Trial Outcomes & Findings for Exenatide Once-weekly as a Treatment for Multiple System Atrophy (NCT NCT04431713)

Exenatide Once-weekly as a Treatment for Multiple System Atrophy

The primary endpoint is the total Unified Multiple System Atrophy Rating Scale (UMSARS) score (Parts I and II), exploring the change from baseline overtime (evaluated at 48-weeks). Part I is a 12-item historical interview (max score 48) and Part II is a 14-item clinical examination (max score 56). Part I and II are added together to give a total score, which can range from 0 to 104. Higher scores indicate worse disease severity.

Proportion of participants with loss of independent ambulation, defined by a score of 4 on the UMSARS-I Item 7 (walking) at 48 weeks.

The Multiple System Atrophy Quality of Life (MSA-QoL) scale measured health-related quality of life across three subscales (motor, nonmotor, and emotional/social functioning). Each item (40 in total) has five increasing levels of impairment (0 to 4), with 0 representing no impairment and 4 representing extreme impairment. Scores for the three subscales were generated by summing items and transformed to a range of 0 to 100 (100 × \[(observed score minus min possible score)/(max possible score minus min possible score)\]).

Number of falls reported by the participant at 48-weeks.

The UMSARS Part 1 is 12-item sub-scale which comprises a historic review of disease-related impairments. A single score using a 0 (no impairment) to 4 (severe impairment) was generated for each item (max score 48 points). We examined the proportion of participants reaching a score of ≥ 3 on UMSARS item 1 (speech), item 2 (swallowing) and item 8 (falling) by 48 weeks.

The CGI evaluates the change from the initiation of treatment on a seven-point scale (1 = =very much improved to 7 = very much worse since the initiation of treatment). A single score at 48 weeks was recorded.

The MoCA is a brief cognitive scale (scored out of 0-30 points). A score of 26 or more reflects normal cognitive abilities, whereas lower scores indicate cognitive impairment. The difference between groups (total score out of 30) was explored at 48 weeks.

The UMSARS Part 4 measures global disability (1 item) scored from 1 (completely independent) to 5 (totally dependent and helpless. Bedridden).

The BDI-II is a self-report questionnaire designed to measure the severity of depression symptomatology (scored out of 0-63 points). A score of 13 or less indicated minimal, 14-19 indicated mild, 20-28 indicated moderate and 29-63 indicated severe depression. The difference between groups was explored at 48 weeks.