Study of AROSOD-1 in Adult Participants With Amyotrophic Lateral Sclerosis (ALS)
Status:
Withdrawn
Trial end date:
2025-03-01
Target enrollment:
Participant gender:
Summary
In this Phase 1 adult symptomatic patients with amyotrophic lateral sclerosis (ALS) carrying
a superoxide dismutase 1 (SOD1) gene mutation thought to be causative of ALS, will receive
single ascending doses of ARO-SOD1 administered by intrathecal (IT) infusion. The study is
primarily intended to evaluate safety, but will also evaluate the effect of ARO-SOD1 on SOD1
cerebrospinal fluid (CSF) levels as a biomarker of pharmacodynamic (PD) effect, therefore
lumbar punctures will be required at timepoints throughout the study. After each participant
has completed their individual final visit, participants whose SOD1 CSF levels have recovered
to a satisfactory level may rescreen and enroll into higher dose cohorts; or if unable or
unwilling to rescreen may enroll into an open-label study to be added by amendment when
supported by nonclinical data for multidose administration.