Biogen SOD1 ASO study
Active but not recruiting anymore
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 a hereditary cause of ALS. Research in animals has shown that it is favorable to slow down the formation of the SOD1 protein if ALS is caused by SOD1 mutations. ASO’s (antisense oligonucleotides) are short oligonucleotides, which can inhibit the production of a specific protein by preventing that the genetic code is converted to the corresponding protein. ASO’s against SOD1 were developed and can slow down the formation of SOD1 in experimental animals and the disease progression. These ASOs must be injected directly in the lumbar fluid by means of a lumbar puncture. This study will examine if 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 the disease progression.
Detailed information and the list of in-and exclusion criteria can be found at this link.