دورية أكاديمية
Towards a multi-arm multi-stage platform trial of disease modifying approaches in Parkinson’s disease
| العنوان: | Towards a multi-arm multi-stage platform trial of disease modifying approaches in Parkinson’s disease |
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| المؤلفون: | Foltynie, Tom, Gandhi, Sonia, Gonzalez-Robles, Cristina, Zeissler, Marie-Louise, Mills, Georgia, Barker, Roger, Carpenter, James, Schrag, Anette, Schapira, Anthony, Bandmann, Oliver, Mullin, Stephen, Duffen, Joy, McFarthing, Kevin, Chataway, Jeremy, Parmar, Mahesh, Carroll, Camille, Shlomo, Yoav Ben, Edwards, Mark, Whone, Alan, Counsell, Carl, Clarke, Caroline, Burnell, Matthew, Salathiel, Dorothy, Whipps, Sue, Jewell, Anna, Barber, Tom, Weil, Rimona, Gray, Caroline Williams, Hu, Michele, Rochester, Lynn, Piccini, Paola, Zetterberg, Henrik, Noyce, Alastair, Chaudhuri, Ray, Lawton, Michael, Jha, Ashwani, Siu, Carroll, Bartlett, Michèle, van Wamelen, Daniel, Stott, Simon, Tofaris, George, Sammler, Esther, Mortiboys, Heather, Wei, Li, Wong, Alan, Duty, Susan, Dexter, David, Scurfield, Paula, Jabbari, Edwin, Morris, Huw |
| المصدر: | Foltynie , T , Gandhi , S , Gonzalez-Robles , C , Zeissler , M-L , Mills , G , Barker , R , Carpenter , J , Schrag , A , Schapira , A , Bandmann , O , Mullin , S , Duffen , J , McFarthing , K , Chataway , J , Parmar , M , Carroll , C , EJS ACT-PD Consortium , Shlomo , Y B , Edwards , M , Whone , A , Counsell , C , Clarke , C , Burnell , M , Salathiel , D , Whipps , .... |
| سنة النشر: | 2023 |
| المجموعة: | Discovery - University of Dundee Online Publications |
| مصطلحات موضوعية: | complex innovative trial design, multi-arm, multi-stage, Parkinson’s disease, platform trial, /dk/atira/pure/subjectarea/asjc/2700/2728, name=Clinical Neurology |
| الوصف: | An increase in the efficiency of clinical trial conduct has been successfully demonstrated in the oncology field, by the use of multi-arm, multi-stage trials allowing the evaluation of multiple therapeutic candidates simultaneously, and seamless recruitment to phase 3 for those candidates passing an interim signal of efficacy. Replicating this complex innovative trial design in diseases such as Parkinson’s disease is appealing, but in addition to the challenges associated with any trial assessing a single potentially disease modifying intervention in Parkinson’s disease, a multiarm platform trial must also specifically consider the heterogeneous nature of the disease, alongside the desire to potentially test multiple treatments with different mechanisms of action. In a multi-arm trial, there is a need to appropriately stratify treatment arms to ensure each are comparable with a shared placebo/standard of care arm; however, in Parkinson’s disease there may be a preference to enrich an arm with a subgroup of patients that may be most likely to respond to a specific treatment approach. The solution to this conundrum lies in having clearly defined criteria for inclusion in each treatment arm as well as an analysis plan that takes account of predefined subgroups of interest, alongside evaluating the impact of each treatment on the broader population of Parkinson’s disease patients. Beyond this, there must be robust processes of treatment selection, and consensus derived measures to confirm target engagement and interim assessments of efficacy, as well as consideration of the infrastructure needed to support recruitment, and the long-term funding and sustainability of the platform. This has to incorporate the diverse priorities of clinicians, triallists, regulatory authorities and above all the views of people with Parkinson’s disease. |
| نوع الوثيقة: | article in journal/newspaper |
| وصف الملف: | application/pdf |
| اللغة: | English |
| العلاقة: | https://discovery.dundee.ac.uk/en/publications/722e8d84-bd00-4419-9880-4165680000afTest |
| DOI: | 10.1093/brain/awad063 |
| الإتاحة: | https://doi.org/10.1093/brain/awad063Test https://discovery.dundee.ac.uk/en/publications/722e8d84-bd00-4419-9880-4165680000afTest https://discovery.dundee.ac.uk/ws/files/122160060/awad063.pdfTest http://www.scopus.com/inward/record.url?scp=85164210946&partnerID=8YFLogxKTest |
| حقوق: | info:eu-repo/semantics/openAccess |
| رقم الانضمام: | edsbas.C2BF1268 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1093/brain/awad063 |
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