The purpose of this 2-part study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).
In Part 1, 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 who complete the double-blind treatment period and who do not enter the OLE will enter the 132-week follow-up period. In Part 2, eligible ALS patients will receive open-label treatment to evaluate dose titration and tolerability of dazucorilant. The dose titration will begin with an initial 75 mg once daily dose, and the dose will be titrated up as tolerated in 75 mg increments until the 300 mg once daily target dose is reached and maintained for 3 weeks. Patients who complete participation in the dose-titration treatment period will be eligible to continue treatment with dazucorilant 300 mg once daily in a 52-week open-label extension portion of the study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
279
300 mg of dazucorilant will be administered once daily in 4 capsules of 75 mg dazucorilant/capsule.
Dazucorilant and placebo will be administered once daily in 4 capsules, 2 capsules with 75 mg dazucorilant/capsule, and 2 capsules of placebo equivalent.
Placebo will be administered once daily in capsules of placebo equivalent.
062
Phoenix, Arizona, United States
Change from Baseline to Week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R) total score.
This outcome measure is assessed in study Part 1.
Time frame: Baseline to Week 24
Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, AEs by severity, and deaths due to AEs
This outcome measure is assessed in study Part 1.
Time frame: Baseline to Week 24
Incidence of treatment-emergent AEs and SAEs
This outcome measure is assessed in study Part 2.
Time frame: Baseline up to Week 12
Incidence of treatment-emergent AEs leading to dose interruptions, dose reductions, and/or discontinuations of study drug
This outcome measure is assessed in study Part 2.
Time frame: Baseline up to Week 12
Change from Baseline to Week 24 in muscle strength (assessed using hand-held dynamometer)
This outcome measure is assessed in study Part 1.
Time frame: Baseline to Week 24
Change from Baseline to Week 24 in Percent Slow Vital Capacity
This outcome measure is assessed in study Part 1.
Time frame: Baseline to Week 24
Change from Baseline to Week 24 in EuroQol-5 Dimensions-5 Levels (EQ-5D-5L)
This outcome measure is assessed in study Part 1.
Time frame: Baseline to Week 24
Time to Death
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Dazucorilant will be administered once daily in 75-mg capsules.
278
San Francisco, California, United States
287
Neptune City, New Jersey, United States
353
New York, New York, United States
108
Leuven, Belgium
425
Hamilton, Ontario, Canada
273
Montreal, Quebec, Canada
422
Bron, France
258
Lille, France
257
Limoges, France
...and 25 more locations
This outcome measure is assessed in study Part 1.
Time frame: From randomization date to the date of death from any cause up to 156 weeks