Simvastatin as a neuroprotective treatment for Parkinson’s disease (PD STAT): protocol for a double-blind, randomised, placebo-controlled futility study
Introduction Parkinson’s disease (PD) is a progressive neurodegenerative condition affecting approximately 185,000 people in the UK. No drug has been proven to slow disease progression. Epidemiological and pre-clinical data support simvastatin, a widely used cholesterol-lowering drug with a well-est...
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BMJ Publishing Group
2019-10-01
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| Series: | BMJ Open |
| Online Access: | https://bmjopen.bmj.com/content/9/10/e029740.full |
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| author | Susan Ball Douglas Webb Camille B Carroll Kara Nicola Stevens Jane Vickery Vicky Eyre Richard Wyse Mike Webber Andy Foggo John Zajicek Alan Whone Siobhan Creanor |
| author_facet | Susan Ball Douglas Webb Camille B Carroll Kara Nicola Stevens Jane Vickery Vicky Eyre Richard Wyse Mike Webber Andy Foggo John Zajicek Alan Whone Siobhan Creanor |
| author_sort | Susan Ball |
| collection | DOAJ |
| description | Introduction Parkinson’s disease (PD) is a progressive neurodegenerative condition affecting approximately 185,000 people in the UK. No drug has been proven to slow disease progression. Epidemiological and pre-clinical data support simvastatin, a widely used cholesterol-lowering drug with a well-established safety profile, having neuroprotective properties. The aim of this study (Simvastatin as a neuroprotective treatment for PD (PD STAT)) is to determine whether simvastatin has the potential to slow PD progression. The study is part of the International Linked Clinical Trials initiative coordinated by The Cure Parkinson’s Trust. This paper describes the protocol for the PD STAT study.Methods and analysis PD STAT is a double-blind, randomised, placebo-controlled, multi-centre, parallel group, futility trial in patients with PD of mild–moderate severity. 235 participants have been recruited and randomly allocated in a 1:1 ratio to receive either oral simvastatin or matched placebo. Treatment involves a 1-month low-dose phase (40 mg daily), followed by a 23-month high-dose phase (80 mg daily) and ends with a 2-month washout period. Participants are reviewed at clinic visits at 1 month, 6, 12, 18, 24 and 26 months post-baseline, with interim telephone follow-up to monitor for adverse events.The primary outcome is the change in the Movement Disorder Society Unified Parkinson’s Disease Rating Scale part III motor subscale score in the practically defined OFF medication state (OFF state) between baseline and 24 months. Primary analysis will be on a modified intention to treat basis and will include only those participants who progress to the high-dose phase of the study.Ethics and dissemination The protocol has been approved by the North East–Newcastle and North Tyneside 2 Research Ethics Committee. The results will be disseminated via research articles in peer-reviewed journals and presentations at local, national and international scientific meetings, as well as disseminated via patient groups, websites and networks. A summary of the study findings will be posted to participants at the end of the study.Trial Registration ISRCTN16108482 (prospectively registered); EudraCT 2015-000148-40; ClinicalTrials.gov NCT02787590; Pre-results. |
| format | Article |
| id | doaj-art-bff309947e1443688e85e2f85179995e |
| institution | Kabale University |
| issn | 2044-6055 |
| language | English |
| publishDate | 2019-10-01 |
| publisher | BMJ Publishing Group |
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| series | BMJ Open |
| spelling | doaj-art-bff309947e1443688e85e2f85179995e2024-12-14T07:40:09ZengBMJ Publishing GroupBMJ Open2044-60552019-10-0191010.1136/bmjopen-2019-029740Simvastatin as a neuroprotective treatment for Parkinson’s disease (PD STAT): protocol for a double-blind, randomised, placebo-controlled futility studySusan Ball0Douglas Webb1Camille B Carroll2Kara Nicola Stevens3Jane Vickery4Vicky Eyre5Richard Wyse6Mike Webber7Andy Foggo8John Zajicek9Alan Whone10Siobhan Creanor111 NIHR Applied Research Collaboration South West Peninsula (PenARC), Department of Health and Community Sciences, University of Exeter Faculty of Health and Life Sciences, Exeter, UK33 UNDP, Tblisi, GeorgiaUniversity Hospitals Plymouth NHS