Clinical trial NCT03338114: Study to Evaluate the Safety & Efficacy of FLX-787-ODT to Treat Fasciculations in Tongue and Upper or Lower Extremity Muscles Most Affected in Subjects With ALS
Official title: 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)
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE1
PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Fasciculation
Keywords: ALS
Amyotrophic Lateral Sclerosis
Fasciculation
FLX-787
Lead sponsor: Flex Pharma, Inc.
Sponsor class: INDUSTRY
First posted: 2017-11-09
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

* 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

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
* 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).

Primary outcomes:
measure: Change from Baseline of Diastolic Blood Pressure in mmHg
timeFrame: Prior to and within 3 hours following administration of investigational product on the single clinic visit
measure: Change from Baseline of Systolic Blood Pressure in mmHg
timeFrame: Prior to and within 3 hours following administration of investigational product on the single clinic visit
measure: Change from Baseline in Heart Rate in beats per minute
timeFrame: Prior to and within 3 hours following administration of investigational product on the single clinic visit
measure: Change from Baseline in Respiration Rate in breaths per minute
timeFrame: Prior to and within 3 hours following administration of investigational product on the single clinic visit
measure: Change from Baseline in Body Temperature in degrees Celsius or Fahrenheit
timeFrame: Prior to and within 3 hours following administration of investigational product on the single clinic visit
measure: Change from Baseline of Oral Cavity Examination
timeFrame: Prior to and twice within 4 hours following administration of investigational product on the clinic visit
measure: Incidence of Treatment-Emergent Adverse Events
timeFrame: Prior to and following administration of investigational product on the clinic visit with AEs followed for 7 days after by telephone contact
Secondary outcomes:
measure: Change from Baseline of Fasciculation Frequency
timeFrame: Prior to and twice within 4 hours following administration of investigational product on the clinic visit
measure: Change from Baseline of Fasciculation Frequency
timeFrame: Prior to and twice within 4 hours following administration of investigational product on the clinic visit
Other outcomes:
Locations:
city: Winston-Salem
country: United States
facility: Wake Forest University Health Sciences
state: North Carolina
Study officials:
References: