Investigating trial design variability in trials of disease-modifying therapies in Parkinson’s disease: a scoping review protocol

• Published in: BMJ open 2023-12, Vol.13 (12), p.e071641-e071641
• Main Authors: Zeissler, Marie-Louise, Boey, Timothy, Chapman, Danny, Rafaloff, Gary, Dominey, Thea, Raphael, Karen G, Buff, Susan, Pai, Hari Venkatesh, King, Emma, Sharpe, Paul, O’Brien, Fintan, Carroll, Camille B
• Format: Article
• Language: English
• Subjects: Anti-inflammatory agents; Clinical trials; Decision trees; Immunosuppressive agents; Immunotherapy; Neurology; Parkinson's disease; Research methodology; systematic review
• ISSN: 2044-6055; 2044-6055
• DOI: 10.1136/bmjopen-2023-071641

IntroductionParkinson’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 analysisThe 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 disseminationThis 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.