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

August 27, 2018 updated by: Flex Pharma, Inc.

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)

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
3

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Beth Israel Deaconess Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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 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).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: FLX-787-ODT (orally disintegrating tablet)
Single dose
Drug: FLX-787-ODT
Oral Disintegrating Tablet

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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
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
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
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
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
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
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
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
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
Prior to and within 3 hours following administration of investigational product on the single clinic visit
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
Prior to and twice within 4 hours following administration of investigational product on the clinic visit
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
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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
Prior to and twice within 4 hours following administration of investigational product on the clinic visit
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
Prior to and twice within 4 hours following administration of investigational product on the clinic visit
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
Prior to and once within 4 hours following administration of investigational product on the clinic visit
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
Prior to and once within 4 hours following administration of investigational product on the clinic visit
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
Prior to and once within 4 hours following administration of investigational product on the clinic visit

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Flex Pharma, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 5, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 23, 2018

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 23, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 26, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 6, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 7, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 29, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 27, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • FLX-787-107

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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