Investigating trial design variability in trials of disease-modifying therapies in Parkinson’s disease: a scoping review protocol
Introduction Parkinson’s disease (PD) is a debilitating neurological disorder for which the identification of disease-modifying interventions represents a major unmet need. Diverse trial designs have attempted to mitigate challenges of population heterogeneity, efficacious symptomatic therapy and la...
| Main Authors: | , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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BMJ Publishing Group
2023-12-01
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| Series: | BMJ Open |
| Online Access: | https://bmjopen.bmj.com/content/13/12/e071641.full |
| _version_ | 1811153624401707008 |
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| author | Camille B Carroll Thea Dominey Hari Venkatesh Pai Emma King Marie-Louise Zeissler Timothy Boey Danny Chapman Gary Rafaloff Karen G Raphael Susan Buff Paul Sharpe Fintan O’Brien |
| author_facet | Camille B Carroll Thea Dominey Hari Venkatesh Pai Emma King Marie-Louise Zeissler Timothy Boey Danny Chapman Gary Rafaloff Karen G Raphael Susan Buff Paul Sharpe Fintan O’Brien |
| author_sort | Camille B Carroll |
| collection | DOAJ |
| description | Introduction Parkinson’s disease (PD) is a debilitating neurological disorder for which the identification of disease-modifying interventions represents a major unmet need. Diverse trial designs have attempted to mitigate challenges of population heterogeneity, efficacious symptomatic therapy and lack of outcome measures that are objective and sensitive to change in a disease modification setting. It is not clear whether consensus is emerging regarding trial design choices. Here, we report the protocol of a scoping review that will provide a contemporary update on trial design variability for disease-modifying interventions in PD.Methods and analysis The Population, Intervention, Comparator, Outcome and Study design (PICOS) framework will be used to structure the review, inform study selection and analysis. The databases MEDLINE, Web of Science, Cochrane and the trial registry ClinicalTrials.gov will be systematically searched to identify published studies and registry entries in English. Two independent reviewers will screen study titles, abstracts and full text for eligibility, with disagreements being resolved through discussion or by a third reviewer where necessary. Data on general study information, eligibility criteria, outcome measures, trial design, retention and statistically significant findings will be extracted into a standardised form. Extracted data will be presented in a descriptive analysis. We will report our findings using the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Scoping Review extension.Ethics and dissemination This work will provide an overview of variation and emerging trends in trial design choices for disease-modifying trials of PD. Due to the nature of this study, there are no ethical or safety considerations. We plan to publish our findings in a peer-reviewed journal. |
| first_indexed | 2024-03-08T00:03:18Z |
| format | Article |
| id | doaj.art-aeea7b7597704455848d3e49d0e11eee |
| institution | Directory Open Access Journal |
| issn | 2044-6055 |
| language | English |
| last_indexed | 2024-03-08T00:03:18Z |
| publishDate | 2023-12-01 |
| publisher | BMJ Publishing Group |
| record_format | Article |
| series | BMJ Open |
| spelling | doaj.art-aeea7b7597704455848d3e49d0e11eee2024-02-17T16:10:08ZengBMJ Publishing GroupBMJ Open2044-60552023-12-01131210.1136/bmjopen-2023-071641Investigating trial design variability in trials of disease-modifying therapies in Parkinson’s disease: a scoping review protocolCamille B Carroll0Thea Dominey1Hari Venkatesh Pai2Emma King3Marie-Louise Zeissler4Timothy Boey5Danny Chapman6Gary Rafaloff7Karen G Raphael8Susan Buff9Paul Sharpe10Fintan O’Brien11Faculty of Health, University of Plymouth, Plymouth, UKFaculty of Health, University of Plymouth, Plymouth, UKQueen`s Medical Centre, Nottingham, UKUniversity Hospitals Plymouth NHS Trust, Plymouth, UKApplied Parkinson`s Research Group, Faculty of Health, University of Plymouth, Plymouth, UKSchool of Medicine, University of Liverpool, Liverpool, Merseyside, UKFaculty of Health, University of Plymouth, Plymouth, UKParkinson`s Research Advocate, Westlake, Florida, USAOral & Maxillofacial, Radiology and Medicine, New York University, Brooklyn, New York, USAParkinson`s Research Advocate, Sunnyvale, California, USAFaculty of Health, University of Plymouth, Plymouth, UKFaculty of Health, University of Plymouth, Plymouth, UKIntroduction Parkinson’s disease (PD) is a debilitating neurological disorder for which the identification of disease-modifying interventions represents a major unmet need. Diverse trial designs have attempted to mitigate challenges of population heterogeneity, efficacious symptomatic therapy and lack of outcome measures that are objective and sensitive to change in a disease modification setting. It is not clear whether consensus is emerging regarding trial design choices. Here, we report the protocol of a scoping review that will provide a contemporary update on trial design variability for disease-modifying interventions in PD.Methods and analysis The Population, Intervention, Comparator, Outcome and Study design (PICOS) framework will be used to structure the review, inform study selection and analysis. The databases MEDLINE, Web of Science, Cochrane and the trial registry ClinicalTrials.gov will be systematically searched to identify published studies and registry entries in English. Two independent reviewers will screen study titles, abstracts and full text for eligibility, with disagreements being resolved through discussion or by a third reviewer where necessary. Data on general study information, eligibility criteria, outcome measures, trial design, retention and statistically significant findings will be extracted into a standardised form. Extracted data will be presented in a descriptive analysis. We will report our findings using the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Scoping Review extension.Ethics and dissemination This work will provide an overview of variation and emerging trends in trial design choices for disease-modifying trials of PD. Due to the nature of this study, there are no ethical or safety considerations. We plan to publish our findings in a peer-reviewed journal.https://bmjopen.bmj.com/content/13/12/e071641.full |
| spellingShingle | Camille B Carroll Thea Dominey Hari Venkatesh Pai Emma King Marie-Louise Zeissler Timothy Boey Danny Chapman Gary Rafaloff Karen G Raphael Susan Buff Paul Sharpe Fintan O’Brien Investigating trial design variability in trials of disease-modifying therapies in Parkinson’s disease: a scoping review protocol BMJ Open |
| title | Investigating trial design variability in trials of disease-modifying therapies in Parkinson’s disease: a scoping review protocol |
| title_full | Investigating trial design variability in trials of disease-modifying therapies in Parkinson’s disease: a scoping review protocol |
| title_fullStr | Investigating trial design variability in trials of disease-modifying therapies in Parkinson’s disease: a scoping review protocol |
| title_full_unstemmed | Investigating trial design variability in trials of disease-modifying therapies in Parkinson’s disease: a scoping review protocol |
| title_short | Investigating trial design variability in trials of disease-modifying therapies in Parkinson’s disease: a scoping review protocol |
| title_sort | investigating trial design variability in trials of disease modifying therapies in parkinson s disease a scoping review protocol |
| url | https://bmjopen.bmj.com/content/13/12/e071641.full |
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