Trial Outcomes & Findings for Riluzole Oral Soluble Film (ROSF) Swallowing Safety in Amyotrophic Lateral Sclerosis (ALS) (NCT NCT03679975)

Last Updated: 2020-08-18

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

Before and after administration of ROSF 50 mg on day 1 (visit 1)

Results posted on

2020-08-18

Participant Flow

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.

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.

Participant milestones

Participant milestones
Measure	Patients With ALS With Single 50 mg Dose ROSF 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.
Overall Study STARTED	9
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Subjects With ALS n=9 Participants 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.
Age, Continuous	61 years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	7 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	9 Participants n=5 Participants
Change in Penetration Aspiration Scale Score on Videofluoroscopic Study before and after a single do	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: Before and after administration of ROSF 50 mg on day 1 (visit 1)

Population: Safety Population comprising all enrolled subjects who received the single ROSF 50 mg administration.

Outcome measures

Outcome measures
Measure	Subjects With ALS n=9 Participants 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.
Number of Participants Categorized by Differences From Baseline in Penetration Aspiration Scale (PAS) - Sum of Scores Method Increase in sum of scores post dose	0 Participants
Number of Participants Categorized by Differences From Baseline in Penetration Aspiration Scale (PAS) - Sum of Scores Method Same sum of scores post dose	7 Participants
Number of Participants Categorized by Differences From Baseline in Penetration Aspiration Scale (PAS) - Sum of Scores Method Decrease in sum of scores post dose	2 Participants

Adverse Events

Subjects With ALS

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Gary Slatko, Chief Medical Officer

Aquestive Therapeutics

Phone: 908-941-7050

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60