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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03334786
• Date of registration: 26/10/2017
• Prospective Registration: Yes
• Primary sponsor: Flex Pharma, Inc.
• Public title: Study to Evaluate Safety & Efficacy of FLX-787-ODT to Treat Fasciculations in Tongue and Appendicular Muscle in Adult Subjects With ALS
• Scientific title: 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)
• Date of first enrolment: April 5, 2018
• Target sample size: 3
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03334786
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

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).



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fasciculation

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: FLX-787-ODT

Primary Outcome(s)

Change from Baseline in Heart Rate in beats per minute [Time Frame: Prior to and within 3 hours following administration of investigational product on the single clinic visit]

Change from Baseline in Respiration Rate in breaths per minute [Time Frame: Prior to and within 3 hours following administration of investigational product on the single clinic visit]

Change from Baseline of Systolic Blood Pressure in mmHg [Time Frame: Prior to and within 3 hours following administration of investigational product on the single clinic visit]

Change from Baseline of Oral Cavity Examination [Time Frame: Prior to and twice within 4 hours following administration of investigational product on the clinic visit]

Incidence of Treatment-Emergent Adverse Events [Time Frame: Prior to and following administration of investigational product on the clinic visit with AEs followed for 7 days after by telephone contact]

Change from Baseline in Body Temperature in degrees Celsius or Fahrenheit [Time Frame: Prior to and within 3 hours following administration of investigational product on the single clinic visit]

Change from Baseline of Diastolic Blood Pressure in mmHg [Time Frame: Prior to and within 3 hours following administration of investigational product on the single clinic visit]

Secondary Outcome(s)

Change from Baseline in Swallowing Assessments [Time Frame: Prior to and once within 4 hours following administration of investigational product on the clinic visit]

Change from Baseline in Speech Assessments [Time Frame: Prior to and once within 4 hours following administration of investigational product on the clinic visit]

Change from Baseline in Peak Tongue Strength in kPa by Iowa Oral Performance Instrument [Time Frame: Prior to and once within 4 hours following administration of investigational product on the clinic visit]

Change from Baseline of Fasciculation Frequency [Time Frame: Prior to and twice within 4 hours following administration of investigational product on the clinic visit]

Secondary ID(s)

FLX-787-107

Source(s) of Monetary Support

Please refer to primary and secondary sponsors