Parkinson's DiseasePhase 2NCT01174810
Exendin-4 as a Treatment for Parkinson's Disease - Pilot Study
A Phase 2 study testing Exenatide in people with Parkinson's Disease, run by University College, London at 1 site in United Kingdom.
Trial summary
Exenatide is a licensed, safe and effective treatment for patients with Diabetes mellitus. Laboratory work has shown strong, reproducible evidence that this drug has beneficial "disease modifying" effects when given to animals with a range of experimental models of Parkinson's disease (PD). Read more about this study
- Who is it for?
- Ages 45 to 70 years
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- Exenatide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Jul 2010to Mar 2013
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 45 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of Idiopathic Parkinson's disease of moderate severity- equivalent to Hoehn/ Yahr stage 2 to 2.5 (Bilateral symptoms but still physically independent).
- Male or female. Female patients to be post menopausal (defined as 12 months of spontaneous amenorrhoea or 6 months spontaneous amenorrhoea with FSH levels greater than 40mIU/ml), surgically sterilised (post hysterectomy and/or oophorectomy). Male patients with female partners that have child bearing potential must use adequate contraception (condoms +/-spermicidal gel/foam) throughout the duration of the trial period.
- Age 45-70 years
- Disease onset after age 40 years
- Disease duration > 5 years
- On L-dopa treatment. Patient must be on oral L-dopa treatment - with or without dopamine agonist including Apomorphine, MAO-B inhibitor, COMT inhibitor, Amantadine, Beta blocker, anticholinergic treatment History of wearing off phenomena- duration of action of single dose of L-dopa < 6 hours Stable PD medication for preceding 3 months- i.e. no change in medication type or dose.
Show all 10Show fewer
- UPDRS motor off medication score >15
- L-dopa responsiveness. Defined as >33% improvement in UPDRS motor off medication score following L-dopa challenge
- Able to give informed consent
- Able to comply with trial protocol and willing to attend necessary clinic visits off medication.
Who cannot join
- Diagnosis or suspicion of other cause for parkinsonism including Vascular parkinsonism, post traumatic parkinsonism, drug or toxin induced parkinsonism, or other neurodegenerative condition including Multiple System Atrophy, Progressive Supranuclear Palsy, Huntington's disease, Wilson's disease, Pantothenate kinase Neurodegeneration (PKAN), Alzheimer's disease, Creutzfeld Jacob disease.
- Known abnormality on CT or MRI brain imaging considered to be causing symptoms or signs of neurological dysfunction, or considered likely to compromise compliance with trial protocol.
- Concurrent dementia defined by a score lower than 120 on the Mattis Dementia Rating Scale.
- Concurrent severe depression defined by a score greater than 16 on the MADRS Exposure to neuroleptic drugs within 6 months prior to baseline assessment Prior intracerebral surgical intervention for Parkinson's disease including Deep Brain stimulation, lesional surgery, growth factor administration, gene therapy or cell transplant
- Already actively participating in a trial of a device, drug or surgical treatment for Parkinson's disease, or trial participation within previous 30 days.
- Type 1 Diabetes mellitus
Show all 14Show fewer
- Type 2 Diabetes mellitus on insulin treatment
- End stage renal disease or severely impaired renal function with creatinine clearance <30ml/min
- History of severe cardiac disease (Angina, Myocardial infarction or cardiac surgery in preceding 2 years)
- History of pancreatitis
- History of alcoholism
- Severe gastrointestinal disease including gastroparesis
- Ongoing treatment with sulphonylurea
- Females that are pregnant or breast feeding or of child bearing potential.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
National Hospital for Neurology and Neurosurgery
London, WC1E 3BG
About this study
Official title: An Open Label, Single Site, 12 Month, Phase II, Randomised Controlled Trial Evaluating the Safety and Efficacy of Exendin-4 (Exenatide) in the Treatment of Patients With Moderate Severity Parkinson's Disease.
Exenatide is a licensed, safe and effective treatment for patients with Diabetes mellitus. Laboratory work has shown strong, reproducible evidence that this drug has beneficial "disease modifying" effects when given to animals with a range of experimental models of Parkinson's disease (PD). This project aims to make an initial evaluation of possible benefits of Exenatide among patients with moderate symptoms of PD. The drug will be given as a twice daily 10microgram injection under the skin in a similar way to one of the conventional "symptomatic" treatments for PD (Apomorphine).
