Back to resultsStudy to Evaluate Safety & Efficacy of FLX-787-ODT to Treat Fasciculations in Tongue and Appendicular Muscle in Adult Subjects With ALS
Primary Purpose
Amyotrophic Lateral Sclerosis, Fasciculation
Status
Terminated
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
FLX-787-ODT
Sponsored by
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Amyotrophic Lateral Sclerosis, Fasciculation, FLX-787
Eligibility 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).
Sites / Locations
- Beth Israel Deaconess Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
FLX-787-ODT (orally disintegrating tablet)
Arm Description
Single dose
Outcomes
Primary Outcome Measures
Change from Baseline of Diastolic Blood Pressure in mmHg
Diastolic blood pressure collected before and after treatment
Change from Baseline of Systolic Blood Pressure in mmHg
Systolic blood pressure collected before and after treatment
Change from Baseline in Heart Rate in beats per minute
Heart rate collected before and after treatment
Change from Baseline in Respiration Rate in breaths per minute
Respiration rate collected before and after treatment
Change from Baseline in Body Temperature in degrees Celsius or Fahrenheit
Body temperature collected before and after treatment
Change from Baseline of Oral Cavity Examination
Oral Cavity Examination performed before and after treatment
Incidence of Treatment-Emergent Adverse Events
Adverse Event Information collected throughout the study
Secondary Outcome Measures
Change from Baseline of Fasciculation Frequency
Fasciculations over time measured by ultrasound before and after treatment
Change from Baseline of Fasciculation Frequency
Fasciculations over time measured by EMG before and after treatment
Change from Baseline in Peak Tongue Strength in kPa by Iowa Oral Performance Instrument
Peak Tongue Strength Measurements before and after treatment
Change from Baseline in Speech Assessments
Timed Speech Assessments before and after treatment
Change from Baseline in Swallowing Assessments
Timed Swallowing Assessments before and after treatment
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03334786
Brief Title
Study to Evaluate Safety & Efficacy of FLX-787-ODT to Treat Fasciculations in Tongue and Appendicular Muscle in Adult Subjects With ALS
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)
Study Type
Interventional
2. Study Status
Record Verification Date
April 2018
Overall Recruitment Status
Terminated
Why Stopped
Development of Current Formulation has Ceased
Study Start Date
April 5, 2018 (Actual)
Primary Completion Date
August 23, 2018 (Actual)
Study Completion Date
August 23, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Flex Pharma, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis, Fasciculation
Keywords
ALS, Amyotrophic Lateral Sclerosis, Fasciculation, FLX-787
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
FLX-787-ODT (orally disintegrating tablet)
Arm Type
Experimental
Arm Description
Single dose
Intervention Type
Drug
Intervention Name(s)
FLX-787-ODT
Intervention Description
Oral Disintegrating Tablet
Primary Outcome Measure Information:
Title
Change from Baseline of Diastolic Blood Pressure in mmHg
Description
Diastolic blood pressure collected before and after treatment
Time Frame
Prior to and within 3 hours following administration of investigational product on the single clinic visit
Title
Change from Baseline of Systolic Blood Pressure in mmHg
Description
Systolic blood pressure collected before and after treatment
Time Frame
Prior to and within 3 hours following administration of investigational product on the single clinic visit
Title
Change from Baseline in Heart Rate in beats per minute
Description
Heart rate collected before and after treatment
Time Frame
Prior to and within 3 hours following administration of investigational product on the single clinic visit
Title
Change from Baseline in Respiration Rate in breaths per minute
Description
Respiration rate collected before and after treatment
Time Frame
Prior to and within 3 hours following administration of investigational product on the single clinic visit
Title
Change from Baseline in Body Temperature in degrees Celsius or Fahrenheit
Description
Body temperature collected before and after treatment
Time Frame
Prior to and within 3 hours following administration of investigational product on the single clinic visit
Title
Change from Baseline of Oral Cavity Examination
Description
Oral Cavity Examination performed before and after treatment
Time Frame
Prior to and twice within 4 hours following administration of investigational product on the clinic visit
Title
Incidence of Treatment-Emergent Adverse Events
Description
Adverse Event Information collected throughout the study
Time Frame
Prior to and following administration of investigational product on the clinic visit with AEs followed for 7 days after by telephone contact
Secondary Outcome Measure Information:
Title
Change from Baseline of Fasciculation Frequency
Description
Fasciculations over time measured by ultrasound before and after treatment
Time Frame
Prior to and twice within 4 hours following administration of investigational product on the clinic visit
Title
Change from Baseline of Fasciculation Frequency
Description
Fasciculations over time measured by EMG before and after treatment
Time Frame
Prior to and twice within 4 hours following administration of investigational product on the clinic visit
Title
Change from Baseline in Peak Tongue Strength in kPa by Iowa Oral Performance Instrument
Description
Peak Tongue Strength Measurements before and after treatment
Time Frame
Prior to and once within 4 hours following administration of investigational product on the clinic visit
Title
Change from Baseline in Speech Assessments
Description
Timed Speech Assessments before and after treatment
Time Frame
Prior to and once within 4 hours following administration of investigational product on the clinic visit
Title
Change from Baseline in Swallowing Assessments
Description
Timed Swallowing Assessments before and after treatment
Time Frame
Prior to and once within 4 hours following administration of investigational product on the clinic visit
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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).
Facility Information:
Facility Name
Beth Israel Deaconess Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
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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