ATLAS: a phase III, double-blind randomised placebo-controlled study with a run-in period and an open-label extension period, to investigate the effect of BIIB067 in presymptomatic adult mutation carriers with confirmed mutation in the Superoxide Dismutase 1 (SOD1) gene. BIIB067 is an antisense oligonucleotide treatment, to be administered monthly via epidural and decreases the expression of the SOD1 protein. It is a promising treatment for ALS patients with an SOD1 mutation.

This phase III study is sponsored by Biogen and aims to determine whether monthly intrathecal administration of BIIB067 can prevent or delay the onset of ALS in SOD1 mutation carriers.

Only specific mutations in the SOD1 gene, associated with a rapidly progressing form of the disease, are eligible. During an initial phase of the study, asymptomatic mutation carriers will be followed with monthly blood draws to monitor neurofilaments. If an increase in neurofilaments occurs (typically several months before disease onset), study participants will start BIIB067 or placebo. When the disease becomes clinically evident, all participants will be able to be administered BIIB067.

In this way, it will be investigated whether preventive treatment with BIIB067 is effective.

Interested, or would you like further information? Contact us at onderzoek@als.be

More info on this study can be found on the website:
https://clinicaltrials.gov/ct2/show/NCT04856982?term=BIIB067&cond=Amyotrophic+Lateral+Sclerosis&draw=2&rank=2.