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    Clinical Trial Results:
    A Phase IIa, open label, single-site, 48 week randomised controlled trial evaluating the safety and efficacy of Exenatide once-weekly in the treatment of patients with Multiple System Atrophy

    Summary
    EudraCT number
    		 2020-000122-26
    Trial protocol
    		 GB  
    Global end of trial date
    31 Jul 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    19 Jun 2025
    First version publication date
    19 Jun 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    125591
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04431713
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    University College London (UCL)
    Sponsor organisation address
    250 Euston Road, London, United Kingdom, NW1 2PG
    Public contact
    Catherine Maidens, University College London (UCL), ctimps@ucl.ac.uk
    Scientific contact
    Catherine Maidens, University College London (UCL), ctimps@ucl.ac.uk
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    22 Mar 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    22 Mar 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Jul 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    This is an open label, randomised trial to collect pilot data from which to estimate the effectiveness of Exenatide in modifying disease progression of patients with Multiple System Atrophy. The primary endpoint will be the difference in total Unified Multiple System Atrophy Rating Scale (UMSARS-I) score (Parts I and II) at 48 weeks comparing exenatide to best medically treated patients. The Unified MSA rating scale is globally recognised as the best available scale to objectively rate the severity of MSA. Part 1 (Historical review of symptom severity) and Part 2 (motor examination) have been previously used many times as an outcome measure in trials of MSA. Part 3 captures additional autonomic symptoms eg dizziness from low blood pressure, while Part 4 captures overall disability from the disease.
    Protection of trial subjects
    NA
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    16 Sep 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 50
    Worldwide total number of subjects
    50
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    30
    From 65 to 84 years
    20
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Patient were recruited from specialist outpatient clinics for MSA at UCLH, referrals from movement disorder specialists across the UK and self-referral to the trial team via email.

    Pre-assignment
    Screening details
    		 Between 23 September 2020 and 06 May 2022, 53 participants were screened for eligibility. Fifty participants were randomly assigned to either exenatide or to act as contemporaneous controls. Reasons for screening failures included low BMI, significant cognitive impairment and previous exposure to IMP.

    Period 1
    Period 1 title
    Baseline
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    Open-label.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Exenatide
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Exenatide
    Investigational medicinal product code
    Other name
    Bydureon, BCise
    Pharmaceutical forms
    Solution for injection in pre-filled pen
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    2mg once weekly for 48-weeks.

    Arm title
    		 Standard of Care
    Arm description
    		 -
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		Exenatide Standard of Care
    Started
    25
    25
    Completed
    25
    25
    Period 2
    Period 2 title
    Overall Trial
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    Open-label.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Exenatide
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Exenatide
    Investigational medicinal product code
    Other name
    Bydureon, BCise
    Pharmaceutical forms
    Solution for injection in pre-filled pen
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    2mg once weekly for 48-weeks.

    Arm title
    		 Standard of Care
    Arm description
    		 -
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 2
    		Exenatide Standard of Care
    Started
    25
    25
    Completed
    21
    23
    Not completed
    4
    2
         Death unrelated to IMP
    2
    2
         Lost to follow-up
    2
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Exenatide
    Reporting group description
    		 -

    Reporting group title
    Standard of Care
    Reporting group description
    		 -

    Reporting group values
    		 Exenatide Standard of Care Total
    Number of subjects
    		 25 25 50
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 63.3 ( 8.4 ) 62.4 ( 7.3 ) -
    Gender categorical
    Units: Subjects
        Female
    		 13 13 26
        Male
    		 12 12 24
    MSA Sub-type
    Units: Subjects
        MSA-P
    		 14 14 28
        MSA-C
    		 11 11 22

    End points

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    End points reporting groups
    Reporting group title
    Exenatide
    Reporting group description
    		 -

    Reporting group title
    Standard of Care
    Reporting group description
    		 -
    Reporting group title
    Exenatide
    Reporting group description
    		 -

    Reporting group title
    Standard of Care
    Reporting group description
    		 -

    Primary: UMSARS Parts I and II Combined

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    End point title
    		 UMSARS Parts I and II Combined
    End point description
    The primary endpoint is the total Unified Multiple System Atrophy Rating Scale (UMSARS) score (Parts I and II). Higher scores indicate worse disease severity.
    End point type
    Primary
    End point timeframe
    48-weeks
    End point values
    		 Exenatide Standard of Care
    Number of subjects analysed
    24
    24
    Units: Score
        arithmetic mean (confidence interval 95%)
    6.1 (3.0 to 9.3)
    13.3 (9.2 to 17.3)
    Statistical analysis title
    		 Intention-to-treat Population
    Statistical analysis description
    Compared exenatide participants to best medically treated participants using a two-level mixed model that included the total UMSARS scores from four time-points (12 weeks, 24 weeks, 36 weeks and 48 weeks) with interaction terms between the intervention and time-point indicator variables to enable estimation of the effect of intervention at 48 weeks. This model also adjusted for baseline UMSARS score and MSA subtype using fixed effects.
    Comparison groups
    Exenatide v Standard of Care
    Number of subjects included in analysis
    48
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.05
    Method
    		 Mixed models analysis
    Confidence interval

