Notice of Intent to Publish a Funding Opportunity Announcement for Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01 Clinical Trial Not Allowed)
Notice Number:

Key Dates

Release Date:
February 23, 2022
Estimated Publication Date of Funding Opportunity Announcement:
April 15, 2022
First Estimated Application Due Date:
August 18, 2022
Earliest Estimated Award Date:
April 04, 2023
Earliest Estimated Start Date:
April 04, 2023
Related Announcements

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

Issued by

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.

Research Initiative Details

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.

Funding Information


Estimated Total Funding

$2.25 M total cost

Expected Number of Awards


Estimated Award Ceiling

$750,000 direct cost

Primary Assistance Listing Number(s)


Anticipated Eligible Organizations
Public/State Controlled Institution of Higher Education
Private Institution of Higher Education
Nonprofit with 501(c)(3) IRS Status (Other than Institution of Higher Education)
Small Business
For-Profit Organization (Other than Small Business)
State Government
Indian/Native American Tribal Government (Federally Recognized)
Indian/Native American Tribally Designated Organization (Native American tribal organizations (other than Federally recognized tribal governments)
U.S. Territory or Possession
Indian/Native American Tribal Government (Other than Federally Recognized)
Non-domestic (non-U.S.) Entity (Foreign Organization)
Regional Organization
Eligible Agencies of the Federal Government

Applications are not being solicited at this time. 


Please direct all inquiries to:

Glen Nuckolls, PhD

National Institute of Neurological Disorders and Stroke (NINDS)