Study to Evaluate Safety & Efficacy of FLX-787-ODT to Treat Fasciculations in Tongue and Appendicular Muscle in Adult Subjects With ALS

An Open Label Study to Evaluate the Safety and Efficacy of FLX-787-ODT for Treatment of Fasciculations in the Tongue and One Appendicular Muscle in Adult Subjects With Amyotrophic Lateral Sclerosis (ALS)

The FLX-787-107 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, if tongue and muscle strength, speech, and swallowing are affected, 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.

Inclusion Criteria:

• Documented diagnosis of ALS diagnosis of less than 5 years.
• Greater than 6 fasciculations per minute noted at least in the tongue by clinical, ultrasound, or EMG evaluation.
• Normal oral cavity exam at screening.

Exclusion Criteria:

• 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.
• 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.
• 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).