The FY20 DMDRP Translational Research Partnership Award mechanism supports collaborative research partnerships between investigators that will accelerate the movement of promising ideas in DMD into clinical applications. This award supports the development of translational research collaborations between two independent investigators to address a critical problem or question in DMD in a manner that would be less readily achievable through separate efforts. Partnerships where one partner in the collaboration is a research scientist and the other a clinician are strongly encouraged. It should be clear that both have had equal intellectual input into the design of the research project. Multi-institutional partnerships are encouraged but not required. At least one member of the partnership must have expertise either in DMD research or DMD patient care. A proposed project in which a clinical partner merely supplies tissue samples or access to patients will not meet the intent of this award mechanism.
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 life-span, 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.
The success of the project must be supported by the unique skills and contributions of each partner. The proposed study must include clearly stated plans for interactions between the Principal Investigators (PIs) and institutions involved. The plans must include communication, coordination of research progress and results, and data transfer. Additionally, multi-institutional applications must provide an intellectual property plan to resolve potential intellectual and material property issues and to remove institutional barriers that might interfere with achieving high levels of cooperation to ensure the successful completion of this award.
The Translational Research Partnership Award requires 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 other PI will be identified as a 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 FY20 DMDRP Translational Research Partnership Award (TRPA) 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: ○ Cognitive function ○ Cardiac ○ Endocrinology ○ Gastrointestinal issues ○ Immunology ○ Orthopaedics ○ Psychosocial issues ○ Pulmonology (including sleep-focused studies) ○ Skeletal muscle
• Assessment of clinical trial tools and outcome measures, such as: ○ Discovery and qualification of pharmacodynamic, prognostic, and predictive biomarkers ○ Novel clinical outcome assessment ○ Patient-centered outcomes (e.g., quality of life, activities of daily living) ○ Potential surrogate markers ○ 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)
• PIs must be at or above the level of Assistant Professor (or equivalent).
• At least one PI must have expertise either in DMD research or DMD patient care.
The anticipated direct costs budgeted for the entire period of performance for an FY20 DMDRP Translational Research Partnership] Award will not exceed $1.2M. Refer to Section II.D.5, Funding Restrictions, for detailed funding information.
