DCRI Launches First Direct-to-Family Study to Improve Medication Dosing and Adherence in Children and Teens with Lupus

The clinical study's home-based design combines on-site nursing visits and mobile technology to embed research into patients' lives.

The DCRI today launched the first direct-to-family, decentralized clinical trial to help determine the most effective dose of hydroxychloroquine, commonly known as Plaquenil®, for children and teens with lupus. The iPERSONAL (Individual Patient Exposure and Response in Pediatric Lupus) study also will test whether a digital pill bottle will help children and teens remember to take their medicine.

Stephen Balevic, MD

“Not only does this study use an exciting new model of clinical research, but it is also answering important questions for pediatric patients with lupus,” said Stephen J. Balevic, MD, MHS, Assistant Professor of Pediatric Rheumatology at Duke University and co-principal investigator for the study alongside Christoph Hornik, MD, PhD, MPH, Associate Professor of Pediatrics at Duke University. “Although hydroxychloroquine is the most commonly prescribed drug to patients with lupus, it has never been studied in children to understand the optimal dose needed to reduce lupus disease activity. By conducting this study using a digital pill bottle cap, we’re also studying new ways to improve medication adherence, which is often a challenge when patients experience onset of lupus during childhood and adolescence.”

The DCRI is working with Science 37, the industry leader in decentralized clinical trials, to provide the clinical trial platform and mobile research team needed to conduct the study directly in participants’ homes. Virtual studies, like iPERSONAL, have become an increasingly important way to conduct research during the COVID-19 pandemic, and all precautions will be taken to safely conduct the study.

“The Science 37-DCRI collaboration highlights how meaningful the integration of technology and decentralized clinical trial operational expertise can be in designing and executing clinical research focused on the patient experience,” said Jonathan Cotliar, MD, Chief Medical Officer at Science 37. “The iPERSONAL study will demonstrate that patient safety and high-quality data do not need to be compromised while we make it easier for children and their families to participate in groundbreaking research from home.”

The iPERSONAL study is funded by a grant from the U.S. Food and Drug Administration, led by investigators from the Pediatric Trials Network, and represents a collaboration between the DCRI, the Childhood Arthritis and Rheumatology Research Alliance (CARRA), and the Lupus Foundation of America. The study is guided by a Stakeholder & Patient Advisory Group comprised of representatives from these organizations, children and teens with lupus, and their caregivers who co-led study design and the development of study materials, including the informed consent and assent.

The iPERSONAL study will recruit 20 children and teens with lupus from the CARRA Registry to participate. The CARRA Registry is the largest established observational registry that collects clinical and patient-reported information about children and adolescents with pediatric-onset rheumatic diseases. Recruiting directly from the CARRA Registry will optimize iPERSONAL study enrollment by identifying eligible potential participants with lupus ahead of time. iPERSONAL study participation is voluntary and will include:

  • Four in-home visits by a mobile research nurse over a six-month period, paired with a TeleResearch exam by a remote physician;
  • Biological sample collection to determine lupus disease activity and optimize hydroxychloroquine dosing;
  • Use of a digital pill bottle, which will provide audio and visual reminders to take medication;
  • Use of mobile technologies, including a FitBit to monitor physical activity and a smartphone to enable research survey completion; and
  • Sharing of study results with participants and the lupus community at large.
Micky Cohen-Wolkowiez

“The DCRI has a long history of advancing innovative approaches to study design, and our sponsors rely on us to lead the way in an area where optimization is most needed—pediatric research,” said Michael Cohen-Wolkowiez, MD, PhD, Duke Professor of Pediatrics and the Head of Pediatric Clinical Research at the DCRI. “At the DCRI, we describe these fully decentralized, virtual trials as direct-to-family trials because we are bringing research directly into families’ homes. We are learning key aspects of how to ethically and appropriately conduct these novel trials in children and teens, which have important differences relative to direct-to-participant trials conducted in adults.”

For more information about the iPERSONAL study, visit the study website.

Related Content

Lay Summary: View a PDF of the study description, design, and start-up lessons learned for the iPERSONAL Direct-to-Family Pediatric Lupus Trial.