The FY22 DMDRP TRA mechanism supports translational research that will accelerate the movement of promising ideas in DMD research into clinical applications. Translational research may be defined as an integration of basic science and clinical observations. Observations that drive a research idea may originate from a laboratory discovery, population-based studies, or a clinician’s firsthand knowledge of patient care. The ultimate goal of translational research is to move a concept or observation forward into clinical application. However, Principal Investigators (PIs) should not view translational research as a one-way continuum from bench to bedside. The research plan must involve a reciprocal flow of ideas and information between basic and clinical science.
This mechanism is intended to support established projects that have moved beyond the realm of basic research and have the potential to result in a near-term impact in clinical research or the clinic. Research projects investigating therapies that will be efficacious across the lifespan, particularly in adolescents and adults, are strongly encouraged. Pilot, proof-of-principle clinical trials and correlative studies to better inform development of drugs, devices, and other interventions are allowed.
All applications must include preliminary data relevant to DMD and the proposed project. The proposed research must be relevant to active-duty Service Members, Veterans, military beneficiaries, and/or the American public.
Early-Career Partnering PI Option: The FY22 DMDRP encourages applications that include meaningful and productive collaborations between investigators. In an effort to promote enhanced research capacity within the DMD field, the FY22 TRA includes an option for an Early-Career Partnering PI. The Partnering PI Option is structured to accommodate two PIs. One PI will be identified as the Initiating PI and will be responsible for the majority of the administrative tasks associated with application submission. The Early-Career PI will be identified as the Partnering PI. Both PIs should contribute significantly to the development of the proposed research project, including the Project Narrative, Statement of Work (SOW), and other required components. If recommended for funding, each PI will be named to an individual award within the recipient organization. For individual submission requirements for the Initiating and Partnering PI, refer to Section II.D.2, Content and Form of the Application Submission.
Deadlines:
All applications for the FY22 DMDRP Translational Research Award (TRA) must address at least one of the following Focus Areas:
• Translational and clinical studies, novel interventions, and drug and biologic delivery technologies designed to improve care and quality of life in areas such as: o Cognitive function o Cardiac o Endocrinology (including bone health and growth) o Skeletal muscle
• Assessment of clinical trial tools and outcome measures such as: o Discovery and qualification of pharmacodynamic, prognostic, and predictive biomarkers, including potential surrogate markers o Novel clinical outcome assessment o Patient-centered outcomes (e.g., quality of life, activities of daily living) o Secondary data analysis that helps to address clinical research tool validation and/or to understand natural history
• Extension or expansion of existing preclinical translational data in support of a specific therapeutic development path (such as optimizing delivery to target tissues, including drug exposure, independent replication, and comparative studies)
Independent investigators at all academic levels may be named as PI on the application.
An eligible PI, regardless of ethnicity, nationality, or citizenship status, must be employed by, or affiliated with, an eligible organization.
Early-Career Partnering PI Option:
• The Partnering PI must be an independent, early-career investigator within 10 years of their first faculty appointment (or equivalent) by the time of application submission. Lapses in research time or appointments as denoted in the biographical sketch should be explained in the application.
• It is encouraged, but not required, that the partnering PI is an M.D. or M.D./Ph.D. to increase collaboration between clinical and non-clinical aspects of DMD research.
The anticipated direct costs budgeted for the entire period of performance for an FY22 DMDRP Translational Research Award will not exceed $1.25M or $1.35M for the Early-Career Partnering PI Option. Refer to Section II.D.5, Funding Restrictions, for detailed funding information