Trust, Plymouth, UKMedical Statistics Group, Faculty of Medicine and Dentistry, University of Plymouth, Plymouth, UK2 Peninsula Clinical Trials Unit, University of Plymouth, Plymouth, UK2 Peninsula Clinical Trials Unit, University of Plymouth, Plymouth, UK5 The Cure Parkinson`s Trust, London, UK6 PPI Representative, University of Plymouth, Plymouth, UK6 PPI Representative, University of Plymouth, Plymouth, UK7 School of Medicine, University of St Andrews, St Andrews, UK8 School of Translational Health Sciences, University of Bristol, Bristol, UKCentre for Health and Environmental Statistics, University of Plymouth, Plymouth, UKIntroduction Parkinson’s disease (PD) is a progressive neurodegenerative condition affecting approximately 185,000 people in the UK. No drug has been proven to slow disease progression. Epidemiological and pre-clinical data support simvastatin, a widely used cholesterol-lowering drug with a well-established safety profile, having neuroprotective properties. The aim of this study (Simvastatin as a neuroprotective treatment for PD (PD STAT)) is to determine whether simvastatin has the potential to slow PD progression. The study is part of the International Linked Clinical Trials initiative coordinated by The Cure Parkinson’s Trust. This paper describes the protocol for the PD STAT study.Methods and analysis PD STAT is a double-blind, randomised, placebo-controlled, multi-centre, parallel group, futility trial in patients with PD of mild–moderate severity. 235 participants have been recruited and randomly allocated in a 1:1 ratio to receive either oral simvastatin or matched placebo. Treatment involves a 1-month low-dose phase (40 mg daily), followed by a 23-month high-dose phase (80 mg daily) and ends with a 2-month washout period. Participants are reviewed at clinic visits at 1 month, 6, 12, 18, 24 and 26 months post-baseline, with interim telephone follow-up to monitor for adverse events.The primary outcome is the change in the Movement Disorder Society Unified Parkinson’s Disease Rating Scale part III motor subscale score in the practically defined OFF medication state (OFF state) between baseline and 24 months. Primary analysis will be on a modified intention to treat basis and will include only those participants who progress to the high-dose phase of the study.Ethics and dissemination The protocol has been approved by the North East–Newcastle and North Tyneside 2 Research Ethics Committee. The results will be disseminated via research articles in peer-reviewed journals and presentations at local, national and international scientific meetings, as well as disseminated via patient groups, websites and networks. A summary of the study findings will be posted to participants at the end of the study.Trial Registration ISRCTN16108482 (prospectively registered); EudraCT 2015-000148-40; ClinicalTrials.gov NCT02787590; Pre-results.https://bmjopen.bmj.com/content/9/10/e029740.full |
| spellingShingle | Susan Ball Douglas Webb Camille B Carroll Kara Nicola Stevens Jane Vickery Vicky Eyre Richard Wyse Mike Webber Andy Foggo John Zajicek Alan Whone Siobhan Creanor Simvastatin as a neuroprotective treatment for Parkinson’s disease (PD STAT): protocol for a double-blind, randomised, placebo-controlled futility study BMJ Open |
| title | Simvastatin as a neuroprotective treatment for Parkinson’s disease (PD STAT): protocol for a double-blind, randomised, placebo-controlled futility study |
| title_full | Simvastatin as a neuroprotective treatment for Parkinson’s disease (PD STAT): protocol for a double-blind, randomised, placebo-controlled futility study |
| title_fullStr | Simvastatin as a neuroprotective treatment for Parkinson’s disease (PD STAT): protocol for a double-blind, randomised, placebo-controlled futility study |
| title_full_unstemmed | Simvastatin as a neuroprotective treatment for Parkinson’s disease (PD STAT): protocol for a double-blind, randomised, placebo-controlled futility study |
| title_short | Simvastatin as a neuroprotective treatment for Parkinson’s disease (PD STAT): protocol for a double-blind, randomised, placebo-controlled futility study |
| title_sort | simvastatin as a neuroprotective treatment for parkinson s disease pd stat protocol for a double blind randomised placebo controlled futility study |
| url | https://bmjopen.bmj.com/content/9/10/e029740.full |
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