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• W4387310712 abstract "Duchenne muscular dystrophy (DMD) is a rare, fatal, X linked, muscle wasting, and progressive disease that predominantly affects boys but has been shown to manifest in some female carriers. Despite recent progress in the drug development pipeline, trial design for DMD remains difficult due to challenges intrinsic to the nature of the DMD population and the limited understanding in rate of change of endpoints in given populations. The Duchenne Regulatory Science Consortium (D-RSC) at Critical Path Institute (C-Path), has developed a model-based clinical trial simulation (CTS) platform based on a series of quantitative models of disease progression that allow researchers to design clinical trials in silico. In November 2022, D-RSC received a Letter of Support from the European Medicines Agency (EMA), and the CTS tool is currently being reviewed by the U.S. Food and Drug Administration (FDA). To allow an open and broad use of the CTS platform, D-RSC generated a web-based user-friendly graphical user interface (GUI). We describe here how to navigate the D-RSC CTS GUI for the implementation of trial design components, such as the length of follow-up, inclusion/exclusion criteria, and sample size, which may be linked to hypothesized drug effect magnitudes in an endpoint-specific fashion. Although this tool is not intended to replace functional evaluation of the assessment of efficacy in trials for DMD, the CTS tool will allow users to optimize population selection and overall trial design with flexible specification of the simulated population. Acknowledgements On behalf of the Duchenne Regulatory Science Consortium (D-RSC) and the CINRG DNHS investigators. With special thanks to PPMD, D-RSC Industry Members, Academic and Clinical Collaborators, Advisors, C-Path Staff, The Cooperative International Neuromuscular Research Group (CINRG) Investigators from the CINRG DMD Natural History Study (DNHS), ImagingDMD Investigators. This presentation is based on research using data from CureDuchenne that has been made available through Vivli, Inc. Vivli has not contributed to or approved and is not in any way responsible for the contents of this publication. ImagingDMD's data was supported by NIH grant R01AR056973. Duchenne muscular dystrophy (DMD) is a rare, fatal, X linked, muscle wasting, and progressive disease that predominantly affects boys but has been shown to manifest in some female carriers. Despite recent progress in the drug development pipeline, trial design for DMD remains difficult due to challenges intrinsic to the nature of the DMD population and the limited understanding in rate of change of endpoints in given populations. The Duchenne Regulatory Science Consortium (D-RSC) at Critical Path Institute (C-Path), has developed a model-based clinical trial simulation (CTS) platform based on a series of quantitative models of disease progression that allow researchers to design clinical trials in silico. In November 2022, D-RSC received a Letter of Support from the European Medicines Agency (EMA), and the CTS tool is currently being reviewed by the U.S. Food and Drug Administration (FDA). To allow an open and broad use of the CTS platform, D-RSC generated a web-based user-friendly graphical user interface (GUI). We describe here how to navigate the D-RSC CTS GUI for the implementation of trial design components, such as the length of follow-up, inclusion/exclusion criteria, and sample size, which may be linked to hypothesized drug effect magnitudes in an endpoint-specific fashion. Although this tool is not intended to replace functional evaluation of the assessment of efficacy in trials for DMD, the CTS tool will allow users to optimize population selection and overall trial design with flexible specification of the simulated population. Acknowledgements On behalf of the Duchenne Regulatory Science Consortium (D-RSC) and the CINRG DNHS investigators. With special thanks to PPMD, D-RSC Industry Members, Academic and Clinical Collaborators, Advisors, C-Path Staff, The Cooperative International Neuromuscular Research Group (CINRG) Investigators from the CINRG DMD Natural History Study (DNHS), ImagingDMD Investigators. This presentation is based on research using data from CureDuchenne that has been made available through Vivli, Inc. Vivli has not contributed to or approved and is not in any way responsible for the contents of this publication. ImagingDMD's data was supported by NIH grant R01AR056973." @default.
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• W4387310712 date "2023-10-01" @default.
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• W4387310712 title "P146 A clinical trial simulation tool to accelerate trial design in DMD: description of the traphical user interface features and applications" @default.
• W4387310712 doi "https://doi.org/10.1016/j.nmd.2023.07.081" @default.
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