Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)

Background
The National Institutes of Health (NIH), specifically the National Institute of Neurological Disorders and Stroke (NINDS), is issuing this Notice of Funding Opportunity (NOFO) to solicit applications for scientific research utilizing data from expanded access (EA) for investigational drugs or biological products. This initiative targets intermediate-sized populations of patients living with amyotrophic lateral sclerosis (ALS) who are not eligible for ongoing clinical trials aimed at the prevention, diagnosis, mitigation, treatment, or cure of ALS.

The goal is to support extramural research that explores fundamental aspects of neurological disorders and develops new methods for treatment and diagnosis.

Grant Details
The NOFO aims to support scientific research that utilizes data from intermediate-size patient population EA protocols for ALS. The proposed research must provide access to investigational drugs or biological products while generating data that supports research on ALS. Specific tasks include:
- Conducting scientific research using EA data to inform understanding of ALS.
- Ensuring that the provision of investigational drugs does not interfere with ongoing clinical trials.
- Collecting safety and outcome information related to ALS treatments.
- Engaging stakeholders, including individuals with lived experiences and advocacy groups, in the study design process.
- Submitting an intermediate-size patient population EA IND application to the FDA by the grant application submission date.
- Documenting IRB approval prior to funding and initiation of participant treatment.

Eligibility Requirements
Eligible applicants include higher education institutions, nonprofits, for-profit organizations, local governments, state governments, tribal governments, and other entities involved in clinical trials. Applicants must be clinical trial sites participating in a phase 2/3 or phase 3 efficacy clinical trial supported by a U.S.-based small business concern designated by the FDA as a sponsor for a drug or biological product related to ALS. Non-domestic entities are not eligible.

Period of Performance
The maximum project period is four years. The funding will continue until one of several specified events occurs, such as marketing authorization by the FDA or withdrawal of the IND.

Grant Value
NINDS intends to commit up to $40 million per fiscal year to fund approximately four new awards annually, contingent upon NIH appropriations and sufficient meritorious applications.

Place of Performance
The grant will be performed at clinical trial sites participating in relevant efficacy trials across the United States.