Riluzole Oral Soluble Film (ROSF) Swallowing Safety in Amyotrophic Lateral Sclerosis (ALS)
Condition: Amyotrophic Lateral Sclerosis
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis
Intervention: Riluzole Oral Soluble film (ROSF) 50 mg
Intervention description: 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.
Intervention MeSH terms: Riluzole
Phase: Phase 2
Study type: Interventional
Primary purpose: Other
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)
Enrollment: 9.0
Sex: All
Minimum age: 18 Years
Maximum age: 80 Years
Healthy volunteers: No

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.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Male or female subjects between 18-80 years of age, inclusive

          2. Subjects with a diagnosis of probable or definite ALS in accordance with the Revised
             El-Escorial Criteria

          3. 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.

          4. Subjects with no known allergy to barium, riluzole or inactive ingredients in ROSF

          5. Subject or subject's legally authorized representative must be willing and able to
             give informed consent/assent and HIPAA authorization.

          6. 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.

          7. 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).

          8. Availability to volunteer for the entire study duration and willing to adhere to all
             protocol requirements

          9. 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.

        Exclusion Criteria:

          1. Subjects who score 2 or below on the Functional Oral Intake Scale.

          2. Subjects with a prior swallowing study that has shown a Penetration Aspiration Scale
             score of 3 or greater

          3. Subjects with a history of 2 or more episodes of aspiration pneumonia requiring
             hospitalization

          4. Subjects with a history of clinically significant liver disease, renal disease, or any
             other medical condition judged to be exclusionary by the Investigator

          5. 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

          6. Female subjects who had a positive urine pregnancy test (βhCG) at screening or Visit
             1, were trying to become pregnant, or were breastfeeding.

          7. Subjects with active cancer within the previous 2 years, except treated basal cell
             carcinoma of the skin

          8. 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.

          9. Subjects with known history of moderate or severe renal impairment as defined by a
             calculated creatinine clearance of ≤50 mL/minute

         10. 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.)

         11. 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).

         12. Use of potentially hepatotoxic drugs: (e.g., allopurinol, methyldopa, sulfasalazine).

         13. Subjects with clinically significant abnormal laboratory values in the judgment of the
             Investigator

         14. 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.

         15. 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.

         16. Employee or immediate relative of an employee of the investigator, Aquestive
             Therapeutics, any of its affiliates or partners, or inVentiv Health.