Observational Registry Study Evaluating Long-Term Safety of Tofersen in People With SOD1-ALS

What this Trial Is About

Researchers are studying the long-term safety of tofersen (Qalsody®) in people with amyotrophic lateral sclerosis (ALS) who have a mutation in the superoxide dismutase 1 (SOD1) gene. This observational study collects health information without changing participants' medical care. It involves participants from two groups of research centers in Europe and the United States, focusing on understanding participant characteristics and serious side effects over time. Participants' data will be collected through ALS disease registries and networks including the Precision-ALS programme and the ALS/Motor Neuron Disease Natural History Consortium (NHC). The study does not involve new treatments being given by researchers but follows participants receiving tofersen or other care as prescribed by their doctors. The study will continue collecting data for at least seven years or until participants choose to leave or pass away. During the study, researchers will gather information from participants' regular clinic visits, including demographics, medical history, pregnancy status, and serious adverse events like neurological problems. They will also track new health conditions, treatment discontinuation, and pregnancy outcomes. The main outcomes focus on safety events and participant characteristics, with ongoing monitoring to learn more about long-term safety and health during participation.

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