<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT04431713</url>
  </required_header>
  <id_info>
    <org_study_id>125591</org_study_id>
    <secondary_id>2020-000122-26</secondary_id>
    <nct_id>NCT04431713</nct_id>
  </id_info>
  <brief_title>Exenatide Once-weekly as a Treatment for Multiple System Atrophy</brief_title>
  <acronym>MSA</acronym>
  <official_title>An 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</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University College, London</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>University College, London</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      Fifty patients with early stage Multiple System Atrophy (MSA) will be recruited and&#xD;
      randomised to receive Exenatide injections, or to act as controls in this open label trial.&#xD;
      For half of the patients, Exenatide will be given as a once weekly subcutaneous injection in&#xD;
      addition to participant's regular medication. All patients will continue to receive standard&#xD;
      of care treatment for MSA. Detailed assessments will be made of all patients at baseline and&#xD;
      periodically for a total of 48 weeks. The primary endpoint will be the difference in total&#xD;
      Uniﬁed Multiple System Atrophy Rating Scale (UMSARS) score (Parts I and II) at 48 weeks&#xD;
      comparing Exenatide treated to best medically treated patients (controls). Secondary measures&#xD;
      will include adverse event reports, self-completed questionnaires, and blood test results.&#xD;
      Aside from these assessments, all patients will continue any regular MSA medications&#xD;
      throughout the trial with adjustments made only according to clinical need.&#xD;
&#xD;
      Standard of care treatment for patients on non IMP arm will be dependant on the patients&#xD;
      individual symptoms - there is no broad standard treatment for every patient.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Fifty patients with early stage MSA will be recruited and randomised to receive Exenatide&#xD;
      injections, or to act as controls in this open label trial.&#xD;
&#xD;
      Once a potential participant has been identified they will receive a patient information&#xD;
      leaflet, and will be given a minimum of 24 hours to read this before being recruited on to&#xD;
      the trial. Patients will need to be eligible for the trial by meeting the inclusion criteria.&#xD;
&#xD;
      During pre-treatment there will be a screening visit and a baseline visit. Pre-treatment&#xD;
      assessments will include: demographics, medical history, family history, any previous genetic&#xD;
      tests recorded, previous drug compliance issues recorded, physical examination, neurological&#xD;
      examination, 12-lead ECG, routine bloods (FBC, U&amp;E, LFT, glucose, amylase, HbA1c, PT and&#xD;
      APTT), height, weight, vital signs, serum or urine pregnancy tests (for women of childbearing&#xD;
      potential), MoCA, BDI-II and Concomitant medications. Patients will then wear a sensor&#xD;
      attached to their lower back for a week. They will then return for their baseline visit. At&#xD;
      the baseline visit assessments will include: physical exam, neurological exam, lumbar&#xD;
      puncture for CSF collection, serum collection, fasting blood tests, vital signs, UMSARS,&#xD;
      COMPASS Select, COMPASS Change scale, timed motor tests, The Unified Dystonia Rating Scale,&#xD;
      MoCA, BDI-II, Concomitant medication review and adverse event review. Participants will then&#xD;
      be randomised to both control arm or trial drug arm and receive the according treatment. The&#xD;
      baseline visit will also include a training session for self-administration of IMP.&#xD;
&#xD;
      Patients randomised to receive the trial drug will receive 2mg Exenatide once a week for 48&#xD;
      weeks via subcutaneous injection. Follow up visits will be every 12 weeks and patients will&#xD;
      be given a sufficient supply to last them till their next follow up appointment (can be&#xD;
      stored in fridge at home). They will also be given a dosing diary to record the time and day&#xD;
      of injection administration.&#xD;
&#xD;
      Patients will continue to attend their normal neurology appointments as well as trial&#xD;
      specific appointments. Patients will have a telephone call with the research nurse at week 4.&#xD;
      Thereafter detailed assessments including Physical and Neurological exam, ECG's, Movement&#xD;
      tests Including the Uniﬁed Multiple System Atrophy Rating Scale (videotaped), concomitant&#xD;
