Delivering clinical trials at home: protocol, design and implementation of a direct-to-family paediatric lupus trial

Introduction Direct-to-family clinical trials efficiently provide data while reducing the participation burden for children and their families. Although these trials can offer significant advantages over traditional clinical trials, the process of designing and implementing direct-to-family studies is poorly defined, especially in children with rheumatic disease. This paper provides lessons learnt from the design and implementation of a self-controlled, direct-to-family pilot trial aimed to evaluate the effects of a medication management device on adherence to hydroxychloroquine in paediatric SLE.Methods Several design features accommodate a direct-to-family approach. Participants meeting eligibility criteria from across the USA were identified a priori through a disease registry, and all outcome data are collected remotely. The primary outcome (medication adherence) is evaluated using electronic medication event-monitoring, plasma drug levels, patient questionnaires and pill counts. Secondary and exploratory endpoints include (1) lupus disease activity measured by a remote SLE Disease Activity Index examination and the Systemic Lupus Activity Questionnaire; and (2) hydroxychloroquine pharmacokinetics and pharmacodynamics. Recruitment of the initial target of 20 participants was achieved within 10 days. Due to initial recruitment success, enrolment was increased to 26 participants. Additional participants who were interested were placed on a waiting list in case of dropouts during the study.Discussion and dissemination Direct-to-family trials offer several advantages but present unique challenges. Lessons learnt from the protocol development, design, and implementation of this trial will inform future direct-to-family trials for children and adults with rheumatic diseases. Additionally, the data collected remotely in this trial will provide critical information regarding the accuracy of teleresearch in lupus, the impact of adherence to hydroxychloroquine on disease activity and a pharmacokinetic analysis to inform paediatric-specific dosing of hydroxychloroquine.Trial registration number ClinicalTrials.gov Registry (NCT04358302)

Properties and performance of the prototype instrument for the Pierre Auger Observatory

Copyright © 2003 Elsevier B.V. All rights reserved.Construction of the first stage of the Pierre Auger Observatory has begun. The aim of the Observatory is to collect unprecedented information about cosmic rays above 1018 eV. The first phase of the project, the construction and operation of a prototype system, known as the engineering array, has now been completed. It has allowed all of the sub-systems that will be used in the full instrument to be tested under field conditions. In this paper, the properties and performance of these sub-systems are described and their success illustrated with descriptions of some of the events recorded thus far.Auger Collaboration, ..., J. A. Bellido, ..., R. W. Clay, ..., B. R. Dawson, ..., G. J. Thornton, ..., N. R. Wild, et al.http://www.elsevier.com/wps/find/journaldescription.cws_home/505701/description#descriptio

AUGER FD: Detector response to simulated showers and real event topologies

The performance of the Auger Fluorescence telescope is discussed on the basis of a mass production chain. In order to get a realistic estimate of the detector resolution, a large number of fully simulated CORSIKA showers have been used for this study. The propagation through the atmosphere and the detector response are taken into account and simulated in detail. Results for the the case of monocular reconstruction are presented here. No quality cuts for the event reconstruction have been applied so far. Finally, a schematic overview of the expected event topologies is given together with the display of a real event recently collected