Amyotrophic Lateral SclerosisPhase 2ROSFNCT03679975
Riluzole Oral Soluble Film (ROSF) Swallowing Safety in Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing Riluzole Oral Soluble film (ROSF) 50 mg in people with Amyotrophic Lateral Sclerosis, run by Aquestive Therapeutics at 1 site in United States.
Trial summary
The primary objective is to evaluate the effect, if any, of a single 50 mg dose of Riluzole Oral Soluble Film (ROSF) on swallowing safety in individuals with amyotrophic lateral sclerosis. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Riluzole Oral Soluble film (ROSF) 50 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Apr 2018to 21 Dec 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 with a diagnosis of probable or definite ALS in accordance with the Revised El-Escorial Criteria
- Subjects must be currently on an oral diet and able to take foods and liquids by mouth equivalent to a score of 3 or above on the Functional Oral Intake Scale.
- Subjects with no known allergy to barium, riluzole or inactive ingredients in ROSF
- Subject or subject's legally authorized representative must be willing and able to give informed consent/assent and HIPAA authorization.
- Subject must have the ability to comprehend and be informed of the nature of the study, as assessed by the Principal Investigator or Sub-Investigator.
Show all 9Show fewer
- Subjects prescribed riluzole at or before the dose of study drug. (The study was open to subjects currently taking riluzole at screening, subjects who were not currently taking riluzole at screening who had taken riluzole in the past, and subjects to be newly started on riluzole (given as ROSF in this study).
- Availability to volunteer for the entire study duration and willing to adhere to all protocol requirements
- 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, agree to abstinence, be practicing double barrier contraception or using an FDA approved barrier method contraceptive (e.g., licensed hormonal or barrier methods) for more than 2 months prior to the 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.
Who cannot join
- Subjects who score 2 or below on the Functional Oral Intake Scale.
- Subjects with a prior swallowing study that has shown a Penetration Aspiration Scale score of 3 or greater
- Subjects with a history of 2 or more episodes of aspiration pneumonia requiring hospitalization
- 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 were unwilling to sign informed consent or subjects who for any other reason in the judgment of investigator were unable to complete the study
- Female subjects who had a positive urine pregnancy test (βhCG) at screening or Visit 1, were trying to become pregnant, or were breastfeeding.
Show all 16Show fewer
- Subjects with active cancer within the previous 2 years, except treated basal cell carcinoma of the skin
- Subjects who had taken any experimental drug within 30 days prior to enrollment or within 5 half-lives of the investigational drug -whichever was the longer period. However, subjects who had previously completed other Aquestive Therapeutics-sponsored ROSF clinical studies within the last 30 days prior to enrollment could be eligible for consideration for entry into this study.
- Subjects with known history of moderate or severe renal impairment as defined by a calculated creatinine clearance of ≤50 mL/minute
- Subjects currently taking riluzole with alanine aminotransferase levels greater than 5 times upper limit of normal or with evidence of clinical jaundice. (Riluzole should be discontinued in these patients.)
- Subjects who would be receiving riluzole for the first time who exhibited 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.
- 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.
- Employee or immediate relative of an employee of the investigator, Aquestive Therapeutics, any of its affiliates or partners, or inVentiv Health.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Florida Center for Movement Disorders & Neuroscience
Gainesville, Florida, 32607
About this study
Official title: A Single Center Study to Evaluate the Effect of Riluzole Oral Soluble Film on Swallowing Safety in Individuals With Amyotrophic Lateral Sclerosis
The primary objective is to evaluate the effect, if any, of a single 50 mg dose of Riluzole Oral Soluble Film (ROSF) on swallowing safety in individuals with amyotrophic lateral sclerosis.
Read the detailed description
This was a single site, single dose, open-label study with one group of male or female patients between 18-80 years old with a diagnosis of Probable or Definite Amyotrophic Lateral Sclerosis (ALS) according to revised El-Escorial Criteria. Patients seen in the ALS Clinic at the University of Florida were screened to determine eligibility for participation in the study according to the specified inclusion/exclusion criteria. Following informed consent and enrollment, subjects were given a complete physical and neurological examination including the validated Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). Swallowing safety was assessed using the gold standard Videofluoroscopic Swallowing Study (VFSS) to allow direct visualization of the swallowing process and any episodes of penetration or aspiration were quantified using the validated Penetration Aspiration Scale (PAS). Subjects participated in a standardized protocol that includes 11 bolus stimuli presentations of different liquid and food materials to test swallowing function using a progression of textures and materials. Immediately following this assessment, subjects were given a single dose of ROSF 50 mg, placed on the median lingual sulcus of the dorsum of the tongue (area under the tongue on the midline groove that separates the surface of the tongue into right and left halves). Three minutes after the administration of ROSF, the identical standardized VFSS protocol was re-administered to allow a comparison of swallowing safety (using the PAS scale) pre- versus post-ROSF administration.
