Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Functional Gait Assessment |
The Functional Gait Assessment (FGA) is a 10-item scale that measures postural stability while individuals perform walking tasks that are scored from 0 to 3 (3 = normal, 2 = mild impairment, 1 = moderate impairment, 0 = severe impairment). Scores range from a minimum of 0 to a maximum of 30. Higher scores indicate better postural stability. (Participants scoring 23 or higher on the baseline FGA were not included in this study because their peripheral neuropathy was not yet affecting their gait/balance significantly.) |
Baseline, Week 2, Week 6, Week 10 (Primary Endpoint), Week 26, and Week 52 |
|
| Secondary |
10-Meter Walk Test |
Gait speed (10-meter walk, timing the middle 6 meters) is assessed under two conditions: Participants are instructed (1) to walk at normal speed and (2) to walk as fast as they can. Higher numbers indicate faster speeds (i.e., more meters per second). |
Baseline, Week 2, Week 6, Week 10 (Primary Endpoint), Week 26, and Week 52 |
|
| Secondary |
Timed Up and Go Test (TUG) |
The Timed Up and Go Test is part of the Centers for Disease Control (CDC)-recommended STEADI test protocol for balance function. From a seated position in a standard armchair, the participant is asked to stand up from the chair, walk to a line on the floor 10 feet away at normal pace, turn, walk back to the chair at normal pace, and sit down again. The tester records the time taken from the command "Go" until the subject sits down again. Lower times indicate better/faster performance. |
Baseline, Week 2, Week 6, Week 10 (Primary Endpoint), Week 26, and Week 52 |
|
| Secondary |
Four-Stage Balance Test |
The 4-Stage Balance Test is part of the Centers for Disease Control's recommended test protocol for balance function. It includes four gradually more challenging postures the subject is exposed to: (1) Stand with feet side by side. (2) Stand with feet in semi-tandem stance. (3) Stand with feet in tandem stance. (4) Stand on one leg. Participants pass each level if they can hold the stance for 10 seconds and then move on to the next stance. If they cannot hold the stance, the test ends. Higher times indicate better balance than lower times. |
Baseline, Week 2, Week 6, Week 10 (Primary Endpoint), Week 26, and Week 52 |
|
| Secondary |
Activities-specific Balance Confidence (ABC) Scale |
Powell and Myers (1995) developed the Activities-specific Balance Confidence (ABC) Scale to detect levels of balance confidence in elderly persons. The ABC scale is a one-page questionnaire that asks questions about balance confidence when performing 16 different tasks. The items are rated on a scale of 0 to 100; a score of 0 indicates no confidence and a score of 100 indicates complete confidence when performing the task. The overall score is calculated by adding the individual items then dividing by the total number of items (16). The higher the score, the greater the person's balance confidence; thus, higher scores indicate that subjects are more confident of their balance. |
Baseline, Week 2, Week 6, Week 10 (Primary Endpoint), Week 26, and Week 52 |
|
| Secondary |
Vestibular Activities of Daily Living Scale (VADL) |
The Vestibular Activities of Daily Living Scale (VADL) is a self-reported questionnaire that was developed to assess self-perceived disability in individuals with vestibular impairment. It evaluates the effects of vertigo and balance disorders on independence in 28 activities of daily living. Participants selected a number ranging from 1 = "Independent" to 10 = "Too Difficult, No Longer Perform." The scores on the 28 activities were averaged. The higher the score (up to 10), the greater is the person's self-perceived disability (i.e., the person feels less independence in activities of daily living). Lower averages of the 28 items indicate greater independence in activities of daily living (e.g., a healthy adult's VADL score most likely would be between 1.0 and 2.0). |
Baseline, Week 2, Week 6, Week 10 (Primary Endpoint), Week 26, and Week 52 |
|
| Secondary |
Patient Health Questionnaire-9 (PHQ-9) |
The PHQ-9 is a nine-question tool for assessing depression and emotional well-being. Participants rate nine areas on a 0 to 3 scale in response to the question, "Over the last 2 weeks, how often have you been bothered by any of the following problems?" The rating scale is as follows: 0 = "Not at All," 1 = "Several Days," 2 = "More Than Half the Days," and 3 = "Nearly Every Day." A PHQ-9 total score of 0-4 points indicates "normal" or minimal depression. Scores between 5-9 points indicate mild depression; 10-14 points, moderate depression; 15-19 points, moderately severe depression; and 20 or more points, severe depression. (The minimum score on the PHQ-9 is 0; the maximum score is 27.) In short, lower scores indicate less depression. |
Baseline, Week 2, Week 6, Week 10 (Primary Endpoint), Week 26, and Week 52 |
|
| Secondary |
Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Interference Short Form 6b |
The Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Interference Short Form 6b measures the self-reported consequences of pain on relevant aspects of a person's life over the past 7 days and may include the extent to which pain hinders engagement with social, cognitive, emotional, physical, and recreational activities. PROMIS measures are scored on the T-score metric (mean = 50, standard deviation = 10). Higher scores (i.e., those greater than 50, the mean) indicate that the participants perceive their pain as interfering in their daily lives more than average; lower scores (i.e., those less than 50, the mean) indicate that the participants perceive their pain as interfering less than average in their daily lives, which would be considered a better outcome. |
Baseline, Week 2, Week 6, Week 10 (Primary Endpoint), Week 26, and Week 52 |
|
| Secondary |
PROMIS Pain Intensity Form 1a |
The PROMIS Pain Intensity Form 1a instrument asks participants to rate the intensity of their pain on average over the previous 7 days. It is universal rather than disease specific. The higher the score on a scale of 0 ("no pain") to 10 ("worst pain imaginable), the more intense is the person's perception of pain; lower scores indicate less intense perception of pain, a better outcome. |
Baseline, Week 2, Week 6, Week 10 (Primary Endpoint), Week 26, and Week 52 |
|
| Secondary |
PROMIS Ability to Participate Short Form 8a |
The PROMIS Ability to Participate in Social Roles and Activities assesses the participants' perceived ability to perform their usual social roles and activities. PROMIS measures are scored on the T-score metric (mean = 50, standard deviation = 10). A higher score (i.e., greater than 50, the mean) indicates participants perceive they have a better ability to participate in their roles and activities (i.e., a better outcome). |
Baseline, Week 2, Week 6, Week 10 (Primary Endpoint), Week 26, and Week 52 |
|
| Secondary |
PROMIS Satisfaction With Participation in Social Roles Short Form 8a |
The PROMIS Satisfaction with Social Roles and Activities items assess satisfaction with performing one's usual social roles and activities (e.g., "I am satisfied with my ability to participate in family activities"). PROMIS measures are scored on the T-score metric (mean = 50, standard deviation = 10). A higher score (i.e., those greater than 50, the mean) indicates the respondents' greater satisfaction in regard to their ability to participate in their roles and activities (i.e., a better outcome). |
Baseline, Week 2, Week 6, Week 10 (Primary Endpoint), Week 26, and Week 52 |
|
| Secondary |
Falls |
Subjects were asked about the number of times they have fallen in the past 6 and 12 months and whether they incurred an injury from falling. (For this study, the researchers used the World Health Organization's definition of a fall: "an event which results in a person coming to rest inadvertently on the ground or floor or other lower level.") These data were compared to falls during their participation in the trial as tracked with a calendar and reported as adverse events. Falls data were monitored as overall number of falls and fallers pre-and post-study participation as well as the number of falls/1000 patient days. A lower number indicates fewer falls over 26 weeks (6 months). |
Baseline and 26 Weeks |
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