Amyotrophic Lateral Sclerosis (ALS)
Conditions
Keywords
Amyotrophic lateral sclerosis, ALS, Nuedexta, Bulbar function, motor neurons
Brief summary
The purpose of this study is to determine whether Nuedexta is effective in the treatment of symptoms (impaired speech, swallowing, and saliva control)associated with Amyotrophic Lateral Sclerosis (ALS).
Detailed description
Muscle weakness, the cardinal feature of ALS, leads to progressive loss of motor function affecting the limbs, tongue, respiratory and pharyngeal muscles. Symptomatic treatments such as the placement of a feeding tube, can compensate for the inability to swallow. Riluzole, the only approved treatment for ALS, may slow disease progression but no treatment is curative and none have improved function. Unexpectedly, Nuedexta®, approved for the treatment of labile emotionality that occurs in association with ALS and other neurological disorders, has been observed to improve bulbar function, primarily speech and swallowing, in a number of neurological disorders, including ALS. The basis for this is conjectural but likely due to a direct effect of the drug on motor neurons in the part of the brain that controls speech and swallowing. The same part of the brain appears to modulate the expression of emotions and interestingly the site of action of the drug is the same as a site that has been implicated in a juvenile form of ALS. This is a multicenter, randomized double-blind, placebo controlled, cross over study evaluating the palliative effect of Nuedexta® on bulbar dysfunction. It is expected that approximately 60 ALS patients from 7 clinical centers in the US will be enrolled.
Interventions
DRUGNuedexta
Nuedexta PO (by mouth) for 28 ± 3 days
DRUGMatching Placebo
matching placebo PO (by mouth) for 28 ± 3 days
Sponsors
ALS Association
State University of New York - Upstate Medical University
Center for Neurologic Study, La Jolla, California,
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria * Age 18 years or older * Exhibits bulbar dysfunction manifested by dysarthria and/or dysphagia, according to PI judgment, exhibits a score of 55 or above on the CNS-Bulbar Function Scale * Capable of providing informed consent and following trial procedures * Geographic accessibility to the site * Women must not be able to become pregnant for the duration of the study and must be willing to be on two contraceptive therapies * Slow vital capacity (SVC) measure ≥50% of predicted for gender, height, and age at the screening visit * Must be able to swallow capsules throughout the course of the study, according to PI judgment * Subjects must not have taken riluzole for at least 30 days or be on a 50mg BID dose of riluzole for at least 30 days prior to randomization (subjects how have never taken riluzole are permitted in the study) * Subjects taking anti-sialorrhea medication(s) must be on a stable dose for at least 30 days prior to randomization (anti-sialorrhea naïve subjects are permitted in the study) * Must be able to safely swallow at least 30 milliliters (mLs) of water for the water swallowing test
Exclusion criteria
* Prior use of Nuedexta® * Current use of dextromethorphan, quinidine, quinine, mefloquine or opioids * History of quinidine, quinine, or mefloquine-induced thrombocytopenia, hepatitis, or other hypersensitivity reactions * History of known sensitivity or intolerability to dextromethorphan * Use of an mono amine oxidase inhibitor (MAOI) or within 14 days of stopping an MAOI * Prolonged QT interval, congenital long QT syndrome, history suggestive of torsades de pointes, or heart failure * Complete atrioventricular (AV) block without implanted pacemaker, or subjects at high risk of complete AV block * Concomitant use with drugs that both prolong QT interval and are metabolized by cytochrome P 2D6 (CYP2D6) (i.e., thioridazine or pimozide) * Exposure to any other experimental agent (off-label use or investigational) within 30 days prior to Baseline Visit * Invasive ventilator dependence, such as tracheostomy * Any history of either substance abuse within the past year, unstable psychiatric disease, cognitive impairment, or dementia, according to PI judgment * Placement and/or usage of feeding tube * Pregnant women or women currently breastfeeding * Unable to turn diaphragm pacing device off during swallowing tests * Salivatory Botox within 90 days (3 months) of screening * Salivatory radiation within 180 days (6 months) of screening
