Tolerability and Efficacy of L-Serine in Patients With Amyotrophic Lateral Sclerosis: A Phase IIa Study
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 43
- 试验地点
- 1
- 主要终点
- Dose Tolerability Based on Subject Reporting
研究概览
简要总结
The purpose of this study is to determine the tolerability of L-Serine oral doses for ALS patients and assess preliminary indications of efficacy
详细描述
All patients will receive the same dose of the study treatment over 6 months. For each participant the study will last approximately one year with follow up visits after the treatment period of 6 months is completed. The visits will include blood draws, vital sign checks, neurological and physical exams, pulmonary testing with forced vital capacity (FVC), and questionnaires.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of probable or definite ALS
- •ALSFRS-R score >25 and FVC score ≥ 60% predicted
- •If currently taking Riluzole and/or Edaravone/Radicava must be on stable dose for 3 months prior to Baseline/Screening. If the dosing has not been stable for 3 months prior to Baseline/Screening or if stopped due to an adverse event, the waiting period off the medication will be 7 days. If not on either of these medications may start if desired either or both medications after enrollment into study.
排除标准
- •Diagnosis of probable or definite ALS more than 3 years prior to study enrollment
- •Diagnosis or previous history of ischemic stroke, brain tumor, uncontrolled diabetes, renal insufficiency, or severe hypertension.
- •Diagnosis or previous history of comorbid progressive neurodegenerative disease such as Alzheimer's disease, Parkinson's disease, Lewy Body disease, Pick's disease, Huntington's disease, Progressive Supranuclear palsy. ALS patients diagnosed with frontotemporal dementia will not be excluded from this study.
- •Diagnosis or previous history of symptomatic peripheral neuropathy. Patients with findings of peripheral neuropathy on electrodiagnostic tests only but no clinical symptoms at the time of enrollment are eligible.
- •Undergoing any chemotherapy or radiation therapy for any cancer
- •Any medical condition likely to interfere with the conduct of the trial or survival of the patient during this study period
- •Pregnant women or women who are breast feeding
- •Has taken L-Serine supplement within 30 days prior to start of study drug
研究组 & 干预措施
L-Serine
L-Serine 15 grams orally twice a day as tolerated for 6 months
干预措施: L-Serine (Drug)
结局指标
主要结局
Dose Tolerability Based on Subject Reporting
时间窗: From baseline through when participants withdrew from study or up to 48 weeks
Tolerability was based off of participant self-reported GI symptoms at any time during their participation.
次要结局
- Mean ALS Functional Rating Scale - Revised (ALSFRS-R) Score(Baseline through 12 weeks)
- Efficacy Based on Pulmonary Forced Vital Capacity (FVC) as Measured by Mean Slope Through Time(No data available)
研究者
Elijah W. Stommel
Staff Physician, Neurology
Dartmouth-Hitchcock Medical Center