A Study of BIIB067 When Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
Status:
Recruiting
Trial end date:
2027-08-07
Target enrollment:
Participant gender:
Summary
The primary objective of this study is to evaluate the efficacy of BIIB067 when initiated in
presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated
neurofilament (NF). The secondary objectives of this study are to evaluate the safety and
tolerability of BIIB067 and to evaluate the effect of BIIB067 on pharmacodynamics
(PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of
clinically manifest amyotrophic lateral sclerosis (ALS).