      medications review, adverse event review, and blood sampling at baseline and every 12 weeks&#xD;
      for a total of 48 weeks. Each patient will also have a Lumbar Puncture at baseline and at&#xD;
      their final visit.&#xD;
&#xD;
      The primary endpoint will be the difference in total Uniﬁed Multiple System Atrophy Rating&#xD;
      Scale (UMSARS) score (Parts I and II) at 48 weeks comparing Exenatide to best medically&#xD;
      treated patients. Secondary measures will include adverse event reports, self-completed&#xD;
      questionnaires, and blood test results. Aside from these assessments, all patients will&#xD;
      continue any regular MSA medications throughout the trial with adjustments made only&#xD;
      according to clinical need.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date type="Actual">September 16, 2020</start_date>
  <completion_date type="Anticipated">April 26, 2022</completion_date>
  <primary_completion_date type="Anticipated">April 26, 2022</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>To collect data to estimate the effectiveness of Exenatide in modifying disease progression of patients with Multiple System Atrophy</measure>
    <time_frame>48 weeks</time_frame>
    <description>The primary endpoint will be the difference in total Unified Multiple System Atrophy Rating Scale (UMSARS) score (Parts I and II)</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>The proportion of patients with loss of independent ambulation by the end of the study</measure>
    <time_frame>48 weeks</time_frame>
    <description>Defined by the score of 3 or more in UMSARS-I item 7 - walking</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The difference in the Multiple System Atrophy Quality of Life (MSA QoL) scale</measure>
    <time_frame>48 weeks</time_frame>
    <description>Defined by completion of QoL scale</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The difference at week 48 in UMSARS III and IV (The proportion of patients with loss of independent ambulation by the end of the study)</measure>
    <time_frame>48 weeks</time_frame>
    <description>Defined by UMSARS III and IV</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The difference in anti-parkinsonian or anti-orthostatic hypotension drugs</measure>
    <time_frame>48 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The number of falls</measure>
    <time_frame>48 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The proportion of patients reaching a score of 3 or more on UMSARS-I items 1 (speech), 2 (swallowing) and 8 (falling)</measure>
    <time_frame>48 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The difference at week 48 in clinical global impression (CGI); difference at week 48 in Montreal Cognitive Assessment (MoCA) scores</measure>
    <time_frame>48 weeks</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">50</enrollment>
  <condition>Multiple System Atrophy</condition>
  <arm_group>
    <arm_group_label>Exenatide</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Standard of care</arm_group_label>
    <arm_group_type>No Intervention</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Exenatide Pen Injector [Bydureon]</intervention_name>
    <description>Exenatide is a treatment licensed for use in Type 2 diabetes.</description>
    <arm_group_label>Exenatide</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Participants aged 30-80 years old with a diagnosis of Possible or Probable MSA of the&#xD;
             parkinsonian subtype (MSA-P) or cerebellar subtype (MSA-C) according to The Gilman&#xD;
             Criteria (Gilman et al. 2008).&#xD;
&#xD;
          -  Participants who are less than five years from the time of documented MSA diagnosis or&#xD;
             from the time of documented parkinsonian / ataxic neurological condition that later&#xD;
             turns out to be MSA.&#xD;
&#xD;
          -  Participants who are able to walk at least 10 metres with or without assistance.&#xD;
             Participants with an anticipated survival of at least three years in the opinion of&#xD;
             the investigator.&#xD;
&#xD;
          -  Participants that are willing to adhere to the study drug regimen.&#xD;
&#xD;
          -  Participants that are willing and able to perform all protocol-specified assessments&#xD;
             and comply with the study visit schedule.&#xD;
&#xD;
          -  Females of childbearing potential and male participants with partners of childbearing&#xD;
             potential must agree to use an effective method of contraception from the time consent&#xD;
