Exenatide Once-weekly as a Treatment for Multiple System Atrophy (MSA)
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|ClinicalTrials.gov Identifier: NCT04431713|
Recruitment Status : Recruiting
First Posted : June 16, 2020
Last Update Posted : November 5, 2020
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.
|Condition or disease||Intervention/treatment||Phase|
|Multiple System Atrophy||Drug: Exenatide Pen Injector [Bydureon]||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||September 16, 2020|
|Estimated Primary Completion Date :||April 26, 2022|
|Estimated Study Completion Date :||April 26, 2022|
Drug: Exenatide Pen Injector [Bydureon]
Exenatide is a treatment licensed for use in Type 2 diabetes.
|No Intervention: Standard of care|
- To collect data to estimate the effectiveness of Exenatide in modifying disease progression of patients with Multiple System Atrophy [ Time Frame: 48 weeks ]The primary endpoint will be the difference in total Unified Multiple System Atrophy Rating Scale (UMSARS) score (Parts I and II)
- The proportion of patients with loss of independent ambulation by the end of the study [ Time Frame: 48 weeks ]Defined by the score of 3 or more in UMSARS-I item 7 - walking
- The difference in the Multiple System Atrophy Quality of Life (MSA QoL) scale [ Time Frame: 48 weeks ]Defined by completion of QoL scale
- 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 ]Defined by UMSARS III and IV
- The difference in anti-parkinsonian or anti-orthostatic hypotension drugs [ Time Frame: 48 weeks ]
- The number of falls [ Time Frame: 48 weeks ]
- 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 ]
- The difference at week 48 in clinical global impression (CGI); difference at week 48 in Montreal Cognitive Assessment (MoCA) scores [ Time Frame: 48 weeks ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04431713
|Contact: Tom Foltynie||020 3448 firstname.lastname@example.org|
|Contact: Dilan Athaudaemail@example.com|
|Leonard Wolfson Experimental Neurology Centre, National Hospital of Neurology and Neurosurgery, UCLH NHS Foundation trust||Recruiting|
|London, United Kingdom, WC1N 3BG|
|Contact: Tom Foltynie firstname.lastname@example.org|
|Contact: Shazia Begum email@example.com|