Practical guidance for running late-phase platform protocols for clinical trials: lessons from experienced UK clinical trials units

Love, Sharon; Cafferty, Fay; Snowdon, Claire; Carty, Karen; Savage, Joshua; Pallmann, Philip; McParland, Lucy; Brown, Louise; Masters, Lindsey; Schiavone, Francesca; Hague, Dominic; Townsend, Stephen; Amos, Claire; South, Annabelle; Sturgeon, Kate; Langley, Ruth E.; Maughan, T; James, Nicholas D.; Hall, Emma; Kernaghan, Sarah; Bliss, Judith; Turner, Nicholas C.; Varga, Andrea; Yap, Christina; Firth, Charlotte; Kong, Anthony Pak‐Hin; Mehanna, Hisham; Watts, Colin; Hills, Robert Kerrin; Thomas, Ian; Copland, Mhairi; Bell, Sue; Sebag‐Montefiore, David; Jones, Robert J.; Parmar, Mahesh; Sydes, Matthew R.

doi:10.1186/s13063-022-06680-4

Cited by 6 publications

(1 citation statement)

References 27 publications

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“…Another benefit of platform trials is that they provide a re-useable clinical trial infrastructure that enhances operational aspects such as reducing accrual rates or simplifying complex logistics through a centralised governance, all of which is optimised by a tailored legal and regulatory pathway. 8 , 9 …”

Section: Integrated Research Platforms Are Centred On a Platform Tria...mentioning

confidence: 99%

Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL

Koenig,

Spiertz,

Millar

et al. 2024

eClinicalMedicine

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Section: Integrated Research Platforms Are Centred On a Platform Tria...mentioning

confidence: 99%

Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL

Koenig,

Spiertz,

Millar

et al. 2024

eClinicalMedicine

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Advantages and Challenges of Platform Trials for Disease Modifying Therapies in Parkinson's Disease

Fabbri,

Rascol,

Foltynie

et al. 2024

Movement Disorders

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Traditional drug development in Parkinson's disease (PD) faces significant challenges because of its protracted timeline and high costs. In response, innovative master protocols are emerging and designed to address multiple research questions within a single overarching protocol. These trials may offer advantages such as increased efficiency, agility in adding new treatment arms, and potential cost savings. However, they also present organizational, methodological, funding, regulatory, and sponsorship challenges. We review the potential of master protocols, focusing on platform trials, for disease modifying therapies in PD. These trials share a common control group and allow for the termination or addition of treatment arms during a trial with non‐predetermined end. Specific issues exist for a platform trial in the PD field considering the heterogeneity of patients in terms of phenotype, genotype and staging, the confounding effects of symptomatic treatments, and the choice of outcome measures with no consensus on a non‐clinical biomarker to serve as a surrogate and the slowness of PD progression. We illustrate these aspects using the examples of the main PD platform trials currently in development with each one targeting distinct goals, populations, and outcomes. Overall, platform trials hold promise in expediting the evaluation of potential therapies for PD. However, it remains to be proven whether these theoretical benefits will translate into increased production of high‐quality trial data. Success also depends on the willingness of pharmaceutical companies to engage in such trials and whether this approach will ultimately hasten the identification and licensing of effective disease‐modifying drugs. © 2024 International Parkinson and Movement Disorder Society.

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Exercise Made Accessible: the Merits of Community-Based Programs for Persons with Parkinson’s Disease

Langbroek-Amersfoort,

Schootemeijer,

Bouten

et al. 2023

Curr Neurol Neurosci Rep

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Purpose of Review Many studies have identified positive effects of physiotherapy and exercise for persons with Parkinson’s disease (PD). Most work has thus far focused on the therapeutic modality of exercise as used within physiotherapy programs. Stimulated by these positive findings, there is now a strong move to take exercise out of the clinical setting and to deliver the interventions in the community. Although the goals and effects of many such community-based exercise programs overlap with those of physiotherapy, it has also become more clear that both exercise modalities also differ in various ways. Here, we aim to comprehensively review the evidence for community-based exercise in PD. Recent Findings Many different types of community-based exercise for people with PD are emerging and they are increasingly being studied. There is a great heterogeneity considering the types of exercise, study designs, and outcome measures used in research on this subject. While this review is positive regarding the feasibility and potential effects of community-based exercise, it is also evident that the general quality of these studies needs improvement. Summary By focusing on community-based exercise, we hope to generate more knowledge on the effects of a wide range of different exercise modalities that can be beneficial for people with PD. This knowledge may help people with PD to select the type and setting of exercise activity that matches best with their personal abilities and preferences. As such, these insights will contribute to an improved self-management of PD.

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Practical guidance for running late-phase platform protocols for clinical trials: lessons from experienced UK clinical trials units

Cited by 6 publications

References 27 publications

Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL

Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL

Advantages and Challenges of Platform Trials for Disease Modifying Therapies in Parkinson's Disease

Exercise Made Accessible: the Merits of Community-Based Programs for Persons with Parkinson’s Disease

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