Analysis of Amyotrophic Lateral Sclerosis (ALS) Clinical Trials Posted on www.clinicaltrials.gov from 1999-2022.
Aaron Zelikovich1, Eric Anderson2, Michael Rubin1, Jinsy Andrews3
1NewYork-Presbyterian, Weill Cornell Medicine, Department of Neurology, 2Synapticure, 3The Neurological Institute, Columbia University Irving Medical Center, Department of Neurology
Objective:
To create a database of Amyotrophic Lateral Sclerosis (ALS) clinical trials on
www.clinicaltrials.gov and analyze trends over the past three decades.
Background:
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.
Design/Methods:
“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.
Results:
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.
Conclusions:
Analyzing current and past trends of ALS clinical trials will help guide future trial designs in the hopes to optimize ALS outcomes and trials.