Change in UMSARS Score (Parts I+II) From Baseline
| Group | Value (score on a scale) |
|---|---|
| Exenatide | 6.1 |
| Standard of Care | 13.3 |
Trial · Interventional
CompletedPhase 2Results postedRegistry dataShort name MSA
United Kingdom
1 trial · 1 completed · 1 with posted results
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
Fifty patients with early stage Multiple System Atrophy (MSA) will be recruited and randomised to receive Exenatide injections, or to act as controls in this open label trial. For half of the patients, Exenatide will be given as a once weekly subcutaneous injection in addition to participant's regular medication. All patients will continue to receive standard of care treatment for MSA. Detailed assessments will be made of all patients at baseline and periodically for a total of 48 weeks. The primary endpoint will be the difference in total Unified Multiple System Atrophy Rating Scale (UMSARS) score (Parts I and II) at 48 weeks comparing Exenatide treated to best medically treated patients (controls). Secondary measures will include adverse event reports, self-completed questionnaires, and blood test results. Aside from these assessments, all patients will continue any regular MSA medications throughout the trial with adjustments made only according to clinical need. Standard of care treatment for patients on non IMP arm will be dependant on the patients individual symptoms - there is no broad standard treatment for every patient.
Experimental
Exenatide
No intervention
Standard of care
Outcomes the sponsor listed as primary.
Change in UMSARS Score (Parts I+II) From Baseline
Time frame · Baseline and 48 weeks
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.
Loss of Independent Ambulation
Time frame · 48 weeks
Proportion of participants with loss of independent ambulation, defined by a score of 4 on the UMSARS-I Item 7 (walking) at 48 weeks.
Multiple System Atrophy Quality of Life (MSA-QoL) Scale
Time frame · 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
Time frame · 48 weeks
Number of falls reported by the participant at 48-weeks.
Milestones on UMSARS Part 1 (Speech, Swallow and Falling)
Time frame · 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.
Clinical Global Impression (CGI) Scale
Time frame · 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.
Montreal Cognitive Assessment (MoCA)
Time frame · 48 weeks
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.
UMSARS Part IV
Time frame · Score at 48 Weeks
The UMSARS Part 4 measures global disability (1 item) scored from 1 (completely independent) to 5 (totally dependent and helpless. Bedridden).
Beck Depression Inventory II (BDI-II)
Time frame · 48 weeks
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.
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
| Group | Value (score on a scale) |
|---|---|
| Exenatide | 6.1 |
| Standard of Care | 13.3 |
| Group | Value (Participants) |
|---|---|
| Exenatide | 2 |
| Standard of Care | 2 |
| Group | Value (score on a scale) | Spread |
|---|---|---|
| Exenatide | 53.1 | 19.8 |
| Standard of Care | 53.7 | 23.7 |
| Group | Value (Number of falls) | Spread |
|---|---|---|
| Exenatide | 1.7 | 2.9 |
| Standard of Care | 3.0 | 6.8 |
| Group | Value (Participants) |
|---|---|
| Exenatide | 3 |
| Standard of Care | 7 |
| Group | Value (score on a scale) | Spread |
|---|---|---|
| Exenatide | 3.1 | 1.0 |
| Standard of Care | 2.4 | 0.8 |
| Group | Value (score on a scale) | Spread |
|---|---|---|
| Exenatide | 26.0 | 2.7 |
| Standard of Care | 27.2 | 2.5 |
| Group | Value (Score on a scale) | Spread |
|---|---|---|
| Exenatide | 2.8 | 1.1 |
| Standard of Care | 3.0 | 0.9 |
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| ExenatideParticipants randomised to receive exenatide. | 2 / 25 | 10 / 25 | 6 / 25 |
| Standard of CareParticipants randomised to receive standard care only. | 2 / 25 | 10 / 25 | 1 / 25 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies that cite this trial.
9 studies on this page
Indexing hint: Randomized controlled trial · 2018
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PMID 28334990PMC6248513DOI 10.1093/brain/awx044PUB-28334990
Indexing hint: Randomized controlled trial · 2017
Athauda et al. 2017Lancet (London, England)cited by 719
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Indexing hint: Observational study · 2016
Madsbad et al. 2016Diabetes, obesity & metabolismcited by 231
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Indexing hint: Randomized controlled trial · 2014
Aviles-Olmos et al. 2014Journal of Parkinson's diseasecited by 231
PMID 24662192DOI 10.3233/jpd-140364PUB-24662192
Indexing hint: Randomized controlled trial · 2013
Aviles-Olmos et al. 2013The Journal of clinical investigationcited by 440
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PubMed tag: none · 2012
Fineman et al. 2012Diabetes, obesity & metabolismcited by 117
PMID 22236356DOI 10.1111/j.1463-1326.2012.01561.xPUB-22236356
Indexing hint: Randomized controlled trial · 2011
Blevins et al. 2011The Journal of clinical endocrinology and metabolismcited by 340
PMID 21307137DOI 10.1210/jc.2010-2081PUB-21307137
Indexing hint: Randomized controlled trial · 2010
Bergenstal et al. 2010Lancet (London, England)cited by 457
PMID 20580422DOI 10.1016/s0140-6736(10)60590-9PUB-20580422
Indexing hint: Randomized controlled trial · 2008
Drucker et al. 2008Lancet (London, England)cited by 809
PMID 18782641DOI 10.1016/s0140-6736(08)61206-4PUB-18782641
2 studies on this page
Indexing hint: Randomized controlled trial · 2019
Athauda et al. 2019JAMA neurologycited by 210
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Indexing hint: Review · 2015
Consoli et al. 2015Expert opinion on drug safetycited by 43
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References listed at the registry that are not in PepBank. They link to PubMed.
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