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Bulbar Function Scale (CNS-BFS) Total Score | Average between Screening Visit to Visit 3 | The Center for Neurologic Study-Bulbar Function Scale (CNS-BFS) is a 21-item self report scale that assesses three domains of bulbar function: speech, swallowing and salivation. Scores for each question range from 1 (does not apply) to 5 (applies most of the time). The higher the score, the worse the speech, swallowing and salivation (sialorrhea). \[Range of score: 21-105\] The scale was modeled on the Center for Neurologic Study Emotional Lability Scale (CNS-LS) that has been a robust endpoint in four clinical trials. The scale was validated in a large population of ALS patients (n=122) and detects impaired bulbar function at a sensitivity of 90% and a specificity of 0.97%. Test re-test correlation was 0.92% at six-months (n=53). |
| Bulbar Function Scale (CNS-BFS) Sialorrhea Score | Average between Screening Visit to Visit 3 | The Center for Neurologic Study-Bulbar Function Scale (CNS-BFS) is a 21-item self report scale that assesses three domains of bulbar function: speech, swallowing and salivation. Scores for each question range from 1 (does not apply) to 5 (applies most of the time). The higher the score, the worse the salivation (sialorrhea). There are 7 salivation (sialorrhea) questions, with a score range of 7 to 35. |
| Bulbar Function Scale (CNS-BFS) Speech Score | Average between Screening Visit to Visit 3 | The Center for Neurologic Study-Bulbar Function Scale (CNS-BFS) is a 21-item self report scale that assesses three domains of bulbar function: speech, swallowing and salivation. Scores for each question range from 1 (does not apply) to 5 (applies most of the time). The higher the score, the worse the speech. There are 7 speech questions, with a score range of 7 to 35. |
| Bulbar Function Scale (CNS-BFS) Swallowing Score | Average between Screening Visit to Visit 3 | The Center for Neurologic Study-Bulbar Function Scale (CNS-BFS) is a 21-item self report scale that assesses three domains of bulbar function: speech, swallowing and salivation. Scores for each question range from 1 (does not apply) to 5 (applies most of the time). The higher the score, the worse the swallowing. There are 7 swallowing questions, with a score range of 7 to 35. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Timed Reading of Test Paragraph Result | Average between Baseline Visit to Visit 3 | Subjects will be asked to read 'The Rainbow Passage' a commonly used test paragraph utilized by speech pathologists to assess speech rate (words/minute). Study staff will time the subject to determine how many words the subject reads per minute. It is used primarily because it contains every sound in the English language. Subjects will also be observed for loudness, nasality, and intelligibility. |
| Average Water Swallowing Test (WST) | Average between Baseline Visit to Visit 3 | The Water Swallowing Test (WST) estimates swallowing speed, a useful and reproducible measure. While sitting, subjects are asked to drink 30 milliliters (mL) of liquid. The time for subjects to complete this task is a sensitive measure for the detection of swallowing dysfunction and is a simple measure for serial assessment of subjects. The test will be completed three times, with the best two scores recorded to obtain an average score. Following completion of the WST, the subject's swallowing abilities (choking, spillage, and effort) will be observed. |
| Visual Analog Scale - Swallowing Score | Average between Baseline Visit to Visit 3 | Visual analog scales are useful for measuring complex clinical events and offer the advantage of self-administration and responsiveness to change over time. The scales designed for this study inventory three domains of bulbar function: speech, swallowing and salivation (sialorrhea). For each of these, subjects score themselves by indicating their level of function on a scale of 1 (severe impairment) to 10 (normal). Scores range from 1 to 10; the higher the score, the more normal the function. |
