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 (Application)
Funding Amounts: $40 million estimated total program funding; 4 expected awards; project periods up to 4 years
Summary: Supports research using expanded-access data from investigational ALS therapies for patients ineligible for ongoing clinical trials.
Key Information: Applicants must be clinical trial sites participating in a pivotal phase 2/3 or phase 3 ALS trial supported by a U.S.-based small-business sponsor.
Description
This NIH U01 cooperative agreement supports scientific research using data from intermediate-size expanded-access (EA) protocols for investigational ALS drugs or biological products. Administered by the National Institute of Neurological Disorders and Stroke (NINDS), with participation from the NIH Office of the Director, it targets people living with ALS who are not eligible for ongoing clinical trials.
Projects must both provide investigational treatment access and generate data advancing ALS research. Research may examine biomarkers, target engagement, safety, survival, significant medical events, or patient experiences not specifically tied to assessing efficacy. Expanded access must not interfere with trials supporting regulatory approval or otherwise compromise product development. New applications, renewals of currently funded EA protocols, and resubmissions are allowed.