             is signed until 10 weeks after treatment discontinuation. Females of childbearing&#xD;
             potential have a negative pregnancy test within 7 days prior to being randomised.&#xD;
&#xD;
          -  Willing and able to provide written informed consent.&#xD;
&#xD;
          -  Subjects who are not able to write may give verbal consent in the presence of at least&#xD;
             one witness, and the witness should sign the informed consent form.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Females who are pregnant, planning pregnancy or breastfeeding.&#xD;
&#xD;
          -  Women of child-bearing potential who do not practice an acceptable method of birth&#xD;
             control. Subjects who meet any of the following criteria which tend to suggest advance&#xD;
             disease:&#xD;
&#xD;
               1. Speech impairment as assessed by a score of ≥ 3 on UMSARS question 1&#xD;
&#xD;
               2. Swallowing impairment as assessed by a score of ≥ 3 on UMSARS question 2&#xD;
&#xD;
               3. Impairment in ambulation as assessed by a score of ≥ 3 on UMSARS question 7&#xD;
&#xD;
               4. Falling more frequently than once per week as assessed by a score of ≥ 3 on&#xD;
                  UMSARS question 8. Participants with a clinically significant or unstable medical&#xD;
                  or surgical condition, which in the opinion of the investigator might preclude&#xD;
                  safe completion of the study.&#xD;
&#xD;
          -  Participants with active malignant neoplasms or history of malignant neoplasm in the&#xD;
             last 5 years. Participants with movement disorders other than MSA.&#xD;
&#xD;
          -  Concurrent dementia defined by a score lower than 21 on the MoCA.&#xD;
&#xD;
          -  Concurrent severe depression defined by a score of ≥30 on the Beck Depression&#xD;
             Inventory-II.&#xD;
&#xD;
          -  History of deep brain stimulation surgery.&#xD;
&#xD;
          -  Participants who have taken any investigational products within 90 days prior to&#xD;
             baseline.&#xD;
&#xD;
          -  Participants with a BMI &lt; 18.5.&#xD;
&#xD;
          -  Participants with diabetes, end stage renal disease or severely impaired renal&#xD;
             function.&#xD;
&#xD;
          -  History of clinically significant cardiac disease, pancreatitis and/or alcoholism.&#xD;
&#xD;
          -  Participants with severe gastrointestinal disease including gastroparesis.&#xD;
&#xD;
          -  Ongoing treatment with sulphonylurea.&#xD;
&#xD;
          -  Known allergies to the IMP and excipients of IMP.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>30 Years</minimum_age>
    <maximum_age>80 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_contact>
    <last_name>Tom Foltynie</last_name>
    <phone>020 3448 4531</phone>
    <email>t.foltynie@ucl.ac.uk</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Dilan Athauda</last_name>
    <email>d.athauda@nhs.net</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Leonard Wolfson Experimental Neurology Centre, National Hospital of Neurology and Neurosurgery, UCLH NHS Foundation trust</name>
      <address>
        <city>London</city>
        <zip>WC1N 3BG</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Tom Foltynie</last_name>
      <email>tom.foltynie@nhs.net</email>
    </contact>
    <contact_backup>
      <last_name>Shazia Begum</last_name>
      <email>shazia.begum4@nhs.net</email>
    </contact_backup>
  </location>
  <location_countries>
    <country>United Kingdom</country>
  </location_countries>
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    <citation>Poewe W, Seppi K, Fitzer-Attas CJ, Wenning GK, Gilman S, Low PA, Giladi N, Barone P, Sampaio C, Eyal E, Rascol O; Rasagiline-for-MSA investigators. Efficacy of rasagiline in patients with the parkinsonian variant of multiple system atrophy: a randomised, placebo-controlled trial. Lancet Neurol. 2015 Feb;14(2):145-52. doi: 10.1016/S1474-4422(14)70288-1. Epub 2014 Dec 8.</citation>
    <PMID>25498732</PMID>
  </reference>
  <reference>
    <citation>Talbot K, Wang HY, Kazi H, Han LY, Bakshi KP, Stucky A, Fuino RL, Kawaguchi KR, Samoyedny AJ, Wilson RS, Arvanitakis Z, Schneider JA, Wolf BA, Bennett DA, Trojanowski JQ, Arnold SE. Demonstrated brain insulin resistance in Alzheimer's disease patients is associated with IGF-1 resistance, IRS-1 dysregulation, and cognitive decline. J Clin Invest. 2012 Apr;122(4):1316-38. doi: 10.1172/JCI59903.</citation>
    <PMID>22476197</PMID>
  </reference>
  <reference>