| Visual Analog Scale - Salivation (Sialorrhea) Score | Average between Baseline Visit to Visit 3 | Visual analog scales are useful for measuring complex clinical events and offer the advantage of self-administration and responsiveness to change over time. The scales designed for this study inventory three domains of bulbar function: speech, swallowing and salivation (sialorrhea). For each of these, subjects score themselves by indicating their level of function on a scale of 1 (severe impairment) to 10 (normal). Scores range from 1 to 10; the higher the score, the more normal the function. |
| Center for Neurologic Study - Lability Scale (CNS-LS) Total Score | Average between Screening Visit to Visit 3 | The Center for Neurologic Study-Lability Scale (CNS-LS) is a 7-item self report scale that assesses pseudobulbar affect (PBA) by measuring the perceived frequency of PBA episodes (laughing or crying). Each item is scored using a 5-point Likert scale, from 1 (applies never) to 5 (applies most of the time). Scores range from 5-35. The higher the score, the worse the PBA. |
| Ashworth Spasticity Scale Score - Left Arm | Average between Baseline Visit to Visit 3 | This is a standard measure for spasticity that has been used in numerous ALS clinical trials to assess spasticity due to upper motor neuron dysfunction in ALS. Data is generated from the clinical exam and scored from 1-5, the lowest score indicating normal tone and the highest muscle rigidity. |
| Ashworth Spasticity Scale Score - Right Leg | Average between Baseline Visit to Visit 3 | This is a standard measure for spasticity that has been used in numerous ALS clinical trials to assess spasticity due to upper motor neuron dysfunction in ALS. Data is generated from the clinical exam and scored from 1-5, the lowest score indicating normal tone and the highest muscle rigidity. |
| Ashworth Spasticity Scale Score - Left Leg | Average between Baseline Visit to Visit 3 | This is a standard measure for spasticity that has been used in numerous ALS clinical trials to assess spasticity due to upper motor neuron dysfunction in ALS. Data is generated from the clinical exam and scored from 1-5, the lowest score indicating normal tone and the highest muscle rigidity. |
| Average Solids Swallowing Test | Average between Baseline Visit to Visit 3 | The Time Swallowing Test assesses the subject's ability to swallow solids. For this test, the subject will be asked to consume a tablespoon of cereal containing 5 cheerios. The subject will be instructed to close their mouth, chew and subsequently swallow the bolus. The time to complete this task will be recorded. The test will be completed three times to obtain an average score. |
| ALS Functional Rating Scale- Revised (ALSFRS-R) Total Score | Average between Screening Visit to Visit 3 | The ALSFRS-R is a quickly administered (5 min) ordinal rating scale used to determine a subject's assessment of their capability and independence in 12 functional activities. There are 12 questions, graded by the subject 0-4 (4 is normal). Score of 0 (worst) to 48 (best). Reflects speech and swallowing, fine motor skills, large motor skills, and breathing. |
| Visual Analog Scale - Speech Scores | Average between Baseline Visit to Visit 3 | Visual analog scales are useful for measuring complex clinical events and offer the advantage of self-administration and responsiveness to change over time. The scales designed for this study inventory three domains of bulbar function: speech, swallowing and salivation (sialorrhea). For each of these, subjects score themselves by indicating their level of function on a scale of 1 (severe impairment) to 10 (normal). Scores range from 1 to 10; the higher the score, the more normal the function. |
| Ashworth Spasticity Scale Score - Right Arm | Average between Baseline Visit to Visit 3 | This is a standard measure for spasticity that has been used in numerous ALS clinical trials to assess spasticity due to upper motor neuron dysfunction in ALS. Data is generated from the clinical exam and scored from 1-5, the lowest score indicating normal tone and the highest muscle rigidity. |
Countries
United States
Participant flow
Pre-assignment details
90 subjects were consented for this trial, however, of the 90 that consented, 30 subjects were found to not meet eligibility criteria, and therefore were not treated.
