8 research outputs found

    Suitability of external controls for drug evaluation in Duchenne muscular dystrophy

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    OBJECTIVE: To evaluate the suitability of real-world data (RWD) and natural history data (NHD) for use as external controls in drug evaluations for ambulatory Duchenne muscular dystrophy (DMD). METHODS: The consistency of changes in the 6-minute walk distance (Δ6MWD) was assessed across multiple clinical trial placebo arms and sources of NHD/RWD. Six placebo arms reporting 48-week Δ6MWD were identified via literature review and represented 4 sets of inclusion/exclusion criteria (n = 383 patients in total). Five sources of RWD/NHD were contributed by Universitaire Ziekenhuizen Leuven, DMD Italian Group, The Cooperative International Neuromuscular Research Group, ImagingDMD, and the PRO-DMD-01 study (n = 430 patients, in total). Mean Δ6MWD was compared between each placebo arm and RWD/NHD source after subjecting the latter to the inclusion/exclusion criteria of the trial for baseline age, ambulatory function, and steroid use. Baseline covariate adjustment was investigated in a subset of patients with available data. RESULTS: Analyses included ∼1,200 patient-years of follow-up. Differences in mean Δ6MWD between trial placebo arms and RWD/NHD cohorts ranged from -19.4 m (i.e., better outcomes in RWD/NHD) to 19.5 m (i.e., worse outcomes in RWD/NHD) and were not statistically significant before or after covariate adjustment. CONCLUSIONS: We found that Δ6MWD was consistent between placebo arms and RWD/NHD subjected to equivalent inclusion/exclusion criteria. No evidence for systematic bias was detected. These findings are encouraging for the use of RWD/NHD to augment, or possibly replace, placebo controls in DMD trials. Multi-institution collaboration through the Collaborative Trajectory Analysis Project rendered this study feasible

    Energy-Aware Fisheye Routing (EA-FSR) algorithm for wireless mobile sensor networks

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    Energy consumption is prominent and critical issue faced by wireless sensor networks. The maximum amount of energy is consumed when the sensors communicate with each other. Therefore energy efficient routing mechanisms are required. In this paper, a routing scheme based on the fisheye state routing with a difference in route selection mechanism has been proposed to ensure the reduction in the overall energy consumption of the network. This scheme is named as Energy-Aware Fisheye State Routing (EA-FSR). It is simulated considering various parameters using QualNet5.0. Performance of EA-FSR has been compared with the original fisheye state routing algorithm which is also simulated in the same environment. For comparison various parameters like end-to-end delay average, energy consumption and throughput have been considered
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