, MD, MMSc; George H. Russell, MD, MSc; Christina Pindar, BA; Tomer Ziv-Baran, PhD; Jenny Sauk, MD; Elizabeth L. Hohmann, MD
1Division of Infectious Diseases, Massachusetts General Hospital, Boston2Harvard Medical School, Boston, Massachusetts3Division of Infectious Diseases, Boston Children’s Hospital, Boston, Massachusetts
2Harvard Medical School, Boston, Massachusetts4Department of Gastroenterology and Nutrition, Boston Children’s Hospital, Boston, Massachusetts
1Division of Infectious Diseases, Massachusetts General Hospital, Boston
5Department of Epidemiology and Preventive Medicine, Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
2Harvard Medical School, Boston, Massachusetts6Division of Gastroenterology, Massachusetts General Hospital, Boston
1Division of Infectious Diseases, Massachusetts General Hospital, Boston2Harvard Medical School, Boston, Massachusetts
JAMA. 2014;312(17):1772-1778. doi:10.1001/jama.2014.13875.
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