Participants by arm
| Arm | Count |
|---|---|
| All Study Participants | 60 |
| Total | 60 |
Baseline characteristics
| Characteristic | All Study Participants |
|---|---|
| Age, Continuous | 58 years |
| Baseline ALSFRS-R Total Score | 34.58 units on a scale STANDARD_DEVIATION 6.95 |
| Baseline CNS-BFS Total Score | 58.15 units on a scale STANDARD_DEVIATION 13.37 |
| Baseline CNS Lability Scale Total Score | 13.67 units on a scale STANDARD_DEVIATION 5.55 |
| Race/Ethnicity, Customized Asian | 1 participants |
| Race/Ethnicity, Customized Black/African American | 2 participants |
| Race/Ethnicity, Customized Caucasian/White | 57 participants |
| Race/Ethnicity, Customized Hispanic or Latino | 0 participants |
| Region of Enrollment United States | 60 participants |
| Sex: Female, Male Female | 26 Participants |
| Sex: Female, Male Male | 34 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 21 / 58 | 4 / 57 |
| serious Total, serious adverse events | 1 / 58 | 0 / 57 |
Outcome results
Primary
Bulbar Function Scale (CNS-BFS) Sialorrhea Score
The Center for Neurologic Study-Bulbar Function Scale (CNS-BFS) is a 21-item self report scale that assesses three domains of bulbar function: speech, swallowing and salivation. Scores for each question range from 1 (does not apply) to 5 (applies most of the time). The higher the score, the worse the salivation (sialorrhea). There are 7 salivation (sialorrhea) questions, with a score range of 7 to 35.
Time frame: Average between Screening Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Bulbar Function Scale (CNS-BFS) Sialorrhea Score | 14.28 units on a scale | Standard Error 0.37 |
| Matching Placebo | Bulbar Function Scale (CNS-BFS) Sialorrhea Score | 15.81 units on a scale | Standard Error 0.38 |
Primary
Bulbar Function Scale (CNS-BFS) Speech Score
The Center for Neurologic Study-Bulbar Function Scale (CNS-BFS) is a 21-item self report scale that assesses three domains of bulbar function: speech, swallowing and salivation. Scores for each question range from 1 (does not apply) to 5 (applies most of the time). The higher the score, the worse the speech. There are 7 speech questions, with a score range of 7 to 35.
Time frame: Average between Screening Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Bulbar Function Scale (CNS-BFS) Speech Score | 22.22 units on a scale | Standard Error 0.56 |
| Matching Placebo | Bulbar Function Scale (CNS-BFS) Speech Score | 24.57 units on a scale | Standard Error 0.57 |
Primary
Bulbar Function Scale (CNS-BFS) Swallowing Score
The Center for Neurologic Study-Bulbar Function Scale (CNS-BFS) is a 21-item self report scale that assesses three domains of bulbar function: speech, swallowing and salivation. Scores for each question range from 1 (does not apply) to 5 (applies most of the time). The higher the score, the worse the swallowing. There are 7 swallowing questions, with a score range of 7 to 35.
Time frame: Average between Screening Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Bulbar Function Scale (CNS-BFS) Swallowing Score | 17.14 units on a scale | Standard Error 0.46 |
| Matching Placebo | Bulbar Function Scale (CNS-BFS) Swallowing Score | 18.92 units on a scale | Standard Error 0.47 |
Primary
Bulbar Function Scale (CNS-BFS) Total Score
The Center for Neurologic Study-Bulbar Function Scale (CNS-BFS) is a 21-item self report scale that assesses three domains of bulbar function: speech, swallowing and salivation. Scores for each question range from 1 (does not apply) to 5 (applies most of the time). The higher the score, the worse the speech, swallowing and salivation (sialorrhea). \[Range of score: 21-105\] The scale was modeled on the Center for Neurologic Study Emotional Lability Scale (CNS-LS) that has been a robust endpoint in four clinical trials. The scale was validated in a large population of ALS patients (n=122) and detects impaired bulbar function at a sensitivity of 90% and a specificity of 0.97%. Test re-test correlation was 0.92% at six-months (n=53).
Time frame: Average between Screening Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Bulbar Function Scale (CNS-BFS) Total Score | 53.45 units on a scale | Standard Error 1.07 |
| Matching Placebo | Bulbar Function Scale (CNS-BFS) Total Score | 59.31 units on a scale | Standard Error 1.1 |
p-value: 0.0003Random Effects Model
Secondary
ALS Functional Rating Scale- Revised (ALSFRS-R) Total Score
The ALSFRS-R is a quickly administered (5 min) ordinal rating scale used to determine a subject's assessment of their capability and independence in 12 functional activities. There are 12 questions, graded by the subject 0-4 (4 is normal). Score of 0 (worst) to 48 (best). Reflects speech and swallowing, fine motor skills, large motor skills, and breathing.
