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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

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ClinicalTrials.gov Identifier: NCT03338114
Recruitment Status : Withdrawn (Sponsor Decision)
First Posted : November 9, 2017
Last Update Posted : January 23, 2018
Sponsor:
Information provided by (Responsible Party):
Flex Pharma, Inc.

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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Fasciculation Drug: FLX-787-ODT (orally disintigrating tablet) Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Estimated Study Start Date : November 2017
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : August 2018


Arm Intervention/treatment
Experimental: FLX-787-ODT (orally disintigrating tablet)
FLX-787-ODT (orally disintigrating tablet)
Drug: FLX-787-ODT (orally disintigrating tablet)
Oral Disintegrating Tablet




Primary Outcome Measures :
  1. Change from Baseline of Diastolic Blood Pressure in mmHg [ Time Frame: Prior to and within 3 hours following administration of investigational product on the single clinic visit ]
    Diastolic blood pressure collected before and after treatment

  2. Change from Baseline of Systolic Blood Pressure in mmHg [ Time Frame: Prior to and within 3 hours following administration of investigational product on the single clinic visit ]
    Systolic blood pressure collected before and after treatment

  3. Change from Baseline in Heart Rate in beats per minute [ Time Frame: Prior to and within 3 hours following administration of investigational product on the single clinic visit ]
    Heart rate collected before and after treatment

  4. Change from Baseline in Respiration Rate in breaths per minute [ Time Frame: Prior to and within 3 hours following administration of investigational product on the single clinic visit ]
    Respiration rate collected before and after treatment

  5. Change from Baseline in Body Temperature in degrees Celsius or Fahrenheit [ Time Frame: Prior to and within 3 hours following administration of investigational product on the single clinic visit ]
    Body temperature collected before and after treatment

  6. Change from Baseline of Oral Cavity Examination [ Time Frame: Prior to and twice within 4 hours following administration of investigational product on the clinic visit ]
    Oral Cavity Examination performed before and after treatment

  7. Incidence of Treatment-Emergent Adverse Events [ Time Frame: 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


Secondary Outcome Measures :
  1. Change from Baseline of Fasciculation Frequency [ Time Frame: 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

  2. Change from Baseline of Fasciculation Frequency [ Time Frame: Prior to and twice within 4 hours following administration of investigational product on the clinic visit ]
    Fasciculations over time measured by ultrasound before and after treatment



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03338114


Locations
United States, North Carolina
Wake Forest University Health Sciences
Winston-Salem, North Carolina, United States, 27157
Sponsors and Collaborators
Flex Pharma, Inc.

Responsible Party: Flex Pharma, Inc.
ClinicalTrials.gov Identifier: NCT03338114     History of Changes
Other Study ID Numbers: FLX-787-106
First Posted: November 9, 2017    Key Record Dates
Last Update Posted: January 23, 2018
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Flex Pharma, Inc.:
ALS
Amyotrophic Lateral Sclerosis
Fasciculation
FLX-787

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Fasciculation
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Signs and Symptoms