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

Study Overview
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Brief Summary
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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.

Official Title
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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)

Conditions
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Amyotrophic Lateral Sclerosis, Fasciculation

Intervention / Treatment
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* Drug: FLX-787-ODT (orally disintigrating tablet)


Participation Criteria
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Eligibility Criteria
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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).

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: FLX-787-ODT (orally disintigrating tablet)<br>FLX-787-ODT (orally disintigrating tablet) | Drug: FLX-787-ODT (orally disintigrating tablet)<br>* Oral Disintegrating Tablet<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline of Diastolic Blood Pressure in mmHg | Diastolic blood pressure collected before and after treatment | 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 | Systolic blood pressure collected before and after treatment | Prior to and within 3 hours following administration of investigational product on the single clinic visit | 
| Change from Baseline in Heart Rate in beats per minute | Heart rate collected before and after treatment | 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 | Respiration rate collected before and after treatment | Prior to and within 3 hours following administration of investigational product on the single clinic visit | 
| Change from Baseline in Body Temperature in degrees Celsius or Fahrenheit | Body temperature collected before and after treatment | Prior to and within 3 hours following administration of investigational product on the single clinic visit | 
| Change from Baseline of Oral Cavity Examination | Oral Cavity Examination performed before and after treatment | Prior to and twice within 4 hours following administration of investigational product on the clinic visit | 
| Incidence of Treatment-Emergent Adverse Events | Adverse Event Information collected throughout the study | Prior to and following administration of investigational product on the clinic visit with AEs followed for 7 days after by telephone contact | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline of Fasciculation Frequency | Fasciculations over time measured by EMG before and after treatment | Prior to and twice within 4 hours following administration of investigational product on the clinic visit | 
| Change from Baseline of Fasciculation Frequency | Fasciculations over time measured by ultrasound before and after treatment | Prior to and twice within 4 hours following administration of investigational product on the clinic visit | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ALS, Amyotrophic Lateral Sclerosis, Fasciculation, FLX-787