The FY23 DMDRP TRA mechanism supports advanced 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 proof of concept studies 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, including infants, toddlers, and non-ambulatory individuals, are strongly encouraged. Pilot, proof-of-principle clinical trials, and correlative studies to better inform development of drugs, devices, and other interventions are allowed.
Early-Career Partnering PI Option: The FY23 DMDRP encourages applications that include meaningful and productive collaborations between investigators. In an effort to promote enhanced research capacity within the DMD field, the FY23 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 FY23 DMDRP Translational Research Award (TRA) must address at least one of the following Focus Areas:
• Investigational New Drug (IND)-enabling or clinical studies designed to improve care and quality of life •
Assessment of clinical trial tools and outcome measures with a focus on understudied systems (e.g., cognitive, cardiac, and/or gastrointestinal [GI]) or age ranges (e.g., infants, toddlers, and/or non-ambulatory). For example: 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
• Extension or expansion of existing preclinical data in support of a pre-IND regulatory submission, such as optimizing delivery to target tissues, including drug exposure, independent replication, and comparative studies
• Natural history studies in understudied systems (e.g., cognitive, cardiac, and/or GI) or age ranges (e.g., infants, toddlers, and/or non-ambulatory) with an aim toward clinical trial readiness
Independent investigators at all academic levels may be named as PI or Initiating PI on the application.
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 FY23 DMDRP TRA should not exceed $1.25M for a single PI or $1.35M for the Early-Career Partnering PI Option. Refer to Section II.D.5, Funding Restrictions, for detailed funding information.
