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Delivering clinical trials at home: protocol, design and implementation of a direct-to-family paediatric lupus trial
Rachel L Randell1, Lindsay Singler2, Anthony Cunningham2
1Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA.
Insights
Direct-to-family trials for pediatric rheumatic diseases are effective. This pilot study on hydroxychloroquine adherence in pediatric SLE demonstrated successful remote data collection and rapid recruitment, offering valuable insights for future research.
Area of Science:
- Pediatric Rheumatology
- Clinical Trial Design
- Pharmacology
Background:
- Direct-to-family clinical trials offer advantages for pediatric research but require defined protocols, especially for rheumatic diseases.
- This study addresses the need for structured direct-to-family trial methodologies in pediatric rheumatic conditions.
Purpose of the Study:
- To evaluate the effectiveness of a medication management device in improving hydroxychloroquine adherence in pediatric Systemic Lupus Erythematosus (SLE).
- To gather lessons learned from designing and implementing a direct-to-family pilot trial for children with rheumatic diseases.
Main Methods:
- A self-controlled, direct-to-family pilot trial was conducted using remote data collection across the USA.
- Participants were identified via a disease registry, with adherence measured by electronic monitoring, drug levels, questionnaires, and pill counts.
- Secondary outcomes included lupus disease activity (remote SLE Disease Activity Index, Systemic Lupus Activity Questionnaire) and hydroxychloroquine pharmacokinetics/pharmacodynamics.
Main Results:
- Rapid recruitment was achieved, enrolling 26 participants within 10 days, exceeding the initial target of 20.
- The study successfully collected remote outcome data, including medication adherence and disease activity measures.
- The trial design facilitated efficient data acquisition for evaluating adherence and its impact on pediatric SLE.
Conclusions:
- Direct-to-family trials are feasible and efficient for pediatric rheumatic diseases, offering advantages in data collection and participant burden.
- Lessons learned from this pilot trial will inform future direct-to-family studies in pediatric rheumatology.
- Remote data collection in this trial provides critical insights into teleresearch accuracy, hydroxychloroquine adherence, and pediatric dosing for SLE.
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).
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