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
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Riluzole Oral Soluble film (ROSF) 50 mg||Phase 2|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Intervention 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)|
|Official Title:||A Single Center Study to Evaluate the Effect of Riluzole Oral Soluble Film on Swallowing Safety in Individuals With Amyotrophic Lateral Sclerosis|
|Actual Study Start Date :||April 4, 2018|
|Actual Primary Completion Date :||September 26, 2018|
|Actual Study Completion Date :||December 21, 2018|
Experimental: Subjects with ALS
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.
Drug: Riluzole Oral Soluble film (ROSF) 50 mg
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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03679975
|United States, Florida|
|University of Florida Center for Movement Disorders & Neuroscience|
|Gainesville, Florida, United States, 32607|
|Study Director:||Cassie Jung||Aquestive Therapeutics|