Time frame: Average between Screening Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | ALS Functional Rating Scale- Revised (ALSFRS-R) Total Score | 34.15 units on a scale | Standard Error 0.33 |
| Matching Placebo | ALS Functional Rating Scale- Revised (ALSFRS-R) Total Score | 33.7 units on a scale | Standard Error 0.33 |
Secondary
Ashworth Spasticity Scale Score - Left Arm
This is a standard measure for spasticity that has been used in numerous ALS clinical trials to assess spasticity due to upper motor neuron dysfunction in ALS. Data is generated from the clinical exam and scored from 1-5, the lowest score indicating normal tone and the highest muscle rigidity.
Time frame: Average between Baseline Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Ashworth Spasticity Scale Score - Left Arm | 1.62 units on a scale | Standard Deviation 0.06 |
| Matching Placebo | Ashworth Spasticity Scale Score - Left Arm | 1.67 units on a scale | Standard Deviation 0.07 |
Secondary
Ashworth Spasticity Scale Score - Left Leg
This is a standard measure for spasticity that has been used in numerous ALS clinical trials to assess spasticity due to upper motor neuron dysfunction in ALS. Data is generated from the clinical exam and scored from 1-5, the lowest score indicating normal tone and the highest muscle rigidity.
Time frame: Average between Baseline Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Ashworth Spasticity Scale Score - Left Leg | 1.91 units on a scale | Standard Deviation 0.09 |
| Matching Placebo | Ashworth Spasticity Scale Score - Left Leg | 1.91 units on a scale | Standard Deviation 0.09 |
Secondary
Ashworth Spasticity Scale Score - Right Arm
This is a standard measure for spasticity that has been used in numerous ALS clinical trials to assess spasticity due to upper motor neuron dysfunction in ALS. Data is generated from the clinical exam and scored from 1-5, the lowest score indicating normal tone and the highest muscle rigidity.
Time frame: Average between Baseline Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Ashworth Spasticity Scale Score - Right Arm | 1.65 units on a scale | Standard Deviation 0.06 |
| Matching Placebo | Ashworth Spasticity Scale Score - Right Arm | 1.53 units on a scale | Standard Deviation 0.06 |
Secondary
Ashworth Spasticity Scale Score - Right Leg
This is a standard measure for spasticity that has been used in numerous ALS clinical trials to assess spasticity due to upper motor neuron dysfunction in ALS. Data is generated from the clinical exam and scored from 1-5, the lowest score indicating normal tone and the highest muscle rigidity.
Time frame: Average between Baseline Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Ashworth Spasticity Scale Score - Right Leg | 1.94 units on a scale | Standard Deviation 0.1 |
| Matching Placebo | Ashworth Spasticity Scale Score - Right Leg | 1.82 units on a scale | Standard Deviation 0.1 |
Secondary
Average Solids Swallowing Test
The Time Swallowing Test assesses the subject's ability to swallow solids. For this test, the subject will be asked to consume a tablespoon of cereal containing 5 cheerios. The subject will be instructed to close their mouth, chew and subsequently swallow the bolus. The time to complete this task will be recorded. The test will be completed three times to obtain an average score.
Time frame: Average between Baseline Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Average Solids Swallowing Test | 18.53 seconds | Standard Error 1.16 |
| Matching Placebo | Average Solids Swallowing Test | 19.45 seconds | Standard Error 1.19 |
Secondary
Average Water Swallowing Test (WST)
The Water Swallowing Test (WST) estimates swallowing speed, a useful and reproducible measure. While sitting, subjects are asked to drink 30 milliliters (mL) of liquid. The time for subjects to complete this task is a sensitive measure for the detection of swallowing dysfunction and is a simple measure for serial assessment of subjects. The test will be completed three times, with the best two scores recorded to obtain an average score. Following completion of the WST, the subject's swallowing abilities (choking, spillage, and effort) will be observed.
