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

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)

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.

Study Overview

• Status: Withdrawn
• Conditions: Amyotrophic Lateral Sclerosis; Fasciculation
• Intervention / Treatment: Drug: FLX-787-ODT (orally disintigrating tablet)

• Study Type: Interventional
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27157
      • Wake Forest University Health Sciences

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: FLX-787-ODT (orally disintigrating tablet)	Drug: FLX-787-ODT (orally disintigrating tablet); Oral Disintegrating Tablet

What is the study measuring?

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 ultrasound 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 EMG before and after treatment	Prior to and twice within 4 hours following administration of investigational product on the clinic visit

Collaborators and Investigators

• Sponsor: Flex Pharma, Inc.

Study record dates

Study Major Dates

• Study Start (Anticipated): November 1, 2017
• Primary Completion (Anticipated): August 1, 2018
• Study Completion (Anticipated): August 1, 2018

Study Registration Dates

• First Submitted: November 6, 2017
• First Submitted That Met QC Criteria: November 6, 2017
• First Posted (Actual): November 9, 2017

Study Record Updates

• Last Update Posted (Actual): January 23, 2018
• Last Update Submitted That Met QC Criteria: January 18, 2018
• Last Verified: November 1, 2017

More Information

Terms related to this study

• Keywords: Amyotrophic Lateral Sclerosis; ALS; Fasciculation; FLX-787
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Fasciculation
• Other Study ID Numbers: FLX-787-106

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No