Biogen SOD1 ASO study (“A Phase 1, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB067 Administered to Adult Subjects With Amyotrophic Lateral Sclerosis”)

Mutations in superoxide dismutase 1 (SOD1) are an inherited cause of ALS. Studies in experimental animals have shown that it is beneficial to inhibit the formation of the SOD1 protein if ALS is caused by SOD1 mutations. ASOs (antisense oligonucleotides) are short oligonucleotides, which can inhibit the production of a particular protein by preventing the genetic code from being converted into the corresponding protein. ASOs against SOD1 were developed and, in experimental animals, can inhibit SOD1 formation and slow down disease progression. These ASOs must be introduced directly into the lumbar fluid, by lumbar puncture. This study will investigate whether, in patients with ALS and a SOD1 mutation, the formation of the SOD1 protein can be reduced. It is a phase 1 study. This means that the study focuses on the safety and possible side effects of the medication and not yet on the effect on disease progression.

Detailed information and the list of inclusion and exclusion criteria can be found at this link.