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

The NINDS and Office of the Director, NIH intend to publish a Notice of Funding Opportunity (NOFO) to solicit applications for research on the conduct of scientific research utilizing data from expanded access (EA) for investigational drugs or biological products as described in section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb). These applications will target intermediate size populations of patients living with amyotrophic lateral sclerosis (ALS) who are not eligible for ongoing clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of ALS ("intermediate EA protocol for ALS").

ALS is a rapidly progressive, ultimately fatal, neurodegenerative disease causing weakness and wasting of skeletal muscles including the diaphragm. There are over 32,000 estimated cases in the United States (U.S.) equivalent to a prevalence of 9.9 per 100,000 U.S. population. While considerable variability in presentation and progression exists, mean survival from symptom onset is only 3-5 years, and treatment options for ALS remain severely limited. Existing disease-modifying drugs for ALS have only modest effects on slowing disease progression, and no known treatment prevents, halts or reverses ALS progression. Thus, development of new effective treatments to prevent disease onset, make ALS a livable disease, or cure ALS is a pressing need.

To address this challenge, individuals with ALS may enroll in ongoing phase 3/efficacy clinical trials of investigational drugs and biological products for ALS. However, trial sponsors typically set inclusion criteria that restrict participation to only a subset of people with ALS to increase the likelihood of detecting efficacy with feasible sample size and trial duration. Thus, a potentially large segment of people with ALS may be ineligible to participate in clinical trials because they do not meet these inclusion criteria.

EA was established to provide treatment access to investigational medical...