Case Study on Vascular Ehlers-Danlos Syndrome

This case study details the approach taken to enroll patients in a Phase 3 trial for Vascular Ehlers-Danlos Syndrome (vEDS), a rare and high-risk vascular disorder. The sponsor faced significant challenges due to the geographic dispersion of potential participants and the lengthy monitoring required over a 40-month period. Traditional site visits would have imposed a substantial burden on patients, potentially impacting recruitment and retention. In response to FDA guidance, the sponsor implemented a remote trial design through Science 37's Direct-to-Patient Site. This solution allowed for nationwide in-home visits, reducing patient burden and facilitating access to participation. The trial involved shipping study medications to participants, who were monitored by research-grade nurses. Daily electronic diaries were utilized for real-time data collection, and local imaging facilities supported the protocol's requirements. The targeted outreach and ongoing virtual engagement strategies contributed to the enrollment of 40 participants across 19 states, demonstrating the effectiveness of this innovative approach.