The purpose of this study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).
Eligible ALS patients will be randomized to one of three treatment arms (1:1:1) across North America and Europe for a 24-week double-blind treatment period. Patients who complete participation (i.e., completed all visits) in the double-blind treatment period will be eligible for participation in a 132-week open-label extension (OLE) study. A daily dose of 300 mg dazucorilant will be used in the 132-week OLE period. Patients that complete the double-blind treatment period and who do not enter the OLE will enter the 132-week follow-up period.
Inclusion Criteria:
- Male and female patients ≥18 years of age with Sporadic or familial ALS
- If taking riluzole, edaravone, and/or sodium phenylbutyrate and taurursodiol, must
be on a stable dose prior to Screening.
Exclusion Criteria:
- History of a clinically significant non-ALS neurologic disorder
- Inability to swallow capsules.
- Human immunodeficiency virus (HIV) or current chronic/active infection with
hepatitis C virus or hepatitis B virus
- Women who are pregnant, planning to become pregnant, or are breastfeeding.
- Use of non-invasive ventilation (NIV) or mechanical ventilation via tracheostomy, or
on any form of oxygen supplementation.
- Current or anticipated need of a diaphragm pacing system (DPS).
- Currently using glucocorticoids or have a history of regular systemic glucocorticoid
use within the last 12 months.
- Previous exposure or treatment with glucocorticoid receptor modulators or
antagonists.