    <citation>Wenning GK, Tison F, Seppi K, Sampaio C, Diem A, Yekhlef F, Ghorayeb I, Ory F, Galitzky M, Scaravilli T, Bozi M, Colosimo C, Gilman S, Shults CW, Quinn NP, Rascol O, Poewe W; Multiple System Atrophy Study Group. Development and validation of the Unified Multiple System Atrophy Rating Scale (UMSARS). Mov Disord. 2004 Dec;19(12):1391-402. doi: 10.1002/mds.20255.</citation>
    <PMID>15452868</PMID>
  </reference>
  <reference>
    <citation>Kollensperger M, Stampfer-Kountchev M, Seppi K, Geser F, Frick C, Del Sorbo F, Albanese A, Gurevich T, Giladi N, Djaldetti R, Schrag A, Low PA, Mathias CJ, Poewe W, Wenning GK. Progression of dysautonomia in multiple system atrophy: a prospective study of self-perceived impairment. Eur J Neurol. 2007 Jan;14(1):66-72. doi: 10.1111/j.1468-1331.2006.01554.x.</citation>
    <PMID>17222116</PMID>
  </reference>
  <reference>
    <citation>Wenning GK, Geser F, Krismer F, Seppi K, Duerr S, Boesch S, Kollensperger M, Goebel G, Pfeiffer KP, Barone P, Pellecchia MT, Quinn NP, Koukouni V, Fowler CJ, Schrag A, Mathias CJ, Giladi N, Gurevich T, Dupont E, Ostergaard K, Nilsson CF, Widner H, Oertel W, Eggert KM, Albanese A, del Sorbo F, Tolosa E, Cardozo A, Deuschl G, Hellriegel H, Klockgether T, Dodel R, Sampaio C, Coelho M, Djaldetti R, Melamed E, Gasser T, Kamm C, Meco G, Colosimo C, Rascol O, Meissner WG, Tison F, Poewe W; European Multiple System Atrophy Study Group. The natural history of multiple system atrophy: a prospective European cohort study. Lancet Neurol. 2013 Mar;12(3):264-74. doi: 10.1016/S1474-4422(12)70327-7. Epub 2013 Feb 5.</citation>
    <PMID>23391524</PMID>
  </reference>
  <verification_date>November 2020</verification_date>
  <study_first_submitted>May 29, 2020</study_first_submitted>
  <study_first_submitted_qc>June 11, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 16, 2020</study_first_posted>
  <last_update_submitted>November 4, 2020</last_update_submitted>
  <last_update_submitted_qc>November 4, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">November 5, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Multiple System Atrophy</mesh_term>
    <mesh_term>Shy-Drager Syndrome</mesh_term>
    <mesh_term>Atrophy</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Exenatide</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
    <ipd_description>Anonymised data will be made available from those patients who have provided consent to do so, following completion of trial data analysis, for any reasonable requests.</ipd_description>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT04431713
org study id: 125591
secondary id: 2020-000122-26
nct id: NCT04431713
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
is us export: No
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.
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. Pre-treatment
      assessments will include: demographics, medical history, family history, any previous genetic
      tests recorded, previous drug compliance issues recorded, physical examination, neurological
      examination, 12-lead ECG, routine bloods (FBC, U&E, LFT, glucose, amylase, HbA1c, PT and
      APTT), height, weight, vital signs, serum or urine pregnancy tests (for women of childbearing
      potential), MoCA, BDI-II and Concomitant medications. Patients will then wear a sensor
      attached to their lower back for a week. They will then return for their baseline visit. At
      the baseline visit assessments will include: physical exam, neurological exam, lumbar
      puncture for CSF collection, serum collection, fasting blood tests, vital signs, UMSARS,
      COMPASS Select, COMPASS Change scale, timed motor tests, The Unified Dystonia Rating Scale,
      MoCA, BDI-II, Concomitant medication review and adverse event review. Participants will then
      be randomised to both control arm or trial drug arm and receive the according treatment. The
      baseline visit will also include a training session for self-administration of IMP.

      Patients randomised to receive the trial drug will receive 2mg Exenatide once a week for 48
      weeks via subcutaneous injection. Follow up visits will be every 12 weeks and patients will
      be given a sufficient supply to last them till their next follow up appointment (can be
      stored in fridge at home). They will also be given a dosing diary to record the time and day
      of injection administration.

      Patients will continue to attend their normal neurology appointments as well as trial
      specific appointments. Patients will have a telephone call with the research nurse at week 4.
