Amyotrophic Lateral SclerosisPhase 1/2NCT03338114
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
A Phase 1/2 study testing FLX-787-ODT (orally disintigrating tablet) in people with Amyotrophic Lateral Sclerosis and Fasciculation, run by Flex Pharma, Inc. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Fasciculation
- What is being tested?
- FLX-787-ODT (orally disintigrating tablet)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2017to Aug 2018
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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).
Trial locations and contacts
1 site in 1 country.
United States1 site
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157
About this study
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)
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.
- Conditions
- Amyotrophic Lateral Sclerosis, Fasciculation
- Being tested
- FLX-787-ODT (orally disintigrating tablet)
- Keywords
- ALS, Amyotrophic Lateral Sclerosis, Fasciculation, FLX-787
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Fasciculation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FLX-787-ODT (orally disintigrating tablet)Drug
Oral Disintegrating Tablet
| Study group | Role | What they receive |
|---|---|---|
| FLX-787-ODT (orally disintigrating tablet) | Experimental | FLX-787-ODT (orally disintigrating tablet) Receives: Drug: FLX-787-ODT (orally disintigrating tablet) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline of Diastolic Blood Pressure in mmHg
Diastolic blood pressure collected before and after treatment
Measured: 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
Systolic blood pressure collected before and after treatment
Measured: 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
Heart rate collected before and after treatment
Measured: 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
Respiration rate collected before and after treatment
Measured: 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
Body temperature collected before and after treatment
Measured: Prior to and within 3 hours following administration of investigational product on the single clinic visit
Change from Baseline of Oral Cavity Examination
Oral Cavity Examination performed before and after treatment
Measured: Prior to and twice within 4 hours following administration of investigational product on the clinic visit
Incidence of Treatment-Emergent Adverse Events
Adverse Event Information collected throughout the study
Measured: Prior to and following administration of investigational product on the clinic visit with AEs followed for 7 days after by telephone contact
Secondary outcomes (2)
Change from Baseline of Fasciculation Frequency
Fasciculations over time measured by EMG before and after treatment
Measured: Prior to and twice within 4 hours following administration of investigational product on the clinic visit
Change from Baseline of Fasciculation Frequency
Fasciculations over time measured by ultrasound before and after treatment
Measured: Prior to and twice within 4 hours following administration of investigational product on the clinic visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Nov 2017
- Study startNov 2017 (estimated)
- Primary completionAug 2018 (estimated)
- Study completionAug 2018 (estimated)
Why it stopped: Sponsor Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Flex Pharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03338114
- Sponsor's protocol ID
- FLX-787-106
- Status last verified
- Nov 2017
- First submitted
- 6 Nov 2017
- Last updated
- 23 Jan 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT03338114
- Acronym / sponsor ID
- FLX-787-106
- Conditions
- Amyotrophic Lateral Sclerosis, Fasciculation
- Keywords
- ALS, Amyotrophic Lateral Sclerosis, Fasciculation, FLX-787
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Sponsor Decision
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Flex Pharma, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2017 (estimated)
- Primary completion
- Aug 2018 (estimated)
- Study completion
- Aug 2018 (estimated)
- First posted
- 9 Nov 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FLX-787-ODT (orally disintigrating tablet) | Experimental | Drug: FLX-787-ODT (orally disintigrating tablet)FLX-787-ODT (orally disintigrating tablet) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FLX-787-ODT (orally disintigrating tablet)Oral Disintegrating Tablet | Drug | FLX-787-ODT (orally disintigrating tablet) |
Primary outcome
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
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
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
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
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
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
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
2 secondary outcomes
Change from Baseline of Fasciculation Frequency
Time frame: 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
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2018.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing FLX-787-ODT (orally disintigrating tablet) in people with Amyotrophic Lateral Sclerosis and Fasciculation, run by Flex Pharma, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 23 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jan 2018.