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

<p>The National Institute of Neurological Disorders and Stroke (NINDS) is interested in supporting the conduct of scientific research utilizing data from intermediate-size patient population expanded access protocols for ALS ("intermediate expanded access protocol for ALS", see&nbsp;<a href="https://www.fda.gov/news-events/expanded-access/expanded-access-categories-drugs-including-biologics">Expanded Access Categories for Drugs (Including Biologics)</a>). Eligible intermediate expanded access protocols for ALS should: 1) provide access to an investigational drug or biological product for people with ALS, and 2) generate data to support research on the prevention, diagnosis, mitigation, treatment, or cure of ALS. The scientific research goal(s) informed by these data could include the investigational drug's or biological product's effect on biomarkers related to the pathophysiology of ALS or target engagement, the collection of safety or outcome information, such as survival or other significant medical events (e.g., hospitalization or need for ventilatory support), or patient experience data that are not specifically tied to assessing efficacy.&nbsp;</p><p>Applications are not being solicited at this time. Notice is being provided to allow potential applicants sufficient time to develop responsive projects.</p>