    Secondary: Loss of Independent Ambulation

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    End point title
    		 Loss of Independent Ambulation
    End point description
    Proportion of patients with loss of independent ambulation, defined by a score of 4 in UMSARS-I Item 7 (walking).
    End point type
    Secondary
    End point timeframe
    48-weeks
    End point values
    		 Exenatide Standard of Care
    Number of subjects analysed
    25
    25
    Units: Number of Participants
    2
    2
    No statistical analyses for this end point

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

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    End point title
    		 Multiple System Atrophy Quality of Life (MSA-QoL) Scale
    End point description
    Higher scores indicate worse quality of life.
    End point type
    Secondary
    End point timeframe
    48-weeks
    End point values
    		 Exenatide Standard of Care
    Number of subjects analysed
    21
    23
    Units: Score
    arithmetic mean (standard deviation)
        Motor Subscore
    53.1 ( 19.8 )
    53.7 ( 23.7 )
        Non-motor Subscore
    37.4 ( 17.8 )
    39.3 ( 15.4 )
        Emotional/Social Functioning Subscore
    33.3 ( 22.8 )
    43.1 ( 23.9 )
    No statistical analyses for this end point

    Secondary: Number of Falls

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    End point title
    		 Number of Falls
    End point description
    Number of falls at 48-weeks.
    End point type
    Secondary
    End point timeframe
    48-weeks
    End point values
    		 Exenatide Standard of Care
    Number of subjects analysed
    21
    23
    Units: Number of Events
        arithmetic mean (standard deviation)
    1.7 ( 2.9 )
    3.0 ( 6.8 )
    No statistical analyses for this end point

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

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    End point title
    		 Milestones on UMSARS Part 1 (Speech, Swallow and Falling)
    End point description
    The proportion of patients reaching a score of ≥ 3 on UMSARS item 1 (speech), item 2 (swallowing) and item 8 (falling).
    End point type
    Secondary
    End point timeframe
    48-weeks
    End point values
    		 Exenatide Standard of Care
    Number of subjects analysed
    21
    23
    Units: Number of Participants
        UMSARS Part 1 Item 1 (Speech)
    3
    7
        UMSARS Part 1 Item 2 (Swallowing)
    2
    8
        UMSARS Part 1 Item 8 (Falling)
    3
    6
    No statistical analyses for this end point

    Secondary: Clinical Global Impression (CGI) Scale

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    End point title
    		 Clinical Global Impression (CGI) Scale
    End point description
    End point type
    Secondary
    End point timeframe
    48-weeks.
    End point values
    		 Exenatide Standard of Care
    Number of subjects analysed
    21
    23
    Units: Score
        arithmetic mean (standard deviation)
    3.1 ( 1.0 )
    2.4 ( 0.8 )
    No statistical analyses for this end point

    Secondary: Montreal Cognitive Assessment (MoCA)

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    End point title
    		 Montreal Cognitive Assessment (MoCA)
    End point description
    Lower scores indicate cognitive decline, and a score of 26 or more reflects normal cognition.
    End point type
    Secondary
    End point timeframe
    48-weeks.
    End point values
    		 Exenatide Standard of Care
    Number of subjects analysed
    21
    23
    Units: Score
        arithmetic mean (standard deviation)
    26.0 ( 2.7 )
    27.2 ( 2.5 )
    No statistical analyses for this end point

    Secondary: UMSARS Part IV

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    End point title
    		 UMSARS Part IV
    End point description
    Higher scores indicate worse disability.
    End point type
    Secondary
    End point timeframe
    48-weeks.
    End point values
    		 Exenatide Standard of Care
    Number of subjects analysed
    21
    23
    Units: Score
        arithmetic mean (standard deviation)
    2.8 ( 1.1 )
    3.0 ( 0.9 )
    No statistical analyses for this end point

    Secondary: Beck Depression Inventory II (BDI-II)

