Exenatide Once-weekly as a Treatment for Multiple System Atrophy

NCT04431713 | Completed Phase 2 Results DMC

• 2 other identifiers: 1255912020-000122-26
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Multiple System Atrophy

Outcome Measures

Primary Outcomes (1)

• Change in UMSARS Score (Parts I+II) From Baseline

  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.

  Baseline and 48 weeks

Secondary Outcomes (8)

• Loss of Independent Ambulation

  48 weeks

• Multiple System Atrophy Quality of Life (MSA-QoL) Scale

  48 weeks

• Number of Falls

  48 weeks

• Milestones on UMSARS Part 1 (Speech, Swallow and Falling)

  48 weeks

• Clinical Global Impression (CGI) Scale

  48 weeks

Study Arms (2)

Exenatide

EXPERIMENTAL

Drug: Exenatide Pen Injector [Bydureon]

Standard of care

NO INTERVENTION

Interventions

Exenatide Pen Injector [Bydureon] DRUG

Exenatide is a treatment licensed for use in Type 2 diabetes.

Exenatide

Eligibility Criteria

• Age: 30 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants aged 30-80 years old with a diagnosis of Possible or Probable MSA of the parkinsonian subtype (MSA-P) or cerebellar subtype (MSA-C) according to The Gilman Criteria (Gilman et al. 2008).
• Participants who are less than five years from the time of documented MSA diagnosis or from the time of documented parkinsonian / ataxic neurological condition that later turns out to be MSA.
• 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.
• Females of childbearing potential and male participants with partners of childbearing potential must agree to use an effective method of contraception from the time consent is signed until 10 weeks after treatment discontinuation. Females of childbearing potential have a negative pregnancy test within 7 days prior to being randomised.
• 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.

You may not qualify if:

• Females who are pregnant, planning pregnancy or breastfeeding.
• Women of child-bearing potential who do not practice an acceptable method of birth control. Subjects who meet any of the following criteria which tend to suggest advance disease:
• 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
• Impairment in ambulation as assessed by a score of ≥ 3 on UMSARS question 7
• Falling more frequently than once per week as assessed by a score of ≥ 3 on UMSARS question 8. Participants with a clinically significant or unstable medical or surgical condition, which in the opinion of the investigator might preclude safe completion of the study.
• Participants with active malignant neoplasms or history of malignant neoplasm in the last 5 years. Participants with movement disorders other than MSA.
• Concurrent dementia defined by a score lower than 21 on the MoCA.
• Concurrent severe depression defined by a score of ≥30 on the Beck Depression Inventory-II.
• History of deep brain stimulation surgery.
• Participants who have taken any investigational products within 90 days prior to baseline.
• Participants with a BMI \< 18.5.
• Participants with diabetes, end stage renal disease or severely impaired renal function.
• History of clinically significant cardiac disease, pancreatitis and/or alcoholism.
• Participants with severe gastrointestinal disease including gastroparesis.

Sponsors & Collaborators

• University College, London: lead

Study Sites (1)

Leonard Wolfson Experimental Neurology Centre, National Hospital of Neurology and Neurosurgery, UCLH NHS Foundation trust

London, WC1N 3BG, United Kingdom

Location

Related Publications (33)

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MeSH Terms

Conditions

Multiple System Atrophy

Interventions

Exenatide

Condition Hierarchy (Ancestors)

Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Intervention Hierarchy (Ancestors)

Peptides; Amino Acids, Peptides, and Proteins; Venoms; Complex Mixtures; Toxins, Biological; Biological Factors

Results Point of Contact

• Title: Professor Foltynie
• Organization: UCL

t.foltynie@ucl.ac.uk

020 3448 8726

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 29, 2020
• First Posted: June 16, 2020
• Study Start: September 16, 2020
• Primary Completion: March 22, 2024
• Study Completion: March 22, 2024
• Last Updated: June 27, 2025
• Results First Posted: June 27, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)