An Open Label Study to Evaluate the Safety and Efficacy of FLX-787-ODT for Treatment of Fasciculations in the Tongue and Upper or Lower Extremity Muscles Most Affected in Subjects With Amyotrophic Lateral Sclerosis (ALS)
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Change from Baseline in Respiration Rate in breaths per minute
研究概览
简要总结
The FLX-787-106 study will determine how well FLX-787-ODT works to reduce fasciculations in patients with Amyotrophic Lateral Sclerosis (ALS). The study will measure how often fasciculations occur, and monitor any side effects that might develop while taking the investigational product. Participants will be assessed before and after taking a single dose of FLX-787-ODT. Approximately 15 people will take part in this study at one center in the United States. Participants will be in the study for a single clinic visit and receive a telephone call 7 days later to monitor for side effects.
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
- •Documented diagnosis of ALS diagnosis of less than 3 years.
- •Expected survival > 6 months
- •Frequent fasciculations noted during clinical examination of any single muscle (>6 visible fasciculations per minute) observed in Part I
- •Normal oral cavity exam at screening
- •Proficient in English
- •Male subjects will either be surgically sterile or agree to use a medically acceptable method of contraception during sexual contact with females of childbearing potential, and will refrain from sperm donation for 45 days following the study
- •Male subjects will either be surgically sterile or agree to use a medically acceptable method of contraception during sexual contact with females of childbearing potential, and will refrain from sperm donation for 45 days following the study
排除标准
- •Presence of clinically significant or unstable condition that would result in an increased risk of study participation or difficulty in interpretation of the study results
- •Tremor or other movement disorder that would interfere with recording
- •Inability to lie flat
- •Presence of major gastrointestinal disorders, such as inflammatory bowel disease, diverticulitis, active peptic ulcer disease, or significant gastroesophageal reflux disease (i.e., not well-controlled on antacids or proton pump inhibitors), or oral or esophageal lesions/ulcers
- •Presence of laryngospasm or significant swallowing problems
- •Presence of percutaneous endoscopic gastrostomy, esophagogastroduodenoscopy, or G-tube
- •Inability to tolerate a spicy sensation in the mouth or stomach
- •Actively using illicit drugs or history of chronic substance abuse within the past year prior to screening, including abuse of alcohol
- •Participated in a clinical study (except natural history studies without administration of an investigational product) within 30 days prior to screening
- •Pregnant, breastfeeding, or planning to become pregnant
- •Blood pressure of ≥160 mmHg systolic and/or ≥100 mmHg diastolic
- •Clinically significant abnormalities in laboratory findings (including screening complete electrolyte panel, complete blood count, liver function tests).
研究组 & 干预措施
FLX-787-ODT (orally disintigrating tablet)
FLX-787-ODT (orally disintigrating tablet)
干预措施: FLX-787-ODT (orally disintigrating tablet) (Drug)
结局指标
主要结局
Change from Baseline in Respiration Rate in breaths per minute
时间窗: Prior to and within 3 hours following administration of investigational product on the single clinic visit
Respiration rate collected before and after treatment
Change from Baseline of Diastolic Blood Pressure in mmHg
时间窗: Prior to and within 3 hours following administration of investigational product on the single clinic visit
Diastolic blood pressure collected before and after treatment
Change from Baseline of Systolic Blood Pressure in mmHg
时间窗: Prior to and within 3 hours following administration of investigational product on the single clinic visit
Systolic blood pressure collected before and after treatment
Change from Baseline in Heart Rate in beats per minute
时间窗: Prior to and within 3 hours following administration of investigational product on the single clinic visit
Heart rate collected before and after treatment
Change from Baseline in Body Temperature in degrees Celsius or Fahrenheit
时间窗: Prior to and within 3 hours following administration of investigational product on the single clinic visit
Body temperature collected before and after treatment
Change from Baseline of Oral Cavity Examination
时间窗: Prior to and twice within 4 hours following administration of investigational product on the clinic visit
Oral Cavity Examination performed before and after treatment
Incidence of Treatment-Emergent Adverse Events
时间窗: Prior to and following administration of investigational product on the clinic visit with AEs followed for 7 days after by telephone contact
Adverse Event Information collected throughout the study
次要结局
- Change from Baseline of Fasciculation Frequency(Prior to and twice within 4 hours following administration of investigational product on the clinic visit)