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    End point title
    		 Beck Depression Inventory II (BDI-II)
    End point description
    Measures depression status, with worse scores indicating more severe depression.
    End point type
    Secondary
    End point timeframe
    48-weeks.
    End point values
    		 Exenatide Standard of Care
    Number of subjects analysed
    21
    23
    Units: Score
        arithmetic mean (standard deviation)
    14.3 ( 8.8 )
    15.2 ( 8.0 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline to week 52 (12 weeks after study drug was discontinued).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 CTCAE
    Dictionary version
    5.0
    Reporting groups
    Reporting group title
    Exenatide
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Serious adverse events
    		 Exenatide Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    10 / 25 (40.00%)
    10 / 25 (40.00%)
         number of deaths (all causes)
    2
    2
         number of deaths resulting from adverse events
    0
    0
    Vascular disorders
    Vascular Disorders
         subjects affected / exposed
    0 / 25 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Myocardial Infarction
         subjects affected / exposed
    0 / 25 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    1 / 1
    Surgical and medical procedures
    Possible Detachment of Percutaneous Endoscopic Gastrostomy Tube
         subjects affected / exposed
    0 / 25 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Dysphagia
         subjects affected / exposed
    0 / 25 (0.00%)
    4 / 25 (16.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Syncope
         subjects affected / exposed
    2 / 25 (8.00%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Falls
         subjects affected / exposed
    2 / 25 (8.00%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Worsening of MSA
         subjects affected / exposed
    2 / 25 (8.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    0 / 1
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    1 / 25 (4.00%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Diarrhea
         subjects affected / exposed
    1 / 25 (4.00%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Serum Amylase Increased
         subjects affected / exposed
    2 / 25 (8.00%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Aspiration
         subjects affected / exposed
    2 / 25 (8.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory Failure
         subjects affected / exposed
    1 / 25 (4.00%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 25 (4.00%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Depression
         subjects affected / exposed
    1 / 25 (4.00%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Urinary Retention
         subjects affected / exposed
    1 / 25 (4.00%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Lung Infection
         subjects affected / exposed
    1 / 25 (4.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 25 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    0 / 25 (0.00%)
    3 / 25 (12.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Upper Respiratory Infection
         subjects affected / exposed
    3 / 25 (12.00%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Urinary Tract Infections
         subjects affected / exposed
    3 / 25 (12.00%)
    2 / 25 (8.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hyponatremia
         subjects affected / exposed
    0 / 25 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Dehydration
         subjects affected / exposed
    0 / 25 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Exenatide Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    25 / 25 (100.00%)
    24 / 25 (96.00%)
    Nervous system disorders
    Gait Disturbance
         subjects affected / exposed
    17 / 25 (68.00%)
    17 / 25 (68.00%)
         occurrences all number
    19
    19
    General disorders and administration site conditions
    Fatigue and Pain
         subjects affected / exposed
    6 / 25 (24.00%)
    6 / 25 (24.00%)
         occurrences all number
    8
    6
    Other, Miscellaneous
         subjects affected / exposed
    21 / 25 (84.00%)
    22 / 25 (88.00%)
         occurrences all number
    64
    52
    Gastrointestinal disorders
    Nausea, Bloating, Constipation
         subjects affected / exposed
    14 / 25 (56.00%)
    12 / 25 (48.00%)
         occurrences all number
    36
    17
    Hepatobiliary disorders
    Serum Amylase increase
         subjects affected / exposed
    1 / 25 (4.00%)
    1 / 25 (4.00%)
         occurrences all number
    1
    1
    Skin and subcutaneous tissue disorders
    Skin Disorders e.g., Nodules, Rashes
         subjects affected / exposed
    12 / 25 (48.00%)
    4 / 25 (16.00%)
         occurrences all number
    14
    5
    Infections and infestations
    Infections e.g., UTI, Chest
         subjects affected / exposed
    16 / 25 (64.00%)
    14 / 25 (56.00%)
         occurrences all number
    38
    16
    Metabolism and nutrition disorders
    Weight Loss, Anorexia
         subjects affected / exposed
    11 / 25 (44.00%)
    4 / 25 (16.00%)
         occurrences all number
    14
    4

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Oct 2020
    Amendment 1 - Non-substantial updates.
    05 Mar 2021
    Amendment 2 - Update to protocol.
    15 Sep 2021
    Amendment 3 - Update to protocol.
    09 Dec 2021
    Amendment 4 - Update to PIS and ICF.
    03 Aug 2022
    Amendment 5 - Update to protocol.
    15 Sep 2022
    Amendment 6 - Update to PIS and ICF.
    28 Jun 2023
    Amendment 7 - SmPc update
    19 Jun 2024
    Amendment 8 - Update to protocol (exploratory analysis).

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Open label trial design.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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