A Phase 2A Safety and Biomarker Study of EPI-589 in Subjects With Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 19
- 试验地点
- 3
- 主要终点
- Number of Participants With Drug-Related Serious Adverse Events (SAEs)
研究概览
简要总结
This is an open label study with 30-day run in phase to establish baseline parameters, 90-day treatment phase, and a 90-day withdrawal phase to determine long-term effects, duration of treatment response, and potential effects of EPI-589 therapy on known trajectory.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 21 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of possible, probable, laboratory supported probable, or definite ALS by E1 Escorial Criteria
- •Forced vital capacity (FVC) ≥ 70% of predicted
- •Weakness onset within 3 years
- •Agreement to use contraception if within reproductive years
- •Willingness and ability to comply with study procedures
- •Stable regimen of dietary supplements and /or riluzole for at least 30 days prior to enrollment
- •Abstention from use of other investigative or non-approved drugs
- •Participants must be able to swallow 0.375 * 0.700 inch tablets
排除标准
- •Allergy to EPI-589
- •Use of ventilation
- •Participation in other intervention studies
- •Diagnosis of any other neurologic disease
- •Malignancy within the past 2 years
- •History of stroke
- •History of brain surgery
- •Hepatic insufficiency with liver function tests (LFTs) greater than 3 times upper limit of normal (ULN)
- •Renal insufficiency requiring dialysis
- •End stage cardiac failure
- •Participation in a trial of a device, drug, or other therapy for ALS within 3 months of screening or during the trial
研究组 & 干预措施
EPI-589
Participants will receive EPI-589 500 milligrams (mg) (2 tablets of 250 mg each) twice daily (BID) for 3 months, unless discontinued for safety or tolerability issues.
干预措施: EPI-589 (Drug)
结局指标
主要结局
Number of Participants With Drug-Related Serious Adverse Events (SAEs)
时间窗: Baseline (Day 0) to Month 6
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
次要结局
- Change From Baseline in Respiratory Rate at Month 6(Baseline, Month 6)
- Area Under the Plasma Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12)(Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3)
- Maximum Observed Plasma Concentration (Cmax)(Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3)
- Change From Baseline in ALSFRS-R Total Score at Month 6(Baseline, Month 6)
- Change From Baseline in Vital Capacity at Month 6(Baseline, Month 6)
- Change From Baseline in MIP at Month 6(Baseline, Month 6)
- Change From Baseline in SpO2 at Month 6(Baseline, Month 6)
- Change From Baseline in Muscle Function at Month 6, as Assessed by Handheld Dynamometry Parameters (Grip Strength, Shoulder Flexion, Knee Extension, Hip Flexion, Elbow Flexion, Elbow Extension, and Ankle Dorsi Flexion)(Baseline, Month 6)
- Change From Baseline in Number of Words Participant Read at Month 6(Baseline, Month 6)
- Change From Baseline in Heart Rate at Month 6(Baseline, Month 6)
- Change From Baseline in ETCO2 at Month 6(Baseline, Month 6)
- Failure to Thrive: Number of Participants With Weight Loss of More Than 5 Percent (%) From Baseline at Month 6(Baseline, Month 6)
- Change From Baseline in Average Solid Swallowing Time and Water Swallowing Time at Month 6(Baseline, Month 6)
- Change From Baseline in Time Spent in Reading at Month 6(Baseline, Month 6)
- Change From Baseline in Number of Words Per Minute Read at Month 6(Baseline, Month 6)
- Number of Participants With Normal Loudness, Normal Nasality, and Normal Intelligibility(Month 6)
- Level of Disease-Related Biomarker (Glutathione) in Plasma(Baseline up to Month 6)
- Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF)(Baseline up to Month 3)
- Level of Disease-Related Biomarker (Glutathione) in Urine(Baseline up to Month 6)