ALSPhase 2NCT03457753
Riluzole Oral Soluble Film Safety and Tolerability in Amyotrophic Lateral Sclerosis
A Phase 2 study testing Riluzole Oral Soluble Film in people with ALS, run by Aquestive Therapeutics at 1 site in United States.
Trial summary
The primary objective of this study is to assess the safety and tolerability, with emphasis on the oral cavity, of ROSF (containing riluzole 50mg) in subjects with amyotrophic lateral sclerosis (ALS) administered twice daily for 12 weeks. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- ALS
- What is being tested?
- Riluzole Oral Soluble Film
- 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
- 1 Mar 2018to 27 Mar 2018
- Phase
- Phase 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female subjects, between 18-80 years of age, inclusive.
- Subjects having a diagnosis of probable or definite ALS in accordance with the Revisited El-Escorial Criteria.
- Subjects must have no known allergy to riluzole or inactive ingredients* in ROSF.
- Subjects or subject's legally authorized representative must be willing and able to complete informed consent/assent and HIPAA authorization.
- Ability to comprehend and be informed of the nature of the study, as assessed by the Primary or Sub-Investigator.
- Subjects prescribed to take riluzole at or before the time of first dose. (The study is open to subjects currently taking riluzole at screening, subjects who are not currently taking riluzole at screening but who have taken riluzole in the past, and subjects to be newly started on riluzole (given as ROSF in the course of this study).
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- Availability to volunteer for the entire study duration and willing to adhere to all protocol requirements.
- Female subjects of childbearing potential must have a negative urine pregnancy test at Screening and Visit 1-3. Female subjects of childbearing potential (i.e. not surgically sterile, not 2 years postmenopausal, or not with a sterile partner) must have a negative pregnancy test at screening and Visit 1-3, agree to abstinence, practicing double barrier contraception or using an FDA approved barrier method contraceptive (e.g., licensed hormonal or barrier methods) for greater than 2 months prior to screening visit and commit to an acceptable form of birth control for the duration of the study and for 30 days after participation in the study.
- Subjects, in the judgment of the investigator, must be suitable candidates for administration of ROSF (riluzole oral soluble film).
Who cannot join
- Subjects with a history of clinically significant liver disease, renal disease, or any other medical condition judged to be exclusionary by the investigator.
- Subjects who are unwilling to sign informed consent or subjects who for any other reason in the judgment of investigator are unable to complete the study.
- Female subjects who have a positive urine pregnancy test (βhCG) at screening or visit 1, are trying to become pregnant or are breastfeeding.
- Subjects with active cancer within the previous 2 years, except treated basal cell carcinoma of the skin.
- Subjects who have taken any experimental drug within 30 days prior to enrollment or within 5 half-lives of the investigational drug -whichever is the longer period. However, subjects who have previously completed other Aquestive sponsored ROSF clinical studies within the last 30 days prior to enrollment may be eligible for consideration for entry into this study.
- Subjects with known history or presence of moderate or severe renal impairment as defined by a calculated creatinine clearance of ≤50 mL/minute.
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- Subjects currently taking riluzole with alanine aminotransferase (ALT) levels greater than 5 times upper limit of normal or with evidence of clinical jaundice. (Riluzole should be discontinued in these patients.)
- Subjects who will be receiving riluzole for the first time who exhibit baseline elevations of several liver function tests (especially elevated bilirubin). (These findings at baseline should preclude the use of riluzole including ROSF.)
- Use of potentially hepatotoxic drugs: (e.g., allopurinol, methyldopa, sulfasalazine).
- Subjects with clinically significant abnormal laboratory values in the judgment of the investigator.
- Use of strong or moderate CYP1A2 inhibitors (e.g., ciprofloxacin, enoxacin, fluvoxamine, methoxsalen, mexiletine, thiabendazole, vemurafenib, zileuton) and CYP1A2 inducers (e.g. rifampin and barbiturates) in the previous 30 days before first drug administration.
