Accelerating the Clinical Trial Process in Duchenne

Lengthy trials with variable results, based on endpoints such as the 6-minute walk test that can limit trial enrollment to ambulant individuals with Duchenne – this is today’s reality for clinical trials in this disease.


But progress is being made and PPMD is proud of our role in changing the clinical trial process. Last week, Critical Path Institute (C-Path) and The Clinical Data Interchange Standards Consortium (CDISC) announced the open availability of a Duchenne Muscular Dystrophy Therapeutic Area User Guide (TAUG-DMD v1.0), which describes the most common clinical concepts relevant to Duchenne studies using the CDISC standard format. These CDISC standards represent a common structure that all data can be translated into and then aggregated into one large dataset to be used for analysis. This format allows datasets from different sources to be compared or combined for data collection, sharing, and analysis.


As you will recall, The Duchenne Regulatory Science Consortium (D-RSC), which PPMD helps lead, is developing a model of how Duchenne progresses over time. D-RSC aims to create a “map” of sorts, so that groups of people with Duchenne – whether ambulatory or non-ambulatory – could be plotted out and disease progression and time to loss of milestones can be predicted. To do this, large amounts of data from different datasets, including placebo arms of clinical trials, patient registries like DuchenneConnect, and natural history studies need to be aggregated. Then decisions have to be made as to which functional measures will be used.


On October 17, at the American Conference of Pharmacometrics, a poster was presented that outlined the model that will be developed to create the “map” of the disease. Next steps are to develop the statistical plan so that a “regulatory-ready” quantitative drug development platform can be developed, which will be acceptable to the regulatory agencies.


We are pleased that the D-RSC effort is making progress that will one day make clinical trials shorter and better for clinical trial participants.

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