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

Opportunity Information: Apply for PAR 19 220

Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01 Clinical Trial Not Allowed), funding opportunity number PAR 19 220, is a National Institutes of Health (NIH) discretionary grant competition using the cooperative agreement mechanism (U01). It is led by NIH institutes and centers focused on neurological disease and translational science, specifically the National Institute of Neurological Disorders and Stroke (NINDS) and the National Center for Advancing Translational Sciences (NCATS). The central purpose of the program is to fund clinical studies that fill the practical gaps that often prevent rare-disease therapeutic programs from moving smoothly into well-designed, interpretable clinical trials. Rather than paying for an interventional clinical trial itself, this announcement supports the preparatory work needed to make future trials more feasible, rigorous, and likely to succeed.

The FOA targets rare neurological and neuromuscular diseases where trial planning is commonly limited by a lack of validated tools to measure disease status, progression, and treatment response. Funded projects are expected to produce clinically validated biomarkers and clinical outcome assessment (COA) measures that can be used as endpoints or supportive measures in upcoming clinical trials. In practice, this means applicants should design human clinical studies that generate evidence a biomarker is reliable, meaningful, and fit-for-purpose, or that a clinical measure (such as a functional scale, performance outcome, patient-reported outcome, or clinician-reported outcome) is sensitive to change, reproducible across sites and raters, and appropriate for the intended context of use. The overarching goal is to reduce uncertainty in trial design, improve endpoint quality, and increase the odds that later therapeutic trials in these rare conditions yield clear, actionable results.

Because the award mechanism is a U01 cooperative agreement, awardees should expect substantial scientific and programmatic involvement from NIH staff compared with a standard research grant. This typically implies active coordination around milestone-driven progress, harmonization of approaches, and frequent communication to ensure the generated biomarkers or outcome measures are robust and useful to the broader field. The FOA explicitly states "Clinical Trial Not Allowed," which signals that the supported work should not be a prospective interventional efficacy or effectiveness trial of an experimental therapy. The emphasis is on readiness activities that enable trials, such as validating measures, characterizing natural history in a way that supports endpoint selection, and establishing the evidentiary basis for biomarkers or COAs that can credibly be carried into later-stage testing.

The opportunity falls under the NIH health research funding activity category and lists CFDA numbers 93.350 and 93.853. The posted award ceiling is $650,000, and the original closing date shown in the source data is 2022-02-16, with a creation date of 2019-03-13. While the listing does not specify the number of expected awards, it indicates NIH intends to make multiple awards depending on available funds and meritorious applications, which is typical for NIH FOAs of this type.

Eligibility is broad and includes a wide range of domestic and certain non-domestic entities. Eligible applicants include state, county, and local governments; special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized Native American tribal governments; Native American tribal organizations that are not federally recognized; public housing authorities/Indian housing authorities; nonprofits with or without 501(c)(3) status (excluding institutions of higher education in those nonprofit categories); for-profit organizations other than small businesses; and small businesses. The FOA also highlights additional eligible applicant types, including Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISI), Hispanic-serving Institutions, Historically Black Colleges and Universities (HBCUs), Tribally Controlled Colleges and Universities (TCCUs), faith-based or community-based organizations, eligible federal agencies, regional organizations, U.S. territories or possessions, and non-U.S. (foreign) organizations. This breadth is meant to encourage participation from diverse research settings and communities, which is especially important in rare diseases where patient populations are geographically dispersed and recruitment and longitudinal follow-up can require multi-institution collaboration.

In sum, PAR 19 220 is a cooperative-agreement grant opportunity designed to strengthen the foundation for future clinical trials in rare neurological and neuromuscular diseases by funding clinical research that validates biomarkers and outcome measures. The program is positioned as a practical bridge between early discovery and definitive therapeutic testing, aiming to improve endpoint readiness, trial design quality, and interpretability so that subsequent trials have a stronger chance of delivering meaningful answers for rare-disease patients and families.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01 Clinical Trial Not Allowed)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.350, 93.853.
• This funding opportunity was created on 2019-03-13.
• Applicants must submit their applications by 2022-02-16. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Each selected applicant is eligible to receive up to $650,000.00 in funding.
• Eligible applicants include: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public and State controlled institutions of higher education, Native American tribal governments (Federally recognized), Public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), Nonprofits having a 501 (c) (3) status with the IRS, other than institutions of higher education, Nonprofits that do not have a 501 (c) (3) status with the IRS, other than institutions of higher education, Private institutions of higher education, For-profit organizations other than small businesses, Small businesses, Others.

Apply for PAR 19 220

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