Decentralized Clinical Trial (Dct) Design with the Potential to Improve Patient Recruitment and Retention in Glisten: A Phase 3 Study of Linerixibat for Cholestatic Pruritus in Patients with Primary Biliary Cholangitis (Pbc)

ABSTRACT

Background: Evolution toward remote lifestyles, including healthcare access, has accelerated due to the COVID-19 pandemic. Challenges related to participant recruitment and retention mean clinical trial design is also evolving toward a remote model where trials use digital health technologies and services to improve patient experience. A Decentralized Clinical Trial (DCT) is an emerging model where assessment of patients can occur outside of a traditional site, including at a patient's own home. Hybrid trials which incorporate some DCT components increase convenience and enhance accessibility to patients. Several professional organizations including the Clinical Trials Transformation Initiative and Decentralized Trials and Research Alliance advocate DCT design in clinical studies. Draft guidance on the use of digital health technology for remote data acquisition from participants in clinical trials has been recently issued by the FDA. They recognize that to improve participant recruitment and retention, and increase patient diversity, remote data collection can allow more frequent or even continuous data collection that may better represent real-world experience with the study drug. DCTs are particularly relevant for rare diseases such as PBC where patients may not be in close proximity to a specialist. In fact, 70% of patients with PBC surveyed indicated they would be more likely to participate in a clinical trial if remote options were available. Method(s) The Phase 3 GLISTEN trial (NCT04950127) is investigating the efficacy and safety of linerixibat for the treatment of cholestatic pruritus in patients with PBC. Several operating models are in place, including a fully decentralized option (Figure). DCT elements included in GLISTEN include digital recruitment of patients via social media/patient groups, remote completion of informed consent, telemedicine, electronic clinical and patient-reported outcome (PRO) assessments, home health visits, and direct shipment of study drug/placebo to the patient's home. The primary endpoint in GLISTEN is a PRO, making it compatible with a DCT approach. Result(s) Patient recruitment is ongoing;the study is active at physical study sites worldwide and as a fully remote DCT in the USA. Conclusion(s) Incorporation of DCT design elements into GLISTEN is expected to address the issues of limited numbers and diversity of patients recruited, which is particularly important for a rare disease such as PBC.