NIH/NIDDK – Pilot and Feasibility Clinical and Translational Research Studies in Digestive Diseases and Nutrition (R21 Clinical Trial Optional)

August 20, 2018 by School of Medicine Webmaster

The goal of this pilot and feasibility mechanism is to provide flexibility for initiating preliminary, short-term clinical studies that will assess feasibility through the identification and assessment of a component or components of protocol operational challenges; or clinical translational studies that are novel and exploratory in nature, thus allowing new ideas to be investigated in a more expeditious manner without stringent requirements for preliminary data.  Such support is needed to encourage experienced investigators as well as new investigators to pursue new clinical or laboratory approaches, novel or underdeveloped topics, or more creative avenues of research.  If successful, these awards should lead to significant scientific understanding of digestive and liver diseases and nutrition research; or for clinical studies, improved understanding of clinical study operational and feasibility issues in order to better execute challenging clinical protocols efficiently.  These successes should facilitate translation into improved understanding of disease pathophysiologic mechanisms or into clinical practice to improve patient outcomes.  This FOA will permit both clinical and translational pilot and feasibility studies that must involve human subjects research.  The research proposed to this FOA must involve direct clinical studies in humans or studies on identifiable specimens from human subjects such as tissues or other biospecimens.

Potential clinical pilot and feasibility studies for this funding opportunity should be on diseases that ordinarily have little research support either because they are uncommon or rare, or difficult to manage, or are not the focus of pharmacological therapy; and the conduct of the proposed study has an identifiable protocol operational challenge which if overcame, would lend to more efficient conduct and improve the feasibility of success of a subsequent more robust and definitive clinical study.  For instance, there are several digestive diseases that may have lasting and major consequences for the patients who have these conditions. These conditions are particularly challenging when they occur in children and have life-long consequences.  The study of these diseases is often challenged by patient and parent acceptability of the particular intervention, of the necessary duration of the study timeline, or other recruitment or retention issues as examples.  Other protocol operational challenges that may be unique to the specific disease or intervention may also be identified by the Principal Investigator whereby a pilot and feasibility study would assist in determining the capability of entertaining the pursuit of a larger trial in the future.  At the conclusion of completing a clinical pilot and feasibility study, the PI should have the necessary data at hand that would determine the ability to either pursue and efficiently undertake, initiate, and complete a larger study; or the need to significantly re-evaluate the protocol conduct strategy.  For the purposes of this FOA, the NIDDK limits multi-centered pilot and feasibility clinical studies or trials to just 2 clinical sites.

Potential clinical translational science pilot and feasibility studies should further understanding of pathophysiologic mechanisms that underpin the development of digestive and hepatic diseases and nutritional research that will shed light on furthering our understanding of the disease process or physiology and inform approaches to strategically envision rational therapeutic approaches.  Innovative therapeutic approaches targeting key pathophysiologic mechanisms may advance the understanding of the digestive disease process and may pioneer the development of future diagnostics or therapeutics.  Several liver and digestive diseases and nutritional research challenges would benefit from further delineation and advancement by more precisely defining the pathophysiologic mechanisms that lead to end-organ injury.  Thus, translational sciences studies may yield improved understanding of pathophysiologic mechanisms, candidate biomarkers, improved delineation of candidate gene functions in disease states, as examples of outcomes, but because of the pilot and feasibility nature of this FOA, are clearly not limited to just these examples.  Use of identifiable human specimens or tissues must be used in translational sciences pilot and feasibility studies.  Translational research studies that propose to utilize de-identified human specimens will be considered non-responsive to this FOA.

Objectives and Scope

This funding opportunity is intended to encourage clinical pilot and feasibility; and laboratory based translational research relevant to the mission of the Division of Digestive Diseases and Nutrition, NIDDK without stringent requirements for preliminary data.  Applications submitted to this FOA should propose pilot and feasibility research studies that will facilitate conduct of a larger clinical study by assessing a clinical research protocol feasibility issue(s); or the translation of promising and relevant new scientific developments into the clinical setting or that will advance our understanding of pathophysiological mechanism that may become the basis for diagnostic or therapeutic development. The emphasis is thus, on the development of small, randomized clinical trials with identified protocol feasibility issue(s) that would be protocol operational hurdles in the conduct of a larger clinical study or trial; and on laboratory based translational feasibility studies that involve human subjects biospecimens relevant to diseases within the research mission of the Division of Digestive Diseases and Nutrition, NIDDK. Translational science projects are required to involve human subjects research.  The following studies will not be supported by this FOA: those that focus on obesity (please refer to the Office of Extramural Research Grants and Funding to search for multiple obesity related Funding Opportunity Announcements at; and translational studies that utilize animal models or de-identified human specimens.

Applications for R21 awards should describe projects that are clinical pilot and feasibility or translational in nature that are distinct from those supported through the traditional R01 mechanism. For example, long-term projects, or projects designed to increase knowledge in a well-established area, will not be considered for R21 awards. Applications submitted under this mechanism should be exploratory and novel.

Deadlines:  standard dates and standard AIDS dates apply


Filed Under: Funding Opportunities