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Trial record 3 of 7 for:    aquestive

Riluzole Oral Soluble Film (ROSF) Swallowing Safety in Amyotrophic Lateral Sclerosis (ALS) (ROSF)

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ClinicalTrials.gov Identifier: NCT03679975
Recruitment Status : Terminated (The study is no longer required)
First Posted : September 21, 2018
Results First Posted : August 10, 2020
Last Update Posted : August 18, 2020
Sponsor:
Collaborators:
inVentiv Health Clinical
Covance
Information provided by (Responsible Party):
Aquestive Therapeutics

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Other
Condition Amyotrophic Lateral Sclerosis
Intervention Drug: Riluzole Oral Soluble film (ROSF) 50 mg
Enrollment 9
Recruitment Details Eleven subjects were screened for the study: Two subjects withdrew after signing informed consent but prior to any study procedure. Nine subjects were enrolled and received study medication and completed the study. There were no discontinuations.
Pre-assignment Details Planned enrollment was 30 patients for a final sample size of 25 completed patients. The Sponsor decided to terminate the study after 9 completed patients (with agreement from the FDA), based on an interim analysis of the first 8 completed patients with no evidence of a harmful effect of 1 dose of 50 mg Riluzole Oral Film on swallowing function.
Arm/Group Title Patients With ALS With Single 50 mg Dose ROSF
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Single group of patients with a diagnosis of probable or definite ALS in accordance with the Revised El-Escorial Criteria.

They participated in a Videofluoroscopic Swallowing Study (VFSS) with 11 bolus trials and were scored by two independent raters on the Penetration Aspiration Scale (PAS). They were then given a single dose of 50 mg Riluzole Oral Film, and the Videofluoroscopic Swallowing Study was repeated at three minutes after dosing.

Period Title: Overall Study
Started 9
Completed 9
Not Completed 0
Arm/Group Title Subjects With ALS
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Subjects with a diagnosis of probable or definite ALS in accordance with the Revisited El-Escorial Criteria will be administered a single dose of the Riluzole Oral Soluble Film (ROSF) 50 mg.

Riluzole Oral Soluble film (ROSF) 50 mg: Enrolled subjects will undergo an instrumental evaluation of swallowing safety before and after administration of a single dose of the ROSF formulation containing 50 mg of riluzole.

Overall Number of Baseline Participants 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 9 participants
61
(37 to 77)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
6
  66.7%
Male
3
  33.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Hispanic or Latino
2
  22.2%
Not Hispanic or Latino
7
  77.8%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
  11.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
  11.1%
White
7
  77.8%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 9 participants
9
 100.0%
Change in Penetration Aspiration Scale Score on Videofluoroscopic Study before and after a single do   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
5
  55.6%
[1]
Measure Description: Subjects participated in a Videofluoroscopic Swallowing Study (VFSS) with 11 bolus trials and were scored by two independent raters on the Penetration Aspiration Scale (PAS). They were then given a single dose of 50 mg Riluzole Oral Film, and the Videofluoroscopic Swallowing Study was repeated at three minutes after dosing. The primary outcome was change on PAS scores from pre- to post-dose.
1.Primary Outcome
Title Number of Participants Categorized by Differences From Baseline in Penetration Aspiration Scale (PAS) - Sum of Scores Method
Hide Description 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.
Time Frame Before and after administration of ROSF 50 mg on day 1 (visit 1)
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Hide Analysis Population Description
Safety Population comprising all enrolled subjects who received the single ROSF 50 mg administration.
Arm/Group Title Subjects With ALS
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Subjects with a diagnosis of probable or definite ALS in accordance with the Revised El-Escorial Criteria will be administered a single dose of the Riluzole Oral Soluble Film (ROSF) 50 mg.

Riluzole Oral Soluble film (ROSF) 50 mg: Enrolled subjects will undergo an instrumental evaluation of swallowing safety before and after administration of a single dose of the ROSF formulation containing 50 mg of riluzole.

Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
Increase in sum of scores post dose
0
   0.0%
Same sum of scores post dose
7
  77.8%
Decrease in sum of scores post dose
2
  22.2%
Time Frame From screening until 1 day post-dose
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Subjects With ALS
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Subjects with a diagnosis of probable or definite ALS in accordance with the Revisited El-Escorial Criteria will be administered a single dose of the Riluzole Oral Soluble Film (ROSF) 50 mg.

Riluzole Oral Soluble film (ROSF) 50 mg: Enrolled subjects will undergo an instrumental evaluation of swallowing safety before and after administration of a single dose of the ROSF formulation containing 50 mg of riluzole.

All-Cause Mortality
Subjects With ALS
Affected / at Risk (%)
Total   0/9 (0.00%) 
Hide Serious Adverse Events
Subjects With ALS
Affected / at Risk (%)
Total   0/9 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Subjects With ALS
Affected / at Risk (%)
Total   0/9 (0.00%) 
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Gary Slatko, Chief Medical Officer
Organization: Aquestive Therapeutics
Phone: 908-941-7050
EMail: clinicaltrials@aquestive.com
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Responsible Party: Aquestive Therapeutics
ClinicalTrials.gov Identifier: NCT03679975    
Other Study ID Numbers: 17MO1R-0012
First Submitted: September 19, 2018
First Posted: September 21, 2018
Results First Submitted: June 24, 2020
Results First Posted: August 10, 2020
Last Update Posted: August 18, 2020