A study into the efficacy and safety of dazucorilant (CORT113176) in patients with Amyotrophic Lateral Sclerosis
EU CTIS ID: 2024-514082-19-00
What this study is testing
Part 1: Double-Blind, Randomized Arms: - To assess the efficacy of dazucorilant in patients with ALS - To assess the safety of dazucorilant in patients with ALS Part 2: Open-Label Dose-Titration Cohort: - To assess the tolerability and safety of a dazucorilant dose-titration regimen in patients with ALS
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male and female patients ≥18 years of age with ALS as defined by Gold Coast criteria
- Patients with sporadic or familial ALS. In Part 1 patients must have a risk of ALS progression characterized by an ENCALS risk profile score ≥ -6 and ≤ -3. In Part 2 patients must have a risk of ALS progression characterized by a TRICALS risk profile score ≥ -7 and ≤ -3.
- Regulatory-authority-approved therapies for the treatment of ALS are permitted. If taking riluzole, edaravone, and/or sodium phenylbutyrate and taurursodiol, must have been on a stable dose of riluzole for ≥30 days, edaravone for ≥60 days and/or sodium phenylbutyrate and taurursodiol maintenance dosage ≥30 days prior to Screening. Sodium phenylbutyrate and taurursodiol are not permitted for patients enrolled in Part 2 of the study.
- 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.
- 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.
- Provide written informed consent for participation in the study. If the patient is willing to sign the ICF but cannot physically sign it, an impartial witness must sign the ICF. Patients who are unable to come to the site in-person following informed consent for initial study participation may subsequently be consented remotely if allowed by local regulations/IRBs.
You likely can't join if
- 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.
- 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.
- History of any other clinically significant cardiovascular, renal, hepatic, endocrine, metabolic, respiratory, gastrointestinal (GI), bleeding, autoimmune, neurological, psychiatric disorder, or unstable medical condition (other than ALS), as judged by the Investigator.
- History and/or symptoms of adrenal insufficiency.
- Abnormal liver function defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3 × upper limit of normal (ULN).
- QTcF interval based on the mean of 2 ECGs of >450 ms, for men and >470 ms for women.
See the full eligibility criteria
Who can join
- Male and female patients ≥18 years of age with ALS as defined by Gold Coast criteria
- Patients with sporadic or familial ALS. In Part 1 patients must have a risk of ALS progression characterized by an ENCALS risk profile score ≥ -6 and ≤ -3. In Part 2 patients must have a risk of ALS progression characterized by a TRICALS risk profile score ≥ -7 and ≤ -3.
- Regulatory-authority-approved therapies for the treatment of ALS are permitted. If taking riluzole, edaravone, and/or sodium phenylbutyrate and taurursodiol, must have been on a stable dose of riluzole for ≥30 days, edaravone for ≥60 days and/or sodium phenylbutyrate and taurursodiol maintenance dosage ≥30 days prior to Screening. Sodium phenylbutyrate and taurursodiol are not permitted for patients enrolled in Part 2 of the study.
- 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.
- 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.
- Provide written informed consent for participation in the study. If the patient is willing to sign the ICF but cannot physically sign it, an impartial witness must sign the ICF. Patients who are unable to come to the site in-person following informed consent for initial study participation may subsequently be consented remotely if allowed by local regulations/IRBs.
- 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
- Part 2 only: Patients with a pathogenic mutation in superoxide dismutase 1 (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.
What rules you out
- 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.
- 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.
- History of any other clinically significant cardiovascular, renal, hepatic, endocrine, metabolic, respiratory, gastrointestinal (GI), bleeding, autoimmune, neurological, psychiatric disorder, or unstable medical condition (other than ALS), as judged by the Investigator.
- History and/or symptoms of adrenal insufficiency.
- Abnormal liver function defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3 × upper limit of normal (ULN).
- QTcF interval based on the mean of 2 ECGs of >450 ms, for men and >470 ms for women.
- History of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, family history of long-QT syndrome).
- Positive nasopharyngeal PCR test for SARS-CoV-2 on Day -1 or within 8 weeks prior to Screening.
- Ongoing use of any strong CYP3A4 inhibitor/inducer, or any medication with a narrow therapeutic index that is predominantly metabolized by CYP2C8 or is a substrate of breast cancer resistance protein (BCRP) or P-glycoprotein 1 (P gp) that cannot be adequately dose adjusted.
- 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.
- Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.
- Inability to swallow capsules.
- Received any live or attenuated vaccine within 30 days, before the first dose of study drug.
- 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.
- Participation in a clinical trial for ALS involving small molecules within 30 days of the Screening, or treatment with another investigational drug (including through compassionate use programs), biological agent, or device within 30 days or 5 half-lives of study agent, whichever is longer. No prior treatment with small interfering RNA, stem cell therapy, or gene therapy is allowed at any time in the patient’s history.
- Unstable or poorly controlled comorbid disease process of any organ system currently requiring active treatment or likely to require treatment adjustment during the study.
- Previous exposure or treatment with glucocorticoid receptor modulators or antagonists.
- History of hypersensitivity or severe reaction to the study drug’s excipients.
- In the Investigator’s opinion, should not participate in the study or may not be capable of providing informed consent or following the study schedule. This includes, but is not limited to, presence of unstable psychiatric disease, cognitive impairment, dementia, or substance abuse within 2 years prior to Screening.
- Is a family member of one of the Sponsor’s employees, the Investigator, or the site staff working directly on the study.
- Blood platelet count <150,000/mm3.
- Renal impairment indicated by eGFR ≤30 mL/min/1.73m2. Part 2 only: Patients with a recent history of acute kidney injury should have returned to their baseline renal function (ie, 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 including patients with chronic or active hepatitis B as diagnosed by serologic tests. 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. Women of childbearing potential who are unwilling or unable to use a highly effective method of contraception from screening through the duration of treatment and up to 28 days after last dose of study drug.
- Known liver impairment (Child-Pugh Class A, B, or C).
- History of Class III/IV heart failure (per New York Heart Association).
- At the time of Screening, any use of non-invasive ventilation (NIV), e.g., continuous positive airway pressure [CPAP], noninvasive bi-level positive airway pressure [NPPV] or noninvasive volume ventilation [NVV] for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation.
The study team makes the final eligibility decision.
Where it's taking place
- Netherlands
- Germany
- United States
- United Kingdom
- Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Netherlands; Germany; United States; United Kingdom; Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from EU CTIS. The study team decides eligibility, and your own doctor gives medical advice.