Analysis of Amyotrophic Lateral Sclerosis (ALS) Clinical Trials Posted on www.clinicaltrials.gov from 1999–2022. (P10-8.011)

Abstract

<h3>Objective:</h3> To create a database of Amyotrophic Lateral Sclerosis (ALS) clinical trials on www.clinicaltrials.gov and analyze trends over the past three decades. <h3>Background:</h3> ALS is a progressive neurodegenerative disease of the brain and spinal cord neurons with a typical lifespan of 3–5 years from symptom onset. Clinical trials have evolved over the past three decades as more awareness and research funding have been dedicated to understanding and treating ALS, partially due to the ALS Ice Bucket Challenge in 2014. A review of the evolution of study design and parameters over time provides insights into the future of clinical trials and is reflective of improvements in the science. <h3>Design/Methods:</h3> “ALS” and “Amyotrophic Lateral Sclerosis” were searched on www.clinicaltrial.gov in August 2022. A database was created to capture information on trials: year posted, study type, primary endpoints, and inclusion criteria. <h3>Results:</h3> The term ALS had 777 results and Amyotrophic Lateral Sclerosis had 891 results, for a total of 954 unique clinical trials from 1999 to 2022. Two hundred and seventy-three trials were removed due to non-ALS trials (other neurological disorders, drug name with ALS, or advanced life support) and 681 were analyzed. Thirty-four countries were represented with the United States (n=288, 43%), France (n=71, 11%), and Italy (n=28, 4.1%) having the highest number. The largest number of new trials posted in one year was in 2021 with 81. Forty-eight percent of studies are completed, and 146 (21%) are actively recruiting. Length of time to primary endpoint ranged between one day to ten years. 40% of studies did not specify ALS diagnostic criteria for eligibility and 4% required genetic testing. <h3>Conclusions:</h3> Analyzing current and past trends of ALS clinical trials will help guide future trial designs in the hopes to optimize ALS outcomes and trials. <b>Disclosure:</b> Dr. Zelikovich has nothing to disclose. Eric Anderson has nothing to disclose. Dr. Rubin has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for NeuroAlert. Dr. Rubin has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Merck Manual. Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Wave Life Sciences. Dr. Andrews has received personal compensation in the range of $0-$499 for serving as a Consultant for Cytokinetics. Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AL-S Pharma.