Amyotrophic Lateral Sclerosis, Fasciculation
Conditions
Keywords
ALS, Amyotrophic Lateral Sclerosis, Fasciculation, FLX-787
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.
Interventions
DRUGFLX-787-ODT (orally disintigrating tablet)
Oral Disintegrating Tablet
Sponsors
Flex Pharma, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events | 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 |
| Change from Baseline of Diastolic Blood Pressure in mmHg | 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 | 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 | 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 Respiration Rate in breaths per minute | 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 in Body Temperature in degrees Celsius or Fahrenheit | 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 | Prior to and twice within 4 hours following administration of investigational product on the clinic visit | Oral Cavity Examination performed before and after treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline of Fasciculation Frequency | Prior to and twice within 4 hours following administration of investigational product on the clinic visit | Fasciculations over time measured by EMG before and after treatment |
Countries
United States
Outcome results
None listed