- Employee or immediate relative of an employee of the investigator, MonoSol Rx LLC, any of its affiliates or partners, or inVentiv Health.
- Anything else that, in the opinion of the investigator, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Multi-Center, Open Label Study to Assess the Safety and Tolerability of Riluzole Oral Soluble Film in Subjects With Amyotrophic Lateral Sclerosis Over 12 Weeks of Twice Daily Treatment.
The primary objective of this study is to assess the safety and tolerability, with emphasis on the oral cavity, of ROSF (containing riluzole 50mg) in subjects with amyotrophic lateral sclerosis (ALS) administered twice daily for 12 weeks. Secondary objectives include (1) to record the subject's assessment of any difficulty taking riluzole administered as ROSF and any difficulty taking riluzole in the tablet formulation and (2) to record the relative preference, if any, of subjects and caretakers, for riluzole administered as ROSF vs. the riluzole tablet.
Read the detailed description
The total study participant time is expected to be approximately 14 weeks from time of screening to completion of study. Subjects will be instructed on the use of ROSF and receive the first dose of ROSF under supervision of the investigator during Visit 1.Subjects will then continue on ROSF 50mg twice daily for 12 weeks.
- Conditions
- ALS
- Being tested
- Riluzole Oral Soluble Film
- Keywords
- Amyotrophic Lateral Sclerosis
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Riluzole Oral Soluble FilmDrug
Also called ROSF
Riluzole Oral Soluble Film (ROSF) containing Riluzole 50mg .
| Study group | Role | What they receive |
|---|---|---|
| Subjects with ALS | Experimental | Riluzole Oral Soluble Film (ROSF) 50 mg will be administered in subjects with ALS twice daily. It is intended that at least five (5) of the twenty-five (25) subjects enrolled will be subjects scoring greater than 20 on the Eating Assessment Tool (EAT-10) (representative of ALS patients reporting moderate swallowing impairments in a patient report validated scale). Receives: Drug: Riluzole Oral Soluble Film |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in NCI-CTC score at week 12
The National Cancer Institute Common Toxicity Criteria (NCI-CTC) score will be used to evaluate the presence or level of oral cavity irritation on an oral examination, with a grading scale ranging from Grade 0 -5 (0 = no toxicity, 1 = painless ulcers, erythema, or mild soreness, 2 = painful erythema, edema, or ulcers but eating or swallowing possible, 3 = painful erythema, edema, or ulcers requiring intravenous hydration, 4 = severe ulceration, and 5 = death related to toxicity).
Measured: Week 12 (visit 3)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2018
- Study start1 Mar 2018
- Primary completion27 Mar 2018
- Study completion27 Mar 2018
Why it stopped: The Sponsor stopped the study due to a change in study plans
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALSStudies 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 2
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a multi-center, open label safety and tolerability trial. Approximately twenty-five (25) individuals with amyotrophic lateral sclerosis (ALS) will be enrolled.
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aquestive Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Inventiv Health, Covance
- Responsible party
- Sponsor
- Investigators
- Emily Plowman, PhD, CCC-SLP, Principal investigator, University of FloridaJames Wymer, MD, PhD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03457753
- Sponsor's protocol ID
- 17MO1R-0016
- Status last verified
- May 2019
- First submitted
- 26 Feb 2018
- Last updated
- 13 May 2019
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Open Label Study to Assess the Safety and Tolerability of Riluzole Oral Soluble Film in Subjects With Amyotrophic Lateral Sclerosis Over 12 Weeks of Twice Daily Treatment.
- ClinicalTrials.gov ID
- NCT03457753
- Acronym / sponsor ID
- 17MO1R-0016
- Conditions
- ALS
- Keywords
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The Sponsor stopped the study due to a change in study plans
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Aquestive TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Inventiv Health, Covance
- Study start
- 1 Mar 2018 (actual)
- Primary completion
- 27 Mar 2018 (actual)
- Study completion
- 27 Mar 2018 (actual)
- First posted
- 8 Mar 2018 (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 primary objective of this study is to assess the safety and tolerability, with emphasis on the oral cavity, of ROSF (containing riluzole 50mg) in subjects with amyotrophic lateral sclerosis (ALS) administered twice daily for 12 weeks. Secondary objectives include (1) to record the subject's assessment of any difficulty taking riluzole administered as ROSF and any difficulty taking riluzole in the tablet formulation and (2) to record the relative preference, if any, of subjects and caretakers, for riluzole administered as ROSF vs. the riluzole tablet.
