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

NCT03338114 | Withdrawn Phase 1 FDA Drug

• 1 other identifier: FLX-787-106
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis; Fasciculation

Keywords

ALS; Amyotrophic Lateral Sclerosis; Fasciculation; FLX-787

Outcome Measures

Primary Outcomes (7)

• 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 Outcomes (2)

• Change from Baseline of Fasciculation Frequency

  Prior to and twice within 4 hours following administration of investigational product on the clinic visit

• Change from Baseline of Fasciculation Frequency

  Prior to and twice within 4 hours following administration of investigational product on the clinic visit

Study Arms (1)

FLX-787-ODT (orally disintigrating tablet)

EXPERIMENTAL

FLX-787-ODT (orally disintigrating tablet)

Drug: FLX-787-ODT (orally disintigrating tablet)

Interventions

FLX-787-ODT (orally disintigrating tablet) DRUG

Oral Disintegrating Tablet

FLX-787-ODT (orally disintigrating tablet)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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).

Sponsors & Collaborators

• Flex Pharma, Inc.: lead

Study Sites (1)

Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Fasciculation

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 6, 2017
• First Posted: November 9, 2017
• Study Start: November 1, 2017
• Primary Completion: August 1, 2018
• Study Completion: August 1, 2018
• Last Updated: January 23, 2018

Record last verified: 2017-11

Locations

US (1)