Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)
This grant will fund research using data from expanded access to investigational drugs for ALS patients who are ineligible for clinical trials, aiming to improve prevention, diagnosis, or treatment of ALS.
Funder: National Institutes of Health
Due Dates: November 10, 2026
Funding Amounts: Estimated total program funding: $40,000,000; ~4 awards expected; award size not specified
Summary: Supports research using expanded access data for investigational drugs/biologics in intermediate-size ALS patient populations not eligible for ongoing clinical trials.
Key Information: Applicants must be U.S.-based clinical trial sites in phase 2/3 or 3 efficacy trials supported by a small business FDA sponsor.
Description
This opportunity invites applications for cooperative agreements to conduct scientific research utilizing data from expanded access (EA) programs for investigational drugs or biological products, as outlined in section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb). The focus is on intermediate-size populations of patients living with amyotrophic lateral sclerosis (ALS) who are not eligible for ongoing phase 2/3 or phase 3 clinical trials aimed at the prevention, diagnosis, mitigation, treatment, or cure of ALS. The program aims to generate pivotal data to support marketing applications for ALS therapies.
