Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01)
The purpose of this funding opportunity announcement (FOA) is to support clinical studies that will fill gaps in the design of upcoming clinical trials in rare neurological or neuromuscular diseases by validating clinical outcome measures or biomarkers, or by characterizing cohorts of relevant patients. Through the support of trial readiness studies, NINDS expects to accelerate the initiation of clinical trials for rare diseases and to increase the likelihood of success in those trials.
This FOA is intended to support clinical studies that address current obstacles to the design of upcoming clinical trials in rare neurological and neuromuscular diseases and conditions. The studies supported through this FOA should be clinical observational studies (cross-sectional or longitudinal), and no intervention studies will be supported through this FOA. Appropriate trial readiness projects can be “stand-alone” studies, or they can be ancillary to other, ongoing clinical studies. For example, a project could propose to validate new or improved COA measures or biomarkers by adding them as experimental outcomes in an ongoing trial or longitudinal study that is supported through other funding sources. Higher priority will be given to diseases/conditions that currently or soon will have multiple candidate therapeutics or devices ready for testing in clinical trials, but that lack critical components of trial readiness that are needed for moving forward. For these higher priority diseases, the justification should be made by the applicants that clinical trials could begin immediately after the completion of the trial readiness study to test the candidate therapeutics that will be available at that time. Disorders will be given lower priority if there is not a strong justification for the urgent need for trial readiness, based on the likelihood of available candidate therapeutics by the time the trial readiness study would be completed. Lower priority will also be given to diseases or conditions for which clinical trials are already ongoing, and advances in clinical trial design through more sensitive or reliable biomarkers or COA measures, or better characterization of disease cohorts would not significantly accelerate progress toward effective treatments.
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