ArticleCPT: pharmacometrics & systems pharmacology2022
Development of a model-based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy.
Article in CPT: pharmacometrics & systems pharmacology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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The trial behind it
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Who cites it
23 citing papers in PubMed.
- Empowering Clinical Development With Disease Progression Modeling: Recommendations From the Clinical Trials Transformation Initiative.Clinical and translational science · 2026Article
- Paediatric medicines development for Duchenne muscular dystrophy: An EU regulatory perspective.Journal of neuromuscular diseases · 2026Review
- A model-informed clinical trial simulation tool with a graphical user interface for Duchenne muscular dystrophy.CPT: pharmacometrics & systems pharmacology · 2025Article
- A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studies.CPT: pharmacometrics & systems pharmacology · 2025Article
- State-of-the-Art on Model-Informed Drug Development Approaches for Pediatric Rare Diseases.CPT: pharmacometrics & systems pharmacology · 2025Review
- Considering Opportunities and Challenges When Implementing the Model Master File Framework - a Meeting Report.Pharmaceutical research · 2025Article
- The Potential of Disease Progression Modeling to Advance Clinical Development and Decision Making.Clinical pharmacology and therapeutics · 2025Review
- Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy.PloS one · 2025Article
- Five multivariate Duchenne muscular dystrophy progression models bridging six-minute walk distance and MRI relaxometry of leg muscles.Journal of pharmacokinetics and pharmacodynamics · 2024Article
- Bioavailability as Proof to Authorize the Clinical Testing of Neurodegenerative Drugs-Protocols and Advice for the FDA to Meet the ALS Act Vision.International journal of molecular sciences · 2024Review
- Population longitudinal analysis of Gait Profile Score and North Star Ambulatory Assessment in children with Duchenne muscular dystrophy.CPT: pharmacometrics & systems pharmacology · 2024Article
- Advancing the utilization of real-world data and real-world evidence in clinical pharmacology and translational research-Proceedings from the ASCPT 2023 preconference workshop.Clinical and translational science · 2024Review
- Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials.PloS one · 2024Article
- Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease.Journal of neuromuscular diseases · 2024Article
- Multivariate modeling of magnetic resonance biomarkers and clinical outcome measures for Duchenne muscular dystrophy clinical trials.CPT: pharmacometrics & systems pharmacology · 2023Article
- Longitudinal estimated glomerular filtration rate (eGFR) modeling in long-term renal function to inform clinical trial design in kidney transplantation.Clinical and translational science · 2023Article
- Machine Learning in Clinical Trials: A Primer with Applications to Neurology.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2023Review
- Disease progression modeling of the North Star Ambulatory Assessment for Duchenne Muscular Dystrophy.CPT: pharmacometrics & systems pharmacology · 2023Article
- Modeling Early Heterogeneous Rates of Progression in Boys with Duchenne Muscular Dystrophy.Journal of neuromuscular diseases · 2023Article
- Platelet thromboxane inhibition by low-dose aspirin in polycythemia vera: Ex vivo and in vivo measurements and in silico simulation.Clinical and translational science · 2022Article
Corrections and comments
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Authors and funding
12 authors.
Funding
Abstract
Early clinical trials of therapies to treat Duchenne muscular dystrophy (DMD), a fatal genetic X-linked pediatric disease, have been designed based on the limited understanding of natural disease progression and variability in clinical measures over different stages of the continuum of the disease. The objective was to inform the design of DMD clinical trials by developing a disease progression model-based clinical trial simulation (CTS) platform based on measures commonly used in DMD trials. Data were integrated from past studies through the Duchenne Regulatory Science Consortium founded by the Critical Path Institute (15 clinical trials and studies, 1505 subjects, 27,252 observations). Using a nonlinear mixed-effects modeling approach, longitudinal dynamics of five measures were modeled (NorthStar Ambulatory Assessment, forced vital capacity, and the velocities of the following three timed functional tests: time to stand from supine, time to climb 4 stairs, and 10 meter walk-run time). The models were validated on external data sets and captured longitudinal changes in the five measures well, including both early disease when function improves as a result of growth and development and the decline in function in later stages. The models can be used in the CTS platform to perform trial simulations to optimize the selection of inclusion/exclusion criteria, selection of measures, and other trial parameters. The data sets and models have been reviewed by the US Food and Drug Administration and the European Medicines Agency; have been accepted into the Fit-for-Purpose and Qualification for Novel Methodologies pathways, respectively; and will be submitted for potential endorsement by both agencies.
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