PAR-19-220 - Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01 Clinical Trial Not Allowed)

National Institute of Neurological Disorders and Stroke (NINDS)

National Center for Advancing Translational Sciences (NCATS)

The National Institute of Neurological Disorders and Stroke (NINDS), with the National Center for Advancing Translational Sciences (NCATS) intends to reissue a Funding Opportunity Announcement (FOA) to solicit applications for research on Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (previously PAR-19-220).

Through this FOA, researchers will be invited to submit applications for support of clinical studies that address critical needs for clinical trial readiness in rare neurological and neuromuscular diseases. These studies should result in clinically validated biomarkers and clinical outcome assessment measures appropriate for use in upcoming clinical trials. Through the support of trial readiness studies, NINDS and NCATS expect to enhance the quality and increase the likelihood of success of clinical trials in these rare diseases.

This Notice is being provided to allow potential applicants sufficient time to develop meaningful collaborations and responsive projects. 

The FOA is expected to be published in Spring of 2022 with an expected application due date in Summer 2022.

This FOA will utilize the U01 Research Project Cooperative Agreement activity code. Details of the planned FOA are provided below.

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.

TBD