Forty patients with moderate symptoms of PD will be recruited and randomised to receive Exenatide injections twice daily, or to act as controls in this open label trial. Detailed assessments will be made of all patients at baseline and periodically for a total of 14 months. The primary outcome measure will be the change between baseline and follow up, in the severity of a validated PD assessment scale (the UPDRS part 3 motor score) after an overnight period free of conventional PD medication. Secondary measures will include adverse event reports, self completed questionnaires, and blood test results. Aside from these assessments, all patients will continue their regular PD medications throughout the trial with adjustments made only according to clinical need.
In a subgroup of patients (n=10), brain scans that assess the severity of PD, will be performed at both baseline and follow up to help understand possible mechanisms of action of Exenatide.
- Conditions
- Parkinson's Disease
- Being tested
- Exenatide
- Keywords
- Parkinson's Disease, PD, Moderate Parkinson's Disease
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExenatideDrug
Also called Byetta, Exendin-4
Exenatide, 5 micrograms twice a day for 1 month and 10 micrograms twice a day for 11 months. Total duration of treatment 12 months
| Study group | Role | What they receive |
|---|---|---|
| Control | No intervention | Not described |
| Exenatide | Experimental | Not described Receives: Drug: Exenatide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline to 12 months & 14 months between patients on active Exenatide treatment and PD controls in respect of their UPDRS-off-medication motor subscore.
Secondary outcomes (1)
Adverse event profile among patients treated with Exenatide compared with matched PD controls. Change from baseline to 12 months/ 14 months between patients on active treatment and PD controls in respect of list given below
- the UPDRS on medication motor subscore
- the UPDRS ADL subscore
- dyskinesia rating scale
- timed motor tests
- the Mattis Dementia rating scale
- the Montgomery \& Asberg Depression rating scale
- the PDQ39
- the EQ-5D
- the NMS Quest
- the SCOPA Sleep scale
- the SCOPA AUT scale
- the Smell Identification test
- DAT (SPECT) scan appearances.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2010 (estimated)
- Study startJul 2010
- Primary completionMar 2013 (estimated)
- Study completionMar 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- More trials run by University College, LondonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University College, London
- Sponsor type
- University, hospital or other
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01174810
- Sponsor's protocol ID
- 09/0391
- Other IDs
- 2009-018137-37, 2009
- Status last verified
- Mar 2012
- First submitted
- 2 Aug 2010
- Last updated
- 23 Mar 2012
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 23728174
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Single Site, 12 Month, Phase II, Randomised Controlled Trial Evaluating the Safety and Efficacy of Exendin-4 (Exenatide) in the Treatment of Patients With Moderate Severity Parkinson's Disease.
- ClinicalTrials.gov ID
- NCT01174810
- Acronym / sponsor ID
- 09/0391
- Conditions
- Parkinson's Disease
- Keywords
- Parkinson's Disease, PD, Moderate Parkinson's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- University College, LondonUniversity, hospital or other
- Study start
- Jul 2010
- Primary completion
- Mar 2013 (estimated)
- Study completion
- Mar 2013 (estimated)
- First posted
- 4 Aug 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Exenatide is a licensed, safe and effective treatment for patients with Diabetes mellitus. Laboratory work has shown strong, reproducible evidence that this drug has beneficial "disease modifying" effects when given to animals with a range of experimental models of Parkinson's disease (PD). This project aims to make an initial evaluation of possible benefits of Exenatide among patients with moderate symptoms of PD. The drug will be given as a twice daily 10microgram injection under the skin in a similar way to one of the conventional "symptomatic" treatments for PD (Apomorphine).
Forty patients with moderate symptoms of PD will be recruited and randomised to receive Exenatide injections twice daily, or to act as controls in this open label trial. Detailed assessments will be made of all patients at baseline and periodically for a total of 14 months. The primary outcome measure will be the change between baseline and follow up, in the severity of a validated PD assessment scale (the UPDRS part 3 motor score) after an overnight period free of conventional PD medication. Secondary measures will include adverse event reports, self completed questionnaires, and blood test results. Aside from these assessments, all patients will continue their regular PD medications throughout the trial with adjustments made only according to clinical need.
