Dazucorilant in Patients With Amyotrophic Lateral Sclerosis

NCT05407324 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: CORT113176-6522021-005611-31
• Study Type: interventional
• Target: 279
• Locations: 10 countries
• Sites: 35

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).

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS, Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (4)

• 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

• Time to Death

  From randomization date to the date of death from any cause up to 156 weeks

Study Arms (4)

Part 1: CORT113176 (Dazucorilant) 300 mg

EXPERIMENTAL

300 mg of dazucorilant will be administered once daily.

Drug: Dazucorilant 300 mg

Part 1: CORT113176 (Dazucorilant) 150 mg

EXPERIMENTAL

150 mg of dazucorilant will be administered once daily.

Drug: Dazucorilant 150 mg; Other: Placebo

Part 1: Placebo (matched to study drug)

PLACEBO COMPARATOR

Placebo will be administered once daily.

Other: Placebo

Part 2: CORT113176 (Dazucorilant) 75 - 300 mg Dose-titration

EXPERIMENTAL

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

Interventions

Dazucorilant 300 mg DRUG

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

Also known as: CORT113176

Part 1: CORT113176 (Dazucorilant) 300 mg

Dazucorilant 150 mg 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.

Also known as: CORT113176

Part 1: CORT113176 (Dazucorilant) 150 mg

Placebo OTHER

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

Part 1: CORT113176 (Dazucorilant) 150 mg; Part 1: Placebo (matched to study drug)

Dazucorilant DRUG

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

Also known as: CORT113176

Part 2: CORT113176 (Dazucorilant) 75 - 300 mg Dose-titration

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 Treatment Research Initiative to Cure ALS (TRICALS) 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.

You may not qualify if:

• 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 (i.e, eGFR prior to acute kidney injury) 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.
• Cancer that is currently being treated (except adequately controlled basal cell carcinoma or squamous cell carcinoma of the skin, stage I endometrial cancer or carcinoma in situ of the cervix or breast) or a history of cancer with an expected survival \< 2 years.
• Current or anticipated need of a diaphragm pacing system (DPS).
• Previous exposure or treatment with glucocorticoid receptor modulators or antagonists.
• Taking, or have taken, any systemic, inhaled, or potent dermatologic topical corticosteroids (Class I to III) within a period equivalent to 5 half-lives of the corticosteroid used prior to first dose of study drug. Patients who have stopped glucocorticoid use should have an alternative option if their condition deteriorates during the study.

Sponsors & Collaborators

• Corcept Therapeutics: lead

Study Sites (35)

062

Phoenix, Arizona, 85013, United States

Location

278

San Francisco, California, 94109, United States

Location

287

Neptune City, New Jersey, 07753, United States

Location

353

New York, New York, 10032, United States

Location

108

Leuven, 3000, Belgium

Location

425

Hamilton, Ontario, L8N 3Z5, Canada

Location

273

Montreal, Quebec, H3A2B4, Canada

Location

422

Bron, 69500, France

Location

258

Lille, 59037, France

Location

257

Limoges, 87042, France

Location

261

Marseille, 13385, France

Location

423

Montpellier, 42395, France

Location

259

Nice, 06001, France

Location

262

Paris, 75651, France

Location

256

Tours, 37000, France

Location

255

Berlin, 13353, Germany

Location

270

Bonn, 53127, Germany

Location

268

Dresden, 1307, Germany

Location

260

Hanover, 30625, Germany

Location

265

Jena, 7747, Germany

Location

386

München, 81675, Germany

Location

267

Rostock, 18147, Germany

Location

269

Ulm, 89081, Germany

Location

253

Dublin, D09 YD60, Ireland

Location

264

Utrecht, 3584 CW, Netherlands

Location

283

Bydgoszcz, 85-163, Poland

Location

385

Krakow, 30721, Poland

Location

254

Warsaw, 01-684, Poland

Location

274

Warsaw, 02-473, Poland

Location

302

Barcelona, 08003, Spain

Location

115

Barcelona, 08035, Spain

Location

303

Madrid, 28046, Spain

Location

282

Málaga, 29010, Spain

Location

194

Valencia, 46026, Spain

Location

263

Stoke-on-Trent, ST4 6QG, United Kingdom

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

CORT113176

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Sophia Majeed, PharmD, PhD

  Corcept Therapeutics Incorporated

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 2, 2022
• First Posted: June 7, 2022
• Study Start: November 15, 2022
• Primary Completion: October 1, 2026
• Study Completion (Estimated): November 1, 2027
• Last Updated: September 24, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1); GB (1); US (4); PL (4); DE (8); IE (1); CA (2); NL (1); ES (5); FR (8)