Disease of nerve cells that control muscles Neurodegenerative syndrome
Conditions
Interventions
Patients are randomized in a 1:1:1 ratio to receive CORT113176 150 mg,
CORT113176 300 mg, or placebo once daily. All patients, regardless of the arm
to which they were randomized, will take one dose
Sponsors
Corcept Therapeutics
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Male and female patients >=18 years of age with ALS as defined by Gold Coast Criteria. 2. Patients with sporadic or familial ALS with a risk of ALS progression characterized by an ENCALS risk profile score >=-6 and =30 days and/or edaravone for >=60 days and/or sodium phenylbutyrate and taurursodial maintenance dosage >=30 days prior to Screening. 4. Medically able to undergo the study procedures and to adhere to the visit schedule at the time of study entry, as determined by the Investigator. 5. Able to understand the purpose and risks of the study; willing and able to adhere to scheduled visits, treatment plans, laboratory tests, and other study evaluations and procedures. 6. Provide written informed consent for participation in the study. 7. Male patients and female patients of childbearing potential must agree to use a protocol-specified method of contraception from screening and during the study until 28 days after last dose of study drug.
Exclusion criteria
Exclusion criteria: 1. History of a clinically significant non-ALS neurologic disorder, including, but not limited to, muscular dystrophy, spinal stenosis, peripheral neuropathy, inherited neuropathies, Alzheimer*s disease, cervical spondylosis, Parkinson*s disease, Lewy body dementia, vascular dementia, Huntington*s disease, epilepsy, stroke, multiple sclerosis, multifocal motor neuropathy, diabetic neuropathy, brain tumor, or brain infection/abscess. 2. Inability to swallow capsules. 3. Blood platelet count 3 × upper limit of normal (ULN). 14. QTcF interval based on the mean of 2 ECGs of >450 ms, for men and >470 ms for women. 15. History of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, family history of long-QT syndrome). 16. Positive nasopharyngeal PCR test for SARS-CoV-2 on Day -1 or within 8 weeks prior to Screening. 17. Ongoing use of any strong CYP3A4 inhibitor/inducer or any medication with a narrow therapeutic index that is predominantly metabolized by CYP2C8. 18. Taking, or have taken, any strong CYP3A inducer within 30 days (or 5 half-lives if longer) before Screening, or any strong CYP3A inhibitor within 14 days before Screening. 19. Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period. 20. Received any live or attenuated vaccine within 30 days, before the first dose of study drug. Exceptions may apply on a case-by-case basis, if considered not to interfere with the objectives of the study, as agreed by the PI and the Corcept Medical Monitor. 21. Currently using glucocorticoids or have a history of r
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary Efficacy Endpoint • Change from Baseline to Week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R) total score. Primary Safety Endpoint • Incidence of AEs, SAEs, treatment-related AEs, AEs by severity, and deaths due to AEs. | — |
Secondary
| Measure | Time frame |
|---|---|
| • Change from Baseline to Week 24 in muscle strength (assessed using hand-held dynamometer). • Change from Baseline to Week 24 in: * Percent Slow Vital Capacity * EQ-5D-5L. • Time to death. • Time to respiratory support >22 hours per day for 7 days. • Time to death or time to respiratory support >22 hours per day for 7 days. • Combined Assessment of Function and Survival (CAFS). • Plasma samples for pharmacokinetic (PK) analysis will be obtained in a dedicated PK substudy in a subset (~20%) of patients at the Week 3 visit. The dazucorilant AUC and Cmax will be reported. | — |
Countries
Netherlands
Outcome results
None listed