2025-522580-15-00招募中2 期
A phase 2b, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of Usnoflast administered to adult subjects with Amyotrophic Lateral Sclerosis (ALS)
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 206
- 试验地点
- 48
- 主要终点
- Change in disease progression from baseline through 36 weeks, as measured by ALSFRS-R total score and survival
研究概览
简要总结
To evaluate the efficacy of Usnoflast versus placebo, using the ALSFRS-R total score and survival
研究设计
- 分配方式
- Na
- 主要目的
- Ole
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 否
入选标准
- •Male and/or female subjects aged 18 years or older at screening
- •Diagnosis of probable or definite ALS, according to the revised version of the El Escorial World Federation of Neurology criteria
- •Time since onset of first symptom of ALS ≤24 months
- •ALSFRS-R score of ≥35 at screening
- •SVC: ≥60% of predicted capacity at the screening visit
- •Be able to swallow capsules
- •Either not currently receiving riluzole/sodium phenylbutyrate and taurursodiol/tofersen or on a stable dose of riluzole/sodium phenylbutyrate and taurursodiol/tofersen for at least 4 weeks before the screening visit. Subjects receiving riluzole/sodium phenylbutyrate and taurursodiol/tofersen are expected to remain on the same dose throughout the duration of the study
- •Either not currently receiving edaravone or on edaravone treatment. Subjects receiving edaravone must have completed at least 1 cycle of treatment before the screening visit and are expected to continue with a stable dose of edaravone treatment throughout the duration of the study
- •Capable of providing informed consent and complying with study procedures in the opinion of the investigator
排除标准
- •Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair the ability of the subject to provide informed consent, in the opinion of the investigator
- •Active herpes zoster infection within 2 months prior to the screening visit
- •Any medical condition that promotes suicidal attempt or behavior within 6 months prior to the screening visit and in the opinion of the investigator might interfere with subject’s participation in the study or is a risk for a suicide attempt
- •History of unstable or severe cardiac, pulmonary, oncological, hepatic, or renal disease or active cancer or another medically significant illness other than ALS, precluding safe participation of subject in this study in the opinion of the investigator
- •Known allergy, sensitivity, or intolerance to IP or excipients
- •Subjects who have taken concomitant medications that are substrates of drug metabolizing enzymes (CYP1A2 and/or CYP2B6) within 7 days or 5 half-lives of the medication (whichever is longer) before the first dose of IP and throughout the study
- •Use of any steroids, colchicine, or anti-IL-1 inhibitors within 7 days or 5 half-lives of the medication (whichever is longer) prior to the first dose of IP administration
- •Use of any investigational drug concurrently or within 4 weeks or 5 half-lives (whichever is longer), prior to the first dose of IP administration
- •Use or intended use of any medications/products known to alter drug absorption, metabolism, or elimination processes, including St. John’s Wort, within 4 weeks of screening and up to end of study. Use of such medication will be considered on a case-by-case basis as per the opinion of the investigator and/or independent medical monitor.
- •Receiving an elemental diet or parenteral nutrition.
- •Received blood transfusion within 3 months prior to screening.
- •Any clinically significant condition and/or laboratory significant value that would prevent the subject from participating in the study in the opinion of the investigator
- •Subjects with HIV, hepatitis B, hepatitis C, coronary artery disease, or active gastrointestinal condition that might interfere with drug absorption
- •Inability to be venipunctured or those not able to tolerate venous puncture
- •Employee of the investigator or study site, with direct involvement in the proposed study or other studies under the direction of that investigator or study site, as well as family members of employees of investigator or the investigator.
- •Any condition not mentioned in any of the above criteria that, as per the investigator, would hinder participation of the subject in the study. This may include, but not limited to, considerations of safety, compliance, or other factors that could impact the integrity of the study or the well-being of the subject
- •If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception during the study and for at least 1 month after administration of last dose of IP. If male of reproductive capacity, unwilling to use effective contraception during the study and for at least 1 month after administration of last dose of IP
- •Received a live vaccine within 14 days before the screening visit or planning to receive during the study duration.
- •Subjects who have received stem cell or gene therapy for ALS at any time in the past
- •Following laboratory test values at screening: - Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) values >3.0 × upper limit of normal (ULN) - Bilirubin >1.5 × ULN unless the subject has documented Gilbert’s syndrome (isolated bilirubin >1.5 × ULN is acceptable if bilirubin is fractionated, and direct bilirubin is <35%) - Estimated glomerular filtration rate <60 mL/min/1.73 m2
- •For those participating in the optional CSF collection, contraindications to lumbar puncture including but not limited to lumbar scoliosis, coagulopathy, infection at site of puncture, or use of anticoagulants.
- •Subjects with history of epilepsy within 6 months of screening visit.
- •Surgery within last 3 months or planned major surgery within next 3 months from the date of screening (other than minor cosmetic surgery and minor dental surgery).
- •Serious illness (e.g., pneumonia, septicemia) within 4 weeks of the screening visit; infection requiring hospitalization or treatment with intravenous antibiotics, antivirals, or antifungals within 4 weeks of screening; chronic bacterial infection (such as tuberculosis) deemed unacceptable as per the judgment of the investigator
研究组 & 干预措施
Placebo will be identical to Usnoflast capsules in terms of size, shape, and color.
Placebo
干预措施: Placebo will be identical to Usnoflast capsules in terms of size, shape, and color. (Drug)
Usnoflast, Usnoflast
Test
干预措施: Usnoflast (Drug)
结局指标
主要结局
Change in disease progression from baseline through 36 weeks, as measured by ALSFRS-R total score and survival
Change in disease progression from baseline through 36 weeks, as measured by ALSFRS-R total score and survival
次要结局
- 2. Change in serum levels of NfL protein from baseline to Week 36
- 3. Change in ALSFRS-R total score and scores of various functional items/domains of the ALSFRS-R total score from baseline to Week 36
- 1. Change in SVC from baseline to Week 36
- 4. Change in ALSAQ-40 score from baseline to Week 36
- 5. Number of TEAEs and SAEs up to Week 36
- 6. Assessment of PK in plasma (baseline, Week 16, and Week 36) and CSF (baseline, Week 16, and Week 36)
- 7. Change in CSF levels of NfL protein from baseline to Week 36
- 8. Time from baseline to the occurrence of death or PAV (>22 hours daily for >7 days) (from baseline through Week 36)
研究者
Dr. Deven Parmar
Scientific
Zydus Therapeutics Inc.
研究点 (48)
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