Detailed description
The total study participant time is expected to be approximately 14 weeks from time of screening to completion of study. Subjects will be instructed on the use of ROSF and receive the first dose of ROSF under supervision of the investigator during Visit 1.Subjects will then continue on ROSF 50mg twice daily for 12 weeks.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a multi-center, open label safety and tolerability trial. Approximately twenty-five (25) individuals with amyotrophic lateral sclerosis (ALS) will be enrolled.
| Arm | Type | Receives |
|---|---|---|
| Subjects with ALS | Experimental | Drug: Riluzole Oral Soluble FilmRiluzole Oral Soluble Film (ROSF) 50 mg will be administered in subjects with ALS twice daily. It is intended that at least five (5) of the twenty-five (25) subjects enrolled will be subjects scoring greater than 20 on the Eating Assessment Tool (EAT-10) (representative of ALS patients reporting moderate swallowing impairments in a patient report validated scale). |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Male or female subjects, between 18-80 years of age, inclusive.
- Subjects having a diagnosis of probable or definite ALS in accordance with the Revisited El-Escorial Criteria.
- Subjects must have no known allergy to riluzole or inactive ingredients* in ROSF.
- Subjects or subject's legally authorized representative must be willing and able to complete informed consent/assent and HIPAA authorization.
- Ability to comprehend and be informed of the nature of the study, as assessed by the Primary or Sub-Investigator.
Key exclusion criteria (13 listed)
- Subjects with a history of clinically significant liver disease, renal disease, or any other medical condition judged to be exclusionary by the investigator.
- Subjects who are unwilling to sign informed consent or subjects who for any other reason in the judgment of investigator are unable to complete the study.
- Female subjects who have a positive urine pregnancy test (βhCG) at screening or visit 1, are trying to become pregnant or are breastfeeding.
- Subjects with active cancer within the previous 2 years, except treated basal cell carcinoma of the skin.
- Subjects who have taken any experimental drug within 30 days prior to enrollment or within 5 half-lives of the investigational drug -whichever is the longer period. However, subjects who have previously completed other Aquestive sponsored ROSF clinical studies within the last 30 days prior to enrollment may be eligible for consideration for entry into this study.
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 |
|---|---|---|
| Riluzole Oral Soluble FilmAlso: ROSFRiluzole Oral Soluble Film (ROSF) containing Riluzole 50mg . | Drug | Subjects with ALS |
Primary outcome
Change from baseline in NCI-CTC score at week 12
Time frame: Week 12 (visit 3)
The National Cancer Institute Common Toxicity Criteria (NCI-CTC) score will be used to evaluate the presence or level of oral cavity irritation on an oral examination, with a grading scale ranging from Grade 0 -5 (0 = no toxicity, 1 = painless ulcers, erythema, or mild soreness, 2 = painful erythema, edema, or ulcers but eating or swallowing possible, 3 = painful erythema, edema, or ulcers requiring intravenous hydration, 4 = severe ulceration, and 5 = death related to toxicity).
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 13 May 2019.
- Individual participant dataSharing planned: No
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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.
Emily Plowman, PhD, CCC-SLP principal investigator
University of Florida
James Wymer, MD, PhD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Riluzole Oral Soluble Film in people with ALS, run by Aquestive Therapeutics 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 9 inclusion and 13 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 13 May 2019. 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 phase mean?
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 in NCI-CTC score at week 12 (time frame: Week 12 (visit 3)). 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 13 May 2019; AzTrials last refreshed its copy on 8 Nov 2026.