ClinicaliQ Trial Snapshot
- Study to Learn If Tofersen (BIIB067) Can Delay ALS Symptoms and About Its Safety in Adults With a SOD1 Gene Mutation Who Have Not Yet Developed ALS Symptoms — Active Not Recruiting • Phase III • NCT04856982.
- Trial testing whether tofersen can delay symptom onset in adults with SOD1 gene mutation before ALS develops.
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What This Trial Is Studying
In this study, researchers will learn more about the effects of a drug called tofersen, also known as BIIB067 and QALSODY®, in adults with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene mutation. One of the causes of ALS is a change (or mutation) in the SOD1 gene, which causes abnormal SOD1 protein to be made. This abnormal protein can cause the motor neurons in people with ALS to break down and die. When neurons are damaged, they release a protein called neurofilament light chain, also…