|Notice of Intent to Publish a Funding Opportunity Announcement for Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01 Clinical Trial Not Allowed)||
This Notice encourages investigators with expertise and insights into this area of neurological/neuromuscular disease clinical trial readiness to begin to consider applying for this reissued FOA.
Applications to this intended FOA must propose multisite, prospective clinical studies for rare diseases within the NINDS mission aimed at validating clinical outcome assessment measures or scales and may also propose clinical validation of biomarkers. Diseases appropriate for this FOA must be rare diseases that lack critical components of trial readiness and should have candidate therapeutics that will be ready for testing in clinical trials by the time the trial readiness study is completed. The applications should include a section that indicates what clinical trial(s) are being planned in the rare disease studied. The project proposed must be necessary to achieve clinical trial readiness for the upcoming trial(s). Furthermore, when combined with existing clinical research tools and other ongoing research efforts, the proposed studies must be sufficient to fill the needs for validated biomarkers and COA measures for the upcoming trial(s) described in the application. Investigators are encouraged to use or modify existing resources, validate existing tools in specific rare disease populations, or add components to existing disease-specific tools (such as symptom scales). Applications must describe milestones for monitoring progress including necessary steps prior to the initiation of enrollment (e.g., institutional review board and other regulatory approvals, certification of personnel training on the study protocol at each site and approval of the protocol by an external advisory committee) and milestones for enrollment of participants for each year of the study for the categories of sex/gender/race/ethnicity.
Information for Researchers
Funding Opportunities for Muscular Dystrophy Research