Exenatide Once-weekly as a Treatment for Multiple System Atrophy
NCT ID: NCT04431713
Last Updated: 2025-06-27
Study Results
Results available
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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Recruitment Status
COMPLETED
Clinical Phase
PHASE2
Total Enrollment
50 participants
Study Classification
INTERVENTIONAL
Study Start Date
2020-09-16
Study Completion Date
2024-03-22
Brief Summary
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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.
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.
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Detailed Description
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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.
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.
Conditions
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Multiple System Atrophy
Study Design
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Allocation Method
RANDOMIZED
Intervention Model
PARALLEL
Primary Study Purpose
TREATMENT
Blinding Strategy
NONE
Study Groups
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Exenatide
Group Type
EXPERIMENTAL
Exenatide Pen Injector [Bydureon]
Intervention Type
DRUG
Exenatide is a treatment licensed for use in Type 2 diabetes.
Standard of care
Group Type
NO_INTERVENTION
No interventions assigned to this group
Interventions
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Exenatide Pen Injector [Bydureon]
Exenatide is a treatment licensed for use in Type 2 diabetes.
Intervention Type
DRUG
Eligibility Criteria
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Inclusion Criteria
* 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.
* 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.
Exclusion Criteria
* 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:
1. Speech impairment as assessed by a score of ≥ 3 on UMSARS question 1
2. Swallowing impairment as assessed by a score of ≥ 3 on UMSARS question 2
3. Impairment in ambulation as assessed by a score of ≥ 3 on UMSARS question 7
4. 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.
* Ongoing treatment with sulphonylurea.
* Known allergies to the IMP and excipients of IMP.
* 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:
1. Speech impairment as assessed by a score of ≥ 3 on UMSARS question 1
2. Swallowing impairment as assessed by a score of ≥ 3 on UMSARS question 2
3. Impairment in ambulation as assessed by a score of ≥ 3 on UMSARS question 7
4. 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.
* Ongoing treatment with sulphonylurea.
* Known allergies to the IMP and excipients of IMP.
Minimum Eligible Age
30 Years
Maximum Eligible Age
80 Years
Eligible Sex
ALL
Accepts Healthy Volunteers
No
Sponsors
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University College, London
OTHER
Sponsor Role
lead
Responsible Party
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Responsibility Role
SPONSOR
Locations
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Leonard Wolfson Experimental Neurology Centre, National Hospital of Neurology and Neurosurgery, UCLH NHS Foundation trust
London, , United Kingdom
Site Status
Countries
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United Kingdom
References
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BACKGROUND
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2020-000122-26
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
125591
Identifier Type: -
Identifier Source: org_study_id
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PHASE4
A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)
NCT04729907
ACTIVE_NOT_RECRUITING
PHASE3
Adults With SMA Treated With Nusinersen
NCT04591678
COMPLETED
Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
NCT03943290
TERMINATED
PHASE2
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
NCT02193074
TERMINATED
PHASE3
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
NCT02462759
TERMINATED
PHASE2
A Biomarker Study of ATH434 in Participants With MSA
NCT05864365
COMPLETED
PHASE2
Study of ATH434 in Participants with Multiple System Atrophy
NCT05109091
COMPLETED
PHASE2
Safety Study of Oral Edaravone Administered in Subjects With ALS
NCT04165824
COMPLETED
PHASE3
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
NCT05996003
RECRUITING
PHASE2
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
NCT06280209
RECRUITING
PHASE1/PHASE2
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
NCT02386553
COMPLETED
PHASE2
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)
NCT02052791
COMPLETED
PHASE1
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
NCT05540860
ACTIVE_NOT_RECRUITING
PHASE2
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
NCT01494701
COMPLETED
PHASE1
A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
NCT02056808
COMPLETED
PHASE1
Compassionate Treatment : An Exploratory Clinical Trial to Assess Treatment of Amyotrophic Lateral Sclerosis
NCT02383654
COMPLETED
PHASE1
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
NCT04089566
COMPLETED
PHASE3
Phase 3 Extension Study of Dexpramipexole in ALS
NCT01622088
TERMINATED
PHASE3