The original plan was to enroll 30 patients for a final sample size of 25 completed patients. The protocol stipulated that, depending upon the study enrollment rate, the Sponsor could elect to perform an interim analysis using available data from the subset of subjects who had completed the study at that time. An interim assessment of the data was performed after 8 subjects had completed the study and a ninth patient had been screened and was awaiting VFSS. With agreement from the FDA, the Sponsor decided to terminate the study after 9 completed patients, based on the analysis of the first 8 completed patients showing no evidence of a harmful effect of one dose of ROSF 50 mg on swallowing function.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Riluzole Oral Soluble film (ROSF) 50 mg
- 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 film (ROSF) 50 mgDrug
Also called ROSF, Riluzole Orodispersible Film (ROF)
Enrolled subjects underwent an instrumental evaluation of swallowing safety before and after administration of a single dose of the ROSF formulation containing 50 mg of riluzole.
| Study group | Role | What they receive |
|---|---|---|
| Subjects with ALS | Experimental | Subjects with a diagnosis of probable or definite ALS in accordance with the Revised El-Escorial Criteria were administered a single dose of the Riluzole Oral Soluble Film (ROSF) 50 mg. Receives: Drug: Riluzole Oral Soluble film (ROSF) 50 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Categorized by Differences From Baseline in Penetration Aspiration Scale (PAS) - Sum of Scores Method
Swallowing safety was assessed using the gold standard Videofluoroscopic Swallowing Study (VFSS) to allow direct visualization of the swallowing process and any episodes of penetration or aspiration were quantified using the validated Penetration Aspiration Scale (PAS). The PAS is an 8-point validated scale of swallowing safety that takes into account both degree/level of airway invasion during swallowing and the patients' response to the penetration or aspiration episode. The assessment is performed via videofluoroscope. Scores of 1 and 2 indicate airway safe swallowing with no airway invasion or ability to clear airway invasion. Scores 3 through 8 indicate airway unsafe swallowing based on penetration of the airway, level of penetration, and the patient's ability to respond. An increase in score/higher score means a worse outcome. A decrease in score/lower score means a better outcome.
Measured: Before and after administration of ROSF 50 mg on day 1 (visit 1)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2018
- Study start4 Apr 2018
- Primary completion26 Sep 2018
- Study completion21 Dec 2018
Why it stopped: The study is no longer required
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 2
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- The original plan was to enroll 30 patients for a final sample size of 25 completed patients. With agreement from the FDA, the Sponsor decided to terminate the study after 9 completed patients, based on an interim analysis of the first 8 completed patients with no evidence of a harmful effect of one dose of 50 mg Riluzole Oral Film on swallowing function.
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aquestive Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- inVentiv Health Clinical, Covance
- Responsible party
- Sponsor
- Investigators
- Cassie Jung, Study director, Aquestive Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03679975
- Sponsor's protocol ID
- 17MO1R-0012
- Acronym
- ROSF
- Status last verified
- Aug 2020
- First submitted
- 19 Sep 2018
- Last updated
- 18 Aug 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- 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 18 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Center Study to Evaluate the Effect of Riluzole Oral Soluble Film on Swallowing Safety in Individuals With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT03679975
- Acronym / sponsor ID
- ROSF / 17MO1R-0012
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study is no longer required
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Aquestive TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- inVentiv Health Clinical, Covance
- Study start
- 4 Apr 2018 (actual)
- Primary completion
- 26 Sep 2018 (actual)
- Study completion
- 21 Dec 2018 (actual)
- First posted
- 21 Sep 2018 (actual)
- Results
- Posted 10 Aug 2020
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 is to evaluate the effect, if any, of a single 50 mg dose of Riluzole Oral Soluble Film (ROSF) on swallowing safety in individuals with amyotrophic lateral sclerosis.
