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

Lookup NU author(s): Dr Marie-Louise Zeissler, Professor Camille CarrollORCiD



This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).


© Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.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 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.

Publication metadata

Author(s): Zeissler M-L, Boey T, Chapman D, Rafaloff G, Dominey T, Raphael KG, Buff S, Pai HV, King E, Sharpe P, O'Brien F, Carroll CB

Publication type: Article

Publication status: Published

Journal: BMJ Open

Year: 2023

Volume: 13

Issue: 12

Print publication date: 01/12/2023

Online publication date: 09/12/2023

Acceptance date: 05/11/2023

Date deposited: 30/01/2024

ISSN (electronic): 2044-6055

Publisher: BMJ Publishing Group


DOI: 10.1136/bmjopen-2023-071641

PubMed id: 38070893


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Funder referenceFunder name
Cure Parkinson’s