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Study to Evaluate Safety & Efficacy of FLX-787-ODT to Treat Fasciculations in Tongue and Appendicular Muscle in Adult Subjects With ALS

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ClinicalTrials.gov Identifier: NCT03334786
Recruitment Status : Recruiting
First Posted : November 7, 2017
Last Update Posted : April 11, 2018
Sponsor:
Information provided by (Responsible Party):
Flex Pharma, Inc.

Brief Summary:
The FLX-787-107 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, if tongue and muscle strength, speech, and swallowing are affected, 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 Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 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 One Appendicular Muscle in Adult Subjects With Amyotrophic Lateral Sclerosis (ALS)
Actual Study Start Date : April 5, 2018
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : August 2018


Arm Intervention/treatment
Experimental: FLX-787-ODT (orally disintegrating tablet)
Single dose
Drug: FLX-787-ODT
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 ultrasound 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 EMG before and after treatment

  3. Change from Baseline in Peak Tongue Strength in kPa by Iowa Oral Performance Instrument [ Time Frame: Prior to and once within 4 hours following administration of investigational product on the clinic visit ]
    Peak Tongue Strength Measurements before and after treatment

  4. Change from Baseline in Speech Assessments [ Time Frame: Prior to and once within 4 hours following administration of investigational product on the clinic visit ]
    Timed Speech Assessments before and after treatment

  5. Change from Baseline in Swallowing Assessments [ Time Frame: Prior to and once within 4 hours following administration of investigational product on the clinic visit ]
    Timed Swallowing Assessments before and after treatment



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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 5 years.
  • Greater than 6 fasciculations per minute noted at least in the tongue by clinical, ultrasound, or EMG evaluation.
  • Normal oral cavity exam at screening.

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.
  • 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.
  • 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): NCT03334786


Contacts
Contact: David Golod, PhD 617-721-1988 dgolod@flex-pharma.com

Locations
United States, Massachusetts
Beth Israel Deaconess Medical Center Recruiting
Boston, Massachusetts, United States, 02215
Contact: Hilda Guttierez    617-667-8130    hgutier1@bidmc.harvard.edu   
Contact: Maria Martucci    617-667-8130    mmartucc@bidmc.harvard.edu   
Sponsors and Collaborators
Flex Pharma, Inc.

Responsible Party: Flex Pharma, Inc.
ClinicalTrials.gov Identifier: NCT03334786     History of Changes
Other Study ID Numbers: FLX-787-107
First Posted: November 7, 2017    Key Record Dates
Last Update Posted: April 11, 2018
Last Verified: April 2018
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