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 2-part study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).

Study Overview

• Status: Recruiting
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: Dazucorilant 300 mg; Drug: Dazucorilant 150 mg; Other: Placebo; Drug: Dazucorilant

Detailed Description

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
• Enrollment (Estimated): 279
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Clinical Trial Lead; Phone Number: +1 (650) 249-9965; Email: study652@corcept.com
• Study Contact Backup: Name: Corcept Therapeutics Incorporated

Study Locations

• Belgium
  • Leuven, Belgium, 3000
    • Active, not recruiting
    • 108
• Canada
  • Ontario
    • Hamilton, Ontario, Canada, L8N 3Z5
      • Active, not recruiting
      • 425
  • Quebec
    • Montreal, Quebec, Canada, H3A2B4
      • Active, not recruiting
      • 273
• France
  • Bron, France, 69500
    • Active, not recruiting
    • 422
  • Lille, France, 59037
    • Active, not recruiting
    • 258
  • Limoges, France, 87042
    • Active, not recruiting
    • 257
  • Marseille, France, 13385
    • Active, not recruiting
    • 261
  • Montpellier, France, 42395
    • Active, not recruiting
    • 423
  • Nice, France, 06001
    • Active, not recruiting
    • 259
  • Paris, France, 75651
    • Active, not recruiting
    • 262
  • Tours, France, 37000
    • Active, not recruiting
    • 256
• Germany
  • Berlin, Germany, 13353
    • Active, not recruiting
    • 255
  • Bonn, Germany, 53127
    • Active, not recruiting
    • 270
  • Dresden, Germany, 1307
    • Active, not recruiting
    • 268
  • Hanover, Germany, 30625
    • Active, not recruiting
    • 260
  • Jena, Germany, 7747
    • Active, not recruiting
    • 265
  • München, Germany, 81675
    • Active, not recruiting
    • 386
  • Rostock, Germany, 18147
    • Active, not recruiting
    • 267
  • Ulm, Germany, 89081
    • Active, not recruiting
    • 269
• Ireland
  • Dublin, Ireland, D09 YD60
    • Active, not recruiting
    • 253
• Netherlands
  • Utrecht, Netherlands, 3584 CW
    • Active, not recruiting
    • 264
• Poland
  • Bydgoszcz, Poland, 85-163
    • Active, not recruiting
    • 283
  • Krakow, Poland, 30721
    • Active, not recruiting
    • 385
  • Warsaw, Poland, 01-684
    • Active, not recruiting
    • 254
  • Warsaw, Poland, 02-473
    • Active, not recruiting
    • 274
• Spain
  • Barcelona, Spain, 08003
    • Active, not recruiting
    • 302
  • Barcelona, Spain, 08035
    • Active, not recruiting
    • 115
  • Madrid, Spain, 28046
    • Active, not recruiting
    • 303
  • Málaga, Spain, 29010
    • Active, not recruiting
    • 282
  • Valencia, Spain, 46026
    • Active, not recruiting
    • 194
• United Kingdom
  • Stoke-on-Trent, United Kingdom, ST4 6QG
    • Active, not recruiting
    • 263
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
      • Recruiting
      • 062
  • California
    • San Francisco, California, United States, 94109
      • Recruiting
      • 278
  • New Jersey
    • Neptune City, New Jersey, United States, 07753
      • Recruiting
      • 287
  • New York
    • New York, New York, United States, 10032
      • Recruiting
      • 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. In Part 1, patients must have a risk of ALS progression characterized by a European Network for the Cure of ALS (ENCALS) risk profile score ≥ -6 and ≤ -3. In Part 2 patients must have a risk of ALS progression characterized by an ENCALS risk profile score ≥ -7 and ≤ -3.
• If taking riluzole, edaravone, and/or sodium phenylbutyrate and taurursodiol, must be on a stable dose prior to Screening. Sodium phenylbutyrate and taurursodiol are not permitted for patients enrolled in Part 2 of the study.
• Part 2 only: Patients with a pathogenic mutation in superoxide dismutase 1 gene (SOD1) must not be receiving treatment with tofersen or eligible for treatment with tofersen if available. Patients who have received prior treatment with tofersen and discontinued due to safety and/or efficacy reasons prior to Screening are eligible.
• Part 2 only: Use of ultra high-dose methylcobalamin for the treatment of ALS is permitted provided the patient has been on a stable dose for ≥11 weeks prior to the Day 1 visit.

Exclusion Criteria:

• History of a clinically significant non-ALS neurologic disorder
• Inability to swallow capsules.
• Blood platelet count <150,000/mm^3.
• Renal impairment indicated by Estimated Glomerular Filtration Rate (eGFR) ≤30 mL/min/1.73 m^2. Part 2 only: Patients with a recent history of acute kidney injury should have returned to their baseline renal function prior to enrollment.
• Human immunodeficiency virus (HIV) or current chronic/active infection with hepatitis C virus or hepatitis B virus. Part 2 only: Known history of HIV or chronic/active infection with hepatitis C or hepatitis B virus; testing does not need to be performed if infection status is unknown.
• 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

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part 1: 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 capsules of 75 mg dazucorilant/capsule.; Other Names: CORT113176
Experimental: Part 1: 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 capsules, 2 capsules with 75 mg dazucorilant/capsule, and 2 capsules of placebo equivalent.; Other Names: CORT113176; Other: Placebo; Placebo will be administered once daily in capsules of placebo equivalent.
Placebo Comparator: Part 1: Placebo (matched to study drug); Placebo will be administered once daily.	Other: Placebo; Placebo will be administered once daily in capsules of placebo equivalent.
Experimental: Part 2: CORT113176 (Dazucorilant) 75 - 300 mg Dose-titration; 75 mg of dazucorilant will be administered once daily for 1 week, and increased weekly in 75 mg increments up to 300 mg once daily.	Drug: Dazucorilant; Dazucorilant will be administered once daily in 75-mg capsules.; Other Names: CORT113176

What is the study measuring?

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.	This outcome measure is assessed in study Part 1.	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.	Baseline to Week 24
Incidence of treatment-emergent AEs and SAEs	This outcome measure is assessed in study Part 2.	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.	Baseline up to Week 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline to Week 24 in muscle strength (assessed using hand-held dynamometer)	This outcome measure is assessed in study Part 1.	Baseline to Week 24
Change from Baseline to Week 24 in Percent Slow Vital Capacity	This outcome measure is assessed in study Part 1.	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.	Baseline to Week 24
Time to Death	This outcome measure is assessed in study Part 1.	From randomization date to the date of death from any cause up to 156 weeks

Collaborators and Investigators

• Sponsor: Corcept Therapeutics
• Investigators: Study Director: Sophia Majeed, PharmD, PhD, Corcept Therapeutics Incorporated

Study record dates

Study Major Dates

• Study Start (Actual): November 15, 2022
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): November 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 (Actual): March 30, 2026
• Last Update Submitted That Met QC Criteria: March 27, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: ALS, Amyotrophic Lateral Sclerosis
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis; CORT113176
• 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