In a subgroup of patients (n=10), brain scans that assess the severity of PD, will be performed at both baseline and follow up to help understand possible mechanisms of action of Exenatide.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control | No intervention | – |
| Exenatide | Experimental | Drug: Exenatide |
Who may qualify
- Ages
- 45 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Diagnosis of Idiopathic Parkinson's disease of moderate severity- equivalent to Hoehn/ Yahr stage 2 to 2.5 (Bilateral symptoms but still physically independent).
- Male or female. Female patients to be post menopausal (defined as 12 months of spontaneous amenorrhoea or 6 months spontaneous amenorrhoea with FSH levels greater than 40mIU/ml), surgically sterilised (post hysterectomy and/or oophorectomy). Male patients with female partners that have child bearing potential must use adequate contraception (condoms +/-spermicidal gel/foam) throughout the duration of the trial period.
- Age 45-70 years
- Disease onset after age 40 years
- Disease duration > 5 years
Key exclusion criteria (14 listed)
- Diagnosis or suspicion of other cause for parkinsonism including Vascular parkinsonism, post traumatic parkinsonism, drug or toxin induced parkinsonism, or other neurodegenerative condition including Multiple System Atrophy, Progressive Supranuclear Palsy, Huntington's disease, Wilson's disease, Pantothenate kinase Neurodegeneration (PKAN), Alzheimer's disease, Creutzfeld Jacob disease.
- Known abnormality on CT or MRI brain imaging considered to be causing symptoms or signs of neurological dysfunction, or considered likely to compromise compliance with trial protocol.
- Concurrent dementia defined by a score lower than 120 on the Mattis Dementia Rating Scale.
- Concurrent severe depression defined by a score greater than 16 on the MADRS Exposure to neuroleptic drugs within 6 months prior to baseline assessment Prior intracerebral surgical intervention for Parkinson's disease including Deep Brain stimulation, lesional surgery, growth factor administration, gene therapy or cell transplant
- Already actively participating in a trial of a device, drug or surgical treatment for Parkinson's disease, or trial participation within previous 30 days.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ExenatideAlso: Byetta, Exendin-4Exenatide, 5 micrograms twice a day for 1 month and 10 micrograms twice a day for 11 months. Total duration of treatment 12 months | Drug | Exenatide |
Primary outcome
Change from baseline to 12 months & 14 months between patients on active Exenatide treatment and PD controls in respect of their UPDRS-off-medication motor subscore.
1 secondary outcome
Adverse event profile among patients treated with Exenatide compared with matched PD controls. Change from baseline to 12 months/ 14 months between patients on active treatment and PD controls in respect of list given below
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2012.
- Derived from this study: PubMed 23728174Aviles-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.
Related trials for Parkinson's Disease
Enrolling by invitationObservational study
Early Molecular Biomarkers for Differentiating Parkinsonian Syndromes
PARKINSON DISEASE (Disorder), Atypical Parkinsonism
Active, not recruitingPhase 1/2
Clinical Safety and Efficacy of Umbilical Cord Mesenchymal Stromal Cells-derived Exosomes in Patients With Parkinson's Disease
Parkinson Disease (PD)
Active, not recruitingObservational study
Adaptive DBS to Offset STN Dysregulation During Exercise in PD
Parkinson Disease
Not yet recruiting
Compulsive Eating and Brain's Reward System in Different Pathologies
PARKINSON DISEASE (Disorder), Bipolar 1 Disorder
Not yet recruiting
Evaluating the Effect of the Zeen Mobility Device on Clinical Practicality, Safety, and Impact (Via Assessments of Walking, Standing Tolerance, Balance, Endurance, Effort, and Participation in Daily Tasks) in an Outpatient Rehabilitation Setting for Individuals With Mobility Impairments.
Stroke, Parkinson's Disease (PD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Exenatide in people with Parkinson's Disease, run by University College, London at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Mar 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline to 12 months & 14 months between patients on active Exenatide treatment and PD controls in respect of their UPDRS-off-medication motor subscore.. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01174810, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2012.