Biomarkers that can effectively measure neurodegeneration are much needed and essential to understanding, diagnosing, and ultimately treating ALS. In this observational study, researchers are seeking to identify and measure the levels of specific biomarkers in ALS patients being treated with edaravone (Radicava). The biomarkers of interest are linked to oxidative stress, inflammation, and neuronal and muscle injury. The study will enroll approximately 300 participants who will be followed over a 24-week period. Biological samples (blood and urine) will be collected from study participants for analyses. Participants can also opt in to receive their genetic results for five common mutations associated with ALS, including SOD1, TARDBP, C9ORF72, FUS and VCP.
Antibodies are proteins that are produced by the immune system to protect the body against foreign invaders like bacteria and viruses, and work by binding to specific proteins on the harmful agents and triggering their removal and/or destruction. In this Phase 2 clinical trial, researchers will be testing the safety of a human antibody (called AP-101) designed to target an ALS-linked protein called SOD1. Evidence suggests that the misfolding of SOD1 in cells can cause the protein to take on a toxic gain of function. Researchers are hopeful that targeting this protein may represent a promising strategy for the treatment of ALS. A previous Phase 1 study found the drug to be safe and well-tolerated at the tested doses. In this follow-up study, researchers expect to enroll 63 participants who will be randomly divided to either receive the active drug (AP-101) or placebo intravenously (IV), for 48 weeks. Both individuals with familial, SOD1-ALS and sporadic disease will be eligible to participate. Researchers will monitor participants to ensure that the drug is safe, identify any side effects, determine the appropriate dosage, and learn more about how the body breaks down the drug internally.