Detailed description
This was a single site, single dose, open-label study with one group of male or female patients between 18-80 years old with a diagnosis of Probable or Definite Amyotrophic Lateral Sclerosis (ALS) according to revised El-Escorial Criteria. Patients seen in the ALS Clinic at the University of Florida were screened to determine eligibility for participation in the study according to the specified inclusion/exclusion criteria. Following informed consent and enrollment, subjects were given a complete physical and neurological examination including the validated Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). Swallowing safety was assessed using the gold standard Videofluoroscopic Swallowing Study (VFSS) to allow direct visualization of the swallowing process and any episodes of penetration or aspiration were quantified using the validated Penetration Aspiration Scale (PAS). Subjects participated in a standardized protocol that includes 11 bolus stimuli presentations of different liquid and food materials to test swallowing function using a progression of textures and materials. Immediately following this assessment, subjects were given a single dose of ROSF 50 mg, placed on the median lingual sulcus of the dorsum of the tongue (area under the tongue on the midline groove that separates the surface of the tongue into right and left halves). Three minutes after the administration of ROSF, the identical standardized VFSS protocol was re-administered to allow a comparison of swallowing safety (using the PAS scale) pre- versus post-ROSF administration.
The original plan was to enroll 30 patients for a final sample size of 25 completed patients. The protocol stipulated that, depending upon the study enrollment rate, the Sponsor could elect to perform an interim analysis using available data from the subset of subjects who had completed the study at that time. An interim assessment of the data was performed after 8 subjects had completed the study and a ninth patient had been screened and was awaiting VFSS. With agreement from the FDA, the Sponsor decided to terminate the study after 9 completed patients, based on the analysis of the first 8 completed patients showing no evidence of a harmful effect of one dose of ROSF 50 mg on swallowing function.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The original plan was to enroll 30 patients for a final sample size of 25 completed patients. With agreement from the FDA, the Sponsor decided to terminate the study after 9 completed patients, based on an interim analysis of the first 8 completed patients with no evidence of a harmful effect of one dose of 50 mg Riluzole Oral Film on swallowing function.
| Arm | Type | Receives |
|---|---|---|
| Subjects with ALS | Experimental | Drug: Riluzole Oral Soluble film (ROSF) 50 mgSubjects with a diagnosis of probable or definite ALS in accordance with the Revised El-Escorial Criteria were administered a single dose of the Riluzole Oral Soluble Film (ROSF) 50 mg. |
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 with a diagnosis of probable or definite ALS in accordance with the Revised El-Escorial Criteria
- Subjects must be currently on an oral diet and able to take foods and liquids by mouth equivalent to a score of 3 or above on the Functional Oral Intake Scale.
- Subjects with no known allergy to barium, riluzole or inactive ingredients in ROSF
- Subject or subject's legally authorized representative must be willing and able to give informed consent/assent and HIPAA authorization.
Key exclusion criteria (16 listed)
- Subjects who score 2 or below on the Functional Oral Intake Scale.
- Subjects with a prior swallowing study that has shown a Penetration Aspiration Scale score of 3 or greater
- Subjects with a history of 2 or more episodes of aspiration pneumonia requiring hospitalization
- 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 were unwilling to sign informed consent or subjects who for any other reason in the judgment of investigator were unable to complete the 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 film (ROSF) 50 mgAlso: ROSF, Riluzole Orodispersible Film (ROF)Enrolled subjects underwent an instrumental evaluation of swallowing safety before and after administration of a single dose of the ROSF formulation containing 50 mg of riluzole. | Drug | Subjects with ALS |
Primary outcome
Number of Participants Categorized by Differences From Baseline in Penetration Aspiration Scale (PAS) - Sum of Scores Method
Time frame: Before and after administration of ROSF 50 mg on day 1 (visit 1)
Swallowing safety was assessed using the gold standard Videofluoroscopic Swallowing Study (VFSS) to allow direct visualization of the swallowing process and any episodes of penetration or aspiration were quantified using the validated Penetration Aspiration Scale (PAS). The PAS is an 8-point validated scale of swallowing safety that takes into account both degree/level of airway invasion during swallowing and the patients' response to the penetration or aspiration episode. The assessment is performed via videofluoroscope. Scores of 1 and 2 indicate airway safe swallowing with no airway invasion or ability to clear airway invasion. Scores 3 through 8 indicate airway unsafe swallowing based on penetration of the airway, level of penetration, and the patient's ability to respond. An increase in score/higher score means a worse outcome. A decrease in score/lower score means a better outcome.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Amyotrophic Lateral Sclerosis
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Clinical Outcome Assessment for AT & BCI
Spinal Cord Injury, ALS (Amyotrophic Lateral Sclerosis)
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.
Cassie Jung study director
Aquestive Therapeutics
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 (ROSF) 50 mg in people with Amyotrophic Lateral Sclerosis, 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 16 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: stopped early, as of the sponsor's last update on 18 Aug 2020. 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?
Yes. Results are posted in the registry (first posted 10 Aug 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants Categorized by Differences From Baseline in Penetration Aspiration Scale (PAS) - Sum of Scores Method (time frame: Before and after administration of ROSF 50 mg on day 1 (visit 1)). 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 18 Aug 2020.