Dazucorilant in Patients With Amyotrophic Lateral Sclerosis

Study Overview
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Brief Summary
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The purpose of this study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).

Detailed Description
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Eligible ALS patients will be randomized to one of three treatment arms (1:1:1) across the United States and Europe for a 24-week double-blind treatment period. Patients who complete participation (i.e., complete all visits) in the double-blind treatment period will be eligible for participation in a 24-week open-label extension (OLE) study. A daily dose of 300 mg dazucorilant will be used in the 24-week OLE period. Patients that complete the double-blind treatment period and who do not enter the OLE will enter the 24-week follow-up period.

Official Title
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A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of CORT113176 (Dazucorilant) in Patients With Amyotrophic Lateral Sclerosis (DAZALS)

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: Dazucorilant 300 mg
* Drug: Dazucorilant 150 mg
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female patients ≥18 years of age with Sporadic or familial ALS If taking riluzole and/or edaravone, 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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CORT113176 (Dazucorilant) 300 mg<br>300 mg of dazucorilant will be administered once daily. | Drug: Dazucorilant 300 mg<br>* 300 mg of dazucorilant will be administered once daily in 4 softgel capsules of 75 mg dazucorilant/capsule.<br>|
| Experimental: CORT113176 (Dazucorilant) 150 mg<br>150 mg of dazucorilant will be administered once daily. | Drug: Dazucorilant 150 mg<br>* Dazucorilant and placebo will be administered once daily in 4 softgel capsules, 2 capsules with 75 mg dazucorilant/capsule and 2 capsules of placebo equivalent.<br>|
| Placebo Comparator: Placebo (matched to study drug)<br>Placebo will be administered once daily. | Other: Placebo<br>* Placebo will be administered once daily in 4 softgel capsules of placebo equivalent.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline to Week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R) total score. |  | Baseline to Week 24 | 
| Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, AEs by severity, and deaths due to AEs. |  | Baseline to Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline to Week 24 in muscle strength (assessed using hand-held dynamometer). |  | Baseline to Week 24 | 
| Change from Baseline to Week 24 in Percent Slow Vital Capacity |  | Baseline to Week 24 | 
| Change from Baseline to Week 24 in EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) |  | Baseline to Week 24 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ALS, Amyotrophic Lateral Sclerosis