The fiscal year 2025 (FY25) Duchenne Muscular Dystrophy Research Program (DMDRP) Clinical/Translational Research Award (CTRA) mechanism supports advanced translational research that will accelerate the movement of promising ideas in Duchenne muscular dystrophy (DMD) research into clinical applications. Research must address at least one of the FY25 CTRA focus areas. Research projects investigating therapies that will be efficacious across the life span, including infants, toddlers, and nonambulatory individuals, are strongly encouraged.
Applications must include preliminary and/or published data relevant to DMD to support the proposed research project. Pilot, proof-of-principle clinical trials, and correlative studies to better inform development of drugs, devices, and other interventions are allowed. The CTRA offers two funding levels: Funding Level 1 to support smaller, less complex preclinical and/or clinical research; and Funding Level 2 to support larger, more complex preclinical and/or clinical research.
Early-Career Partnering PI Option (PPIO): 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 nonclinical aspects of DMD research.
Deadlines:
- Required Pre-Application Submission Deadline: July 25, 2025
- Application Submission Deadline: August 8, 2025
To meet the intent of the funding opportunity, all applications for the FY25 DMDRP CTRA must address at least one of the following focus areas:
Preclinical Translational Research
• Extension or expansion of existing preclinical data in support of Investigational New Drug (IND) application-enabling studies. For example:
○ Optimizing delivery to target tissues.
○ Drug exposure.
○ Independent replication.
○ Comparative studies.
○ Assay development, outcome measures, and/or biomarkers (e.g., pharmacodynamic, prognostic, or predictive biomarkers, including potential surrogate markers).
Clinical Research
• Clinical studies designed to improve care and quality of life.
• Prospective/real world data/post market studies for combination or sequential therapies, and/or long-term safety and efficacy studies.
• Assessment of clinical trial tools and outcome measures:
○ Studies in understudied systems (e.g., cognitive, cardiac, or gastrointestinal [GI]) or age ranges (e.g., infants, toddlers, and/or nonambulatory)
o Discovery and qualification of pharmacodynamic, prognostic, and predictive biomarkers, including potential surrogate markers.
o Novel clinical outcome assessments.
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.
• Natural history studies in understudied systems (e.g., cognitive, cardiac, GI) or age ranges (e.g., infants, toddlers, nonambulatory adults) with an aim toward clinical trial readiness.
Independent investigators at all career levels may be named as Principal Investigator (PI) or Initiating PI on the application. For titles outside of academia that may not be analogous to traditional hierarchies, investigators at or above an independent scientist level may be named by their organization as the 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 nonclinical aspects of DMD research.
Individuals affiliated with an eligible organization are eligible to be named as PI regardless of ethnicity, nationality, or citizenship status.
The Congressionally Directed Medical Research Programs (CDMRP) expects to allot approximately $8.56 million (M) to fund approximately 5 CTRA applications with direct cost caps of $0.65M for Funding Level 1; $0.75M for Funding Level 1 with a PPIO; $1.25M for Funding Level 2; and $1.35M for Funding Level 2 with a PPIO. The maximum period of performance is 3 years for Funding Level 1. The maximum period of performance for Funding Level 2 is 4 years. It is anticipated that awards made from this FY25 funding opportunity will be funded with FY25 funds, which will expire for use on September 30, 2031. Awards supported with FY25 funds will be made no later than September 30, 2026.