Time frame: Average between Baseline Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Average Water Swallowing Test (WST) | 12.16 seconds | Standard Error 0.71 |
| Matching Placebo | Average Water Swallowing Test (WST) | 13.11 seconds | Standard Error 0.72 |
Secondary
Center for Neurologic Study - Lability Scale (CNS-LS) Total Score
The Center for Neurologic Study-Lability Scale (CNS-LS) is a 7-item self report scale that assesses pseudobulbar affect (PBA) by measuring the perceived frequency of PBA episodes (laughing or crying). Each item is scored using a 5-point Likert scale, from 1 (applies never) to 5 (applies most of the time). Scores range from 5-35. The higher the score, the worse the PBA.
Time frame: Average between Screening Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Center for Neurologic Study - Lability Scale (CNS-LS) Total Score | 10.79 units on a scale | Standard Error 0.47 |
| Matching Placebo | Center for Neurologic Study - Lability Scale (CNS-LS) Total Score | 13.72 units on a scale | Standard Error 0.49 |
p-value: 0.0001Random Effects Model
Secondary
Timed Reading of Test Paragraph Result
Subjects will be asked to read 'The Rainbow Passage' a commonly used test paragraph utilized by speech pathologists to assess speech rate (words/minute). Study staff will time the subject to determine how many words the subject reads per minute. It is used primarily because it contains every sound in the English language. Subjects will also be observed for loudness, nasality, and intelligibility.
Time frame: Average between Baseline Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Timed Reading of Test Paragraph Result | 107.12 words per minute | Standard Error 1.79 |
| Matching Placebo | Timed Reading of Test Paragraph Result | 103.37 words per minute | Standard Error 1.83 |
Secondary
Visual Analog Scale - Salivation (Sialorrhea) Score
Visual analog scales are useful for measuring complex clinical events and offer the advantage of self-administration and responsiveness to change over time. The scales designed for this study inventory three domains of bulbar function: speech, swallowing and salivation (sialorrhea). For each of these, subjects score themselves by indicating their level of function on a scale of 1 (severe impairment) to 10 (normal). Scores range from 1 to 10; the higher the score, the more normal the function.
Time frame: Average between Baseline Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Visual Analog Scale - Salivation (Sialorrhea) Score | 6.78 units on a scale | Standard Error 0.31 |
| Matching Placebo | Visual Analog Scale - Salivation (Sialorrhea) Score | 6.78 units on a scale | Standard Error 0.32 |
Secondary
Visual Analog Scale - Speech Scores
Visual analog scales are useful for measuring complex clinical events and offer the advantage of self-administration and responsiveness to change over time. The scales designed for this study inventory three domains of bulbar function: speech, swallowing and salivation (sialorrhea). For each of these, subjects score themselves by indicating their level of function on a scale of 1 (severe impairment) to 10 (normal). Scores range from 1 to 10; the higher the score, the more normal the function.
Time frame: Average between Baseline Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Visual Analog Scale - Speech Scores | 4.97 units on a scale | Standard Error 0.21 |
| Matching Placebo | Visual Analog Scale - Speech Scores | 4.11 units on a scale | Standard Error 0.21 |
Secondary
Visual Analog Scale - Swallowing Score
Visual analog scales are useful for measuring complex clinical events and offer the advantage of self-administration and responsiveness to change over time. The scales designed for this study inventory three domains of bulbar function: speech, swallowing and salivation (sialorrhea). For each of these, subjects score themselves by indicating their level of function on a scale of 1 (severe impairment) to 10 (normal). Scores range from 1 to 10; the higher the score, the more normal the function.
Time frame: Average between Baseline Visit to Visit 3
Population: Includes all participants that received at least one dose of intervention.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Active Drug (Neudexta) | Visual Analog Scale - Swallowing Score | 7.23 units on a scale | Standard Error 0.29 |
| Matching Placebo | Visual Analog Scale - Swallowing Score | 6.93 units on a scale | Standard Error 0.3 |