Long-Term Safety Study of Tofersen Qalsody in Chinese Adults with SOD1 Mutation Amyotrophic Lateral Sclerosis ALS

What this Trial Is About

Researchers are studying the long-term safety of the drug tofersen, also known as Qalsody, in adults with amyotrophic lateral sclerosis ALS caused by a mutation in the superoxide dismutase 1 SOD1 gene. This postmarketing study aims to collect safety information in Chinese participants since tofersen was approved there in September 2024. The main focus is on counting how many participants experience adverse events or serious adverse events during the study. Researchers will also examine how the drug is processed in the body and how much is present in the cerebrospinal fluid surrounding the brain and spinal cord. Participants will receive up to 13 doses of tofersen over about 44 weeks. The first three doses, called loading doses, are 100 milligrams each and given about two weeks apart through injections into the fluid around the spine intrathecal injection. Then, participants receive 10 more maintenance doses approximately every 28 days by the same method. This study includes a screening period of up to 4 weeks before treatment begins. During the study, participants will visit the research center up to 15 times over a total duration of about one year. Researchers will monitor safety by tracking any health problems or serious issues that occur. They will also perform tests to measure the drug levels in the blood and cerebrospinal fluid. The study will help better understand tofersens safety and how it behaves in the body over time in people with SOD1-ALS.

A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)