      Thereafter detailed assessments including Physical and Neurological exam, ECG's, Movement
      tests Including the Uniﬁed Multiple System Atrophy Rating Scale (videotaped), concomitant
      medications review, adverse event review, and blood sampling at baseline and every 12 weeks
      for a total of 48 weeks. Each patient will also have a Lumbar Puncture at baseline and at
      their final visit.

      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 to best medically
      treated patients. 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.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: To collect data to estimate the effectiveness of Exenatide in modifying disease progression of patients with Multiple System Atrophy
time frame: 48 weeks
description: The primary endpoint will be the difference in total Unified Multiple System Atrophy Rating Scale (UMSARS) score (Parts I and II)
measure: The proportion of patients with loss of independent ambulation by the end of the study
time frame: 48 weeks
description: Defined by the score of 3 or more in UMSARS-I item 7 - walking
measure: The difference in the Multiple System Atrophy Quality of Life (MSA QoL) scale
time frame: 48 weeks
description: Defined by completion of QoL scale
measure: The difference at week 48 in UMSARS III and IV (The proportion of patients with loss of independent ambulation by the end of the study)
time frame: 48 weeks
description: Defined by UMSARS III and IV
measure: The difference in anti-parkinsonian or anti-orthostatic hypotension drugs
time frame: 48 weeks
measure: The number of falls
time frame: 48 weeks
measure: The proportion of patients reaching a score of 3 or more on UMSARS-I items 1 (speech), 2 (swallowing) and 8 (falling)
time frame: 48 weeks
measure: The difference at week 48 in clinical global impression (CGI); difference at week 48 in Montreal Cognitive Assessment (MoCA) scores
time frame: 48 weeks
arm group label: Exenatide
arm group type: Experimental
arm group label: Standard of care
arm group type: No Intervention
intervention type: Drug
intervention name: Exenatide Pen Injector [Bydureon]
description: Exenatide is a treatment licensed for use in Type 2 diabetes.
arm group label: Exenatide
criteria: 
gender: All
minimum age: 30 Years
maximum age: 80 Years
healthy volunteers: No
last name: Tom Foltynie
phone: 020 3448 4531
email: t.foltynie@ucl.ac.uk
last name: Dilan Athauda
email: d.athauda@nhs.net
facility: 
status: Recruiting
contact: 
contact backup: 
country: United Kingdom
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PMID: 27713036
citation: Athauda D, Foltynie T. The glucagon-like peptide 1 (GLP) receptor as a therapeutic target in Parkinson's disease: mechanisms of action. Drug Discov Today. 2016 May;21(5):802-18. doi: 10.1016/j.drudis.2016.01.013. Epub 2016 Feb 3.
PMID: 26851597
citation: Athauda D, Gulyani S, Karnati HK, Li Y, Tweedie D, Mustapic M, Chawla S, Chowdhury K, Skene SS, Greig NH, Kapogiannis D, Foltynie T. Utility of Neuronal-Derived Exosomes to Examine Molecular Mechanisms That Affect Motor Function in Patients With Parkinson Disease: A Secondary Analysis of the Exenatide-PD Trial. JAMA Neurol. 2019 Apr 1;76(4):420-429. doi: 10.1001/jamaneurol.2018.4304. Erratum In: JAMA Neurol. 2019 Apr 1;76(4):509.
PMID: 30640362
citation: Athauda D, Maclagan K, Budnik N, Zampedri L, Hibbert S, Skene SS, Chowdhury K, Aviles-Olmos I, Limousin P, Foltynie T. What Effects Might Exenatide have on Non-Motor Symptoms in Parkinson's Disease: A Post Hoc Analysis. J Parkinsons Dis. 2018;8(2):247-258. doi: 10.3233/JPD-181329.
PMID: 29843254
citation: Athauda D, Maclagan K, Skene SS, Bajwa-Joseph M, Letchford D, Chowdhury K, Hibbert S, Budnik N, Zampedri L, Dickson J, Li Y, Aviles-Olmos I, Warner TT, Limousin P, Lees AJ, Greig NH, Tebbs S, Foltynie T. Exenatide once weekly versus placebo in Parkinson's disease: a randomised, double-blind, placebo-controlled trial. Lancet. 2017 Oct 7;390(10103):1664-1675. doi: 10.1016/S0140-6736(17)31585-4. Epub 2017 Aug 3.
