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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

Primary Purpose

Amyotrophic Lateral Sclerosis, Fasciculation

Status

Withdrawn

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

FLX-787-ODT (orally disintigrating tablet)

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

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

Wake Forest University Health Sciences

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

FLX-787-ODT (orally disintigrating tablet)

Arm Description

FLX-787-ODT (orally disintigrating tablet)

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 EMG before and after treatment

Change from Baseline of Fasciculation Frequency

Fasciculations over time measured by ultrasound before and after treatment

Full Information

NCT ID

NCT03338114

First Posted

November 6, 2017

Last Updated

January 18, 2018

Sponsor

Flex Pharma, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT03338114

Brief Title

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

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)

Study Type

Interventional

2. Study Status

Record Verification Date

November 2017

Overall Recruitment Status

Withdrawn

Why Stopped

Sponsor Decision

Study Start Date

November 2017 (Anticipated)

Primary Completion Date

August 2018 (Anticipated)

Study Completion Date

August 2018 (Anticipated)

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

Data Monitoring Committee

5. Study Description

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.

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

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

FLX-787-ODT (orally disintigrating tablet)

Arm Type

Experimental

Arm Description

FLX-787-ODT (orally disintigrating tablet)

Intervention Type

Drug

Intervention Name(s)

FLX-787-ODT (orally disintigrating tablet)

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 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 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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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).

Facility Information:

Facility Name

Wake Forest University Health Sciences

City

Winston-Salem

State/Province

North Carolina

ZIP/Postal Code

27157

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Undecided

Learn more about this trial

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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