Dazucorilant in Patients With Amyotrophic Lateral Sclerosis

NCT ID: NCT05407324

Last Updated: 2025-12-22

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 279 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-11-15
• Study Completion Date: 2027-11-30

Brief Summary

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

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS, Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Part 1: Placebo (matched to study drug)

Placebo will be administered once daily.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Placebo will be administered once daily in capsules of placebo equivalent.

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.

Group Type: EXPERIMENTAL

Dazucorilant

Intervention Type: DRUG

Dazucorilant will be administered once daily in 75-mg capsules.

Part 1: CORT113176 (Dazucorilant) 300 mg

300 mg of dazucorilant will be administered once daily.

Group Type: EXPERIMENTAL

Dazucorilant 300 mg

Intervention Type: DRUG

300 mg of dazucorilant will be administered once daily in 4 capsules of 75 mg dazucorilant/capsule.

Part 1: CORT113176 (Dazucorilant) 150 mg

150 mg of dazucorilant will be administered once daily.

Group Type: EXPERIMENTAL

Dazucorilant 150 mg

Intervention Type: DRUG

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

Intervention Type: OTHER

Placebo will be administered once daily in capsules of placebo equivalent.

Interventions

Dazucorilant 300 mg

300 mg of dazucorilant will be administered once daily in 4 capsules of 75 mg dazucorilant/capsule.

Intervention Type: 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.

Intervention Type: DRUG

Placebo

Placebo will be administered once daily in capsules of placebo equivalent.

Intervention Type: OTHER

Dazucorilant

Dazucorilant will be administered once daily in 75-mg capsules.

Intervention Type: DRUG

Other Intervention Names

CORT113176; CORT113176; CORT113176

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Corcept Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Corcept Therapeutics Incorporated

Role: STUDY_DIRECTOR

Corcept Therapeutics Incorporated

Locations

062

Phoenix, Arizona, United States

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

261

Marseille, , France

423

Montpellier, , France

259

Nice, , France

262

Paris, , France

256

Tours, , France

255

Berlin, , Germany

270

Bonn, , Germany

268

Dresden, , Germany

260

Hanover, , Germany

265

Jena, , Germany

386

München, , Germany

267

Rostock, , Germany

269

Ulm, , Germany

253

Dublin, , Ireland

264

Utrecht, , Netherlands

283

Bydgoszcz, , Poland

385

Krakow, , Poland

254

Warsaw, , Poland

274

Warsaw, , Poland

302

Barcelona, , Spain

115

Barcelona, , Spain

303

Madrid, , Spain

282

Málaga, , Spain

194

Valencia, , Spain

263

Stoke-on-Trent, , United Kingdom

Countries

United States; Belgium; Canada; France; Germany; Ireland; Netherlands; Poland; Spain; United Kingdom

Other Identifiers

2021-005611-31

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CORT113176-652

Identifier Type: -

Identifier Source: org_study_id