Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases (R01) Clinical Trials Required
Funds clinical trials for new treatments or labeling changes for rare neurodegenerative diseases in adults and children to increase approved therapies.
This grant is no longer accepting proposals
Food and Drug Administration has archived this opportunity.
Funder: Food and Drug Administration
Due Dates: September 21, 2025 (Letter of Intent) | October 21, 2025 (Application)
Funding Amounts: Up to $650,000 total costs/year (direct + indirect) for up to 4 years; up to $900,000/year for innovative trial designs with justification.
Summary: Supports clinical trials of products for new indications or labeling changes to address unmet needs in rare neurodegenerative diseases in adults and children.
Key Information: Only clinical trials are eligible; foreign and domestic applicants may apply; strict page limits and FDA-specific review criteria apply.
Description
This opportunity, offered by the FDA Office of Orphan Products Development (OOPD), funds clinical trials evaluating the efficacy and/or safety of products intended to address unmet needs in rare neurodegenerative diseases for both children and adults. The goal is to increase the number of approved treatments and have a broad, positive impact on rare disease drug development. Projects may support new indications or changes in labeling for drugs, biologics, medical devices, or medical foods.
The program encourages collaborative, efficient, and innovative clinical trial designs, including adaptive, basket, umbrella, and platform trials, as well as the use of data modeling and simulations. Early and ongoing patient engagement in trial design is highly encouraged.
