Exenatide Once-weekly as a Treatment for Multiple System Atrophy

What Was Tested

This registered phase 2 trial tested Exenatide.

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.

It had two arms (groups): Exenatide (experimental) and Standard of care (no intervention).

The registry classes the interventions as drug.

More on the Plan

Fifty patients with early stage MSA will be recruited and randomised to receive Exenatide injections, or to act as controls in this open label trial.

Once a potential participant has been identified they will receive a patient information leaflet, and will be given a minimum of 24 hours to read this before being recruited on to the trial.

Patients will need to be eligible for the trial by meeting the inclusion criteria.

During pre-treatment there will be a screening visit and a baseline visit.

Who Took Part

50 actual participants took part.

It was open to people of any sex aged from 30 years up to 80 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Participants who are able to walk at least 10 metres with or without assistance. Participants with an anticipated survival of at least three years in the opinion of the investigator. Participants that are willing to adhere to the study drug regimen. Participants that are willing and able to perform all protocol-specified assessments and comply with the study visit schedule. Willing and able to provide written informed consent. Subjects who are not able to write may give verbal consent in the presence of at least one witness, and the witness should sign the informed consent form.

The full record lists 8 inclusion and 17 exclusion criteria; among the exclusions: females who are pregnant, planning pregnancy or breastfeeding; speech impairment as assessed by a score of ≥ 3 on umsars question 1; swallowing impairment as assessed by a score of ≥ 3 on umsars question 2.

How the Study Was Run

The set-up was randomized, parallel-group and open-label.

The participants were allocated by chance, so the Exenatide group and the comparison group should start out alike.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Nothing was hidden in this open-label Exenatide study: the participants and staff both knew the treatment.

It started in 2020-09-16 with completion listed for 2024-03-22.

It ran at 1 site in United Kingdom.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Change in UMSARS Score (Parts I+II) From Baseline (time frame: Baseline and 48 weeks).

It also tracked 8 secondary outcomes, for example Loss of Independent Ambulation, Multiple System Atrophy Quality of Life (MSA-QoL) Scale, Number of Falls, Milestones on UMSARS Part 1 (Speech, Swallow and Falling), Clinical Global Impression (CGI) Scale and Montreal Cognitive Assessment (MoCA).

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Exenatide in 50 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Exenatide compound page, all human trials.