Duke Clinical Research Institute (DCRI) recently announced its First Direct-to-Family Study to improve medication dosing and adherence in children and teens with lupus. Capitalizing on the decentralized study trend, the clinical trial’s home-based design involves on-site nursing visits and mobile technology to more deeply integrate patient life and research. They also carefully design patient community and stakeholder buy-in and participation in what could be a game-changing approach. Known as iPERSONAL (Individual Patient Exposure and Response in Pediatric Lupus), the study will test if a digital pill bottle can help teens keep to a medication regimen as well as assess optimal medical dosage. Perhaps most important about the potential for iPERSONAL and the Direct-to-Family model are the details considered and the various elements combined to better ensure a positive and progressive outcome for a vulnerable patient population. DCRI, known as an innovator in the world of clinical research, looks to keep this brand, introducing a model for what could be a game-changing approach to bringing the trial site and patient community to the home for critical pediatric studies.
A New Model—Part of a Movement
Led by co-investigators Stephen J. Balevic, MD, MHS, and Christoph Hornik, MD, PhD, MPH, the iPERSONAL study combines both a refined model and a novel way to pursue medical evidence about how to better work with young patients with lupus. The study is but one of many fitting into the virtual or decentralized clinical trials movement as captured by TrialSite’s Joe Constance. But DCRI steps up and not only considers important details, but boldly puts a stake in the ground for what TrialSite considers has the potential for a novel, holistic model moving forward.
The iPERSONAL study, powered by Science 37, an industry leader in decentralized clinical trials, reflects a trend accelerated during the COVID-19 pandemic. Virtual clinical trials and studies become ever more popular and, in fact, required as a standard way to conduct research.
When the TrialSite Becomes Home
TrialSite has profiled Science37, a visionary and impressive group, but also one that, as TrialSite called out, was forced to grasp the implications of not only competing in the big pharma vendor shark tank, but also evolve a model that could sustain and potentially thrive in the future while trying to please investors—not an easy task.
With their mobile, telehealth-powered platform for clinical trials, the study participant’s home becomes the trial site. Key, as called out by Science37 Chief Medical Officer Jonathan Cotliar, MD, is the focus on patient safety and high-quality data—these cannot be compromised or the notion of a trial site as a participant’s home won’t last too long. But technology isn’t enough. And that’s where some of the Duke smarts come in.
DCRI: Generating New Evidence Takes a Digital & Real Village
Of course, these studies must produce material knowledge that progresses medical evidence. That’s the point, otherwise time and money are wasted. In this case, Duke’s Stephen J. Balevic reports that iPERSONAL not only brings the trial site home but also generates “…[I]mportant questions for pediatric patients with lupus.”
In this case, the Duke Professor suggests iPERSONAL can also generate evidence for the clinic. Why? Well first, finding the optimal dosage of hydroxychloroquine for children is no trivial matter. The stakes are real: this TrialSite News author can attest to the loss of a beloved cousin due to lupus arising out of childhood (ultimately leading to kidney failure). What if dosage adjustments can reduce lupus disease activity? The implications for those that lost loved ones come to mind.
Second, by employing a “digital pill bottle cap,” the investigators potentially produce evidence for ways to improve medication adherence. Again, this is novel territory and could potentially save lives down the line. And importantly, third. What makes all of this possible isn’t just the technology nor the protocols, but also the underpinnings: the partnerships with patient groups and advocates, not to mention regulators and the formation of a stakeholder and patient advisory group, which brings credibility and a community backbone leading to longevity.
Vital: Stakeholder & Patient Advisory Group
DCRI introduces an important underpinning to this novel study. A “Stakeholder & Patient Advisory Group” augments or supports this study to ensure not only lupus community buy-in, but also a way to elicit a strong feedback loop for continuous improvements, not to mention channels for knowledge dissemination and inputs for greater collaboration. The formation and use of this model cannot be underestimated in importance and impact if done correctly.
For iPERSONAL, the group consists of representatives from collaborative organizations and children and teens with lupus and their caregivers: all contribute to study design and development of study materials, including the all-important informed consent.
The iPERSONAL Study
As described in Clinicaltrials.gov, the iPERSONAL study (NCT04358302) was designed to investigate whether an electronic pill bottle cap can support children and teens with systemic lupus better remember to take their medicine. Moreover, it will also gather and store information on the best dose of hydroxychloroquine (Plaquenil®) for children and teens. During the study, participants will take their usual medication as prescribed by the physician. In the study, the participants receive an electronic pill bottle cap (pillsy), a smartphone, and a Fitbit. The study will include 20 children and teens with lupus from the CARRA Registry to participate.
During the six-month duration, a nurse will visit each participant 4 times to monitor for symptoms, draw blood, and take a urine sample. After the study, participants can keep the electronic pill bottle cap but must return the smartphone and Fitbit.
With a simple but powerful mission centered on the development and sharing of knowledge that improves the care of patients worldwide via innovative clinical research, DCRI is known in the clinical trials world as an innovator, even at times a pathbreaker. They are at it again with iPERSONAL. Although a virtual or home-based study is not new, in this case, what is noteworthy are the details of how this study came—including the target patient population. Lives can potentially be saved or at least extended based on the findings derived from this study.
Michael Cohen-Wolkowiez, MD, PhD, commented, “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.” He continued, “At the DCRI, we describe these full 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.”
Funding & Partnerships
The iPERSONAL study is supported by the U.S. Food and Drug Administration (FDA); the study is led by the Pediatric Trials Network investigators and represents a collaboration between the DCRI, the Childhood Arthritis and Rheumatology Research Alliance (CARRA), and the Lupus Foundation of America.
Stephen J. Balevic, MD, MHS Assistant Professor of Pediatrics, Assistant Professor of Medicine, Member of DCRI
Christoph Hornik, MD, PhD, MPH Associate Professor Pediatrics, Chief, Division of Quantitative Sciences, Member of DCRI
Lindsay E. Singler, MPH Associate Director Research Communications & Engagement
Call to Action: For more information, see the study website or contact clinical trial coordinator Amanda Harrington (916) 668-4626 or [email protected]. Can you imagine the implications for Direct-to-Family in other critical pediatric disease areas?