2024-514082-19-00招募中2 期
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of CORT113176 (Dazucorilant) in Patients with Amyotrophic Lateral Sclerosis (DAZALS)
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 225
- 试验地点
- 28
- 主要终点
- Primary Efficacy Endpoint: Change from Baseline to Week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R) total score.
研究概览
简要总结
To assess the efficacy of dazucorilant in patients with ALS. To assess the safety of dazucorilant in patients with ALS.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Male and female patients ≥18 years of age with ALS as defined by Gold Coast criteria
- •Patients with sporadic or familial ALS with a risk of ALS progression characterized by an ENCALS risk profile score ≥ -6 and ≤ -
- •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.
- •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.
- •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
排除标准
- •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.
- •Any form of cancer within the 5 years before first dose in this study (with the exception of basal cell and/or squamous cell cancer of the skin that has been treated completely and is without evidence of local recurrence or metastasis).
- •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 CYP2C
- •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. 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.
- •Currently using glucocorticoids or have a history of regular systemic glucocorticoid use at any dose within the last 12 months or 3 months for inhaled products before 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/mm
- •Renal impairment indicated by eGFR ≤30 mL/min/1.73m
- •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.
- •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.
结局指标
主要结局
Primary Efficacy Endpoint: Change from Baseline to Week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R) total score.
Primary Efficacy Endpoint: Change from Baseline to Week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R) total score.
Primary Safety Endpoints: Incidence of AEs, SAEs, treatment-related AEs, AEs by severity, and death due to AEs.
Primary Safety Endpoints: Incidence of AEs, SAEs, treatment-related AEs, AEs by severity, and death due to AEs.
次要结局
- Time to death or time to respiratory support >22 hours per day for 7 days.
- Combined Assessment of Function and Survival (CAFS).
- 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.
- 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.
研究者
Corcept Therapeutics Incorporated
Scientific
Corcept Therapeutics Inc.
研究点 (28)
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