Dazucorilant in Patients With Amyotrophic Lateral Sclerosis (DAZALS)

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

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

Study Overview

• Status: Active, not recruiting
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: Dazucorilant 300 mg; Drug: Dazucorilant 150 mg; Other: Placebo

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 249
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Clinical Trial Lead; Phone Number: (650) 327-3270; Email: study652@corcept.com

Study Locations

• Belgium
  • Leuven, Belgium, 3000
    • 108
• Canada
  • Ontario
    • Hamilton, Ontario, Canada, L8N 3Z5
      • 425
  • Quebec
    • Montréal, Quebec, Canada, H3A2B4
      • 273
• France
  • Bron, France, 69500
    • 422
  • Lille, France, 59037
    • 258
  • Limoges, France, 87042
    • 257
  • Marseille, France, 13385
    • 261
  • Montpellier, France, 42395
    • 423
  • Nice, France, 06001
    • 259
  • Paris, France, 75651
    • 262
  • Tours, France, 37000
    • 256
• Germany
  • Berlin, Germany, 13353
    • 255
  • Bonn, Germany, 53127
    • 270
  • Dresden, Germany, 1307
    • 268
  • Hannover, Germany, 30625
    • 260
  • Jena, Germany, 7747
    • 265
  • München, Germany, 81675
    • 386
  • Rostock, Germany, 18147
    • 267
  • Ulm, Germany, 89081
    • 269
• Ireland
  • Dublin, Ireland, D09 YD60
    • 253
• Netherlands
  • Utrecht, Netherlands, 3584 CW
    • 264
• Poland
  • Bydgoszcz, Poland, 85-163
    • 283
  • Kraków, Poland, 30721
    • 385
  • Warszawa, Poland, 01-684
    • 254
  • Warszawa, Poland, 02-473
    • 274
• Spain
  • Barcelona, Spain, 08003
    • 302
  • Barcelona, Spain, 08035
    • 115
  • Madrid, Spain, 28046
    • 303
  • Málaga, Spain, 29010
    • 282
  • Valencia, Spain, 46026
    • 194
• United Kingdom
  • Stoke on Trent, United Kingdom, ST4 6QG
    • 263
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
      • 062
  • California
    • San Francisco, California, United States, 94109
      • 278
  • New Jersey
    • Neptune, New Jersey, United States, 07753
      • 287
  • New York
    • New York, New York, United States, 10032
      • 353

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CORT113176 (Dazucorilant) 300 mg; 300 mg of dazucorilant will be administered once daily.	Drug: Dazucorilant 300 mg; 300 mg of dazucorilant will be administered once daily in 4 softgel capsules of 75 mg dazucorilant/capsule.
Experimental: CORT113176 (Dazucorilant) 150 mg; 150 mg of dazucorilant will be administered once daily.	Drug: Dazucorilant 150 mg; 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.
Placebo Comparator: Placebo (matched to study drug); Placebo will be administered once daily.	Other: Placebo; Placebo will be administered once daily in 4 softgel capsules of placebo equivalent.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Corcept Therapeutics
• Investigators: Study Director: Grace Mann, PhD, Corcept Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): November 15, 2022
• Primary Completion (Estimated): October 1, 2024
• Study Completion (Estimated): May 1, 2027

Study Registration Dates

• First Submitted: June 2, 2022
• First Submitted That Met QC Criteria: June 2, 2022
• First Posted (Actual): June 7, 2022

Study Record Updates

• Last Update Posted (Estimated): April 26, 2024
• Last Update Submitted That Met QC Criteria: April 24, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: ALS, Amyotrophic Lateral Sclerosis
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: CORT113176-652; 2021-005611-31 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No