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citation: Aviles-Olmos I, Dickson J, Kefalopoulou Z, Djamshidian A, Ell P, Soderlund T, Whitton P, Wyse R, Isaacs T, Lees A, Limousin P, Foltynie T. Exenatide and the treatment of patients with Parkinson's disease. J Clin Invest. 2013 Jun;123(6):2730-6. doi: 10.1172/JCI68295.
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citation: Bassil, Fares, Marie-Hélène Canron, Anne Vital, Erwan Bezard, Pierre-Olivier Fernagut, and Wassilios G. Meissner. 2017. "Brain Insulin Resistance in Parkinson's Disease [MDS Abstracts]." Movement Disorders 32 (suppl.
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citation: Poewe W, Seppi K, Fitzer-Attas CJ, Wenning GK, Gilman S, Low PA, Giladi N, Barone P, Sampaio C, Eyal E, Rascol O; Rasagiline-for-MSA investigators. Efficacy of rasagiline in patients with the parkinsonian variant of multiple system atrophy: a randomised, placebo-controlled trial. Lancet Neurol. 2015 Feb;14(2):145-52. doi: 10.1016/S1474-4422(14)70288-1. Epub 2014 Dec 8.
PMID: 25498732
citation: Talbot K, Wang HY, Kazi H, Han LY, Bakshi KP, Stucky A, Fuino RL, Kawaguchi KR, Samoyedny AJ, Wilson RS, Arvanitakis Z, Schneider JA, Wolf BA, Bennett DA, Trojanowski JQ, Arnold SE. Demonstrated brain insulin resistance in Alzheimer's disease patients is associated with IGF-1 resistance, IRS-1 dysregulation, and cognitive decline. J Clin Invest. 2012 Apr;122(4):1316-38. doi: 10.1172/JCI59903.
PMID: 22476197
citation: Wenning GK, Tison F, Seppi K, Sampaio C, Diem A, Yekhlef F, Ghorayeb I, Ory F, Galitzky M, Scaravilli T, Bozi M, Colosimo C, Gilman S, Shults CW, Quinn NP, Rascol O, Poewe W; Multiple System Atrophy Study Group. Development and validation of the Unified Multiple System Atrophy Rating Scale (UMSARS). Mov Disord. 2004 Dec;19(12):1391-402. doi: 10.1002/mds.20255.
PMID: 15452868
citation: Kollensperger M, Stampfer-Kountchev M, Seppi K, Geser F, Frick C, Del Sorbo F, Albanese A, Gurevich T, Giladi N, Djaldetti R, Schrag A, Low PA, Mathias CJ, Poewe W, Wenning GK. Progression of dysautonomia in multiple system atrophy: a prospective study of self-perceived impairment. Eur J Neurol. 2007 Jan;14(1):66-72. doi: 10.1111/j.1468-1331.2006.01554.x.
PMID: 17222116
citation: Wenning GK, Geser F, Krismer F, Seppi K, Duerr S, Boesch S, Kollensperger M, Goebel G, Pfeiffer KP, Barone P, Pellecchia MT, Quinn NP, Koukouni V, Fowler CJ, Schrag A, Mathias CJ, Giladi N, Gurevich T, Dupont E, Ostergaard K, Nilsson CF, Widner H, Oertel W, Eggert KM, Albanese A, del Sorbo F, Tolosa E, Cardozo A, Deuschl G, Hellriegel H, Klockgether T, Dodel R, Sampaio C, Coelho M, Djaldetti R, Melamed E, Gasser T, Kamm C, Meco G, Colosimo C, Rascol O, Meissner WG, Tison F, Poewe W; European Multiple System Atrophy Study Group. The natural history of multiple system atrophy: a prospective European cohort study. Lancet Neurol. 2013 Mar;12(3):264-74. doi: 10.1016/S1474-4422(12)70327-7. Epub 2013 Feb 5.
PMID: 23391524
responsible party type: Sponsor
mesh term: Multiple System Atrophy
mesh term: Shy-Drager Syndrome
mesh term: Atrophy
mesh term: Exenatide
sharing ipd: No
ipd description: Anonymised data will be made available from those patients who have provided consent to do so, following completion of trial data analysis, for any reasonable requests.