Case Study on Vascular Ehlers-Danlos Syndrome Trial

This document is a case study detailing the Phase 3 clinical trial for Vascular Ehlers-Danlos Syndrome (vEDS), a rare vascular disorder. The study faced significant challenges in patient enrollment due to the rarity of the condition and the geographical dispersion of potential participants. Traditional site visits posed a burden on patients, which could have negatively impacted recruitment and retention over the 40-month trial period. In response to these challenges, the sponsor implemented a remote trial design based on FDA guidance, partnering with Science 37 to facilitate nationwide in-home visits. The solution involved shipping study medications to participants and administering them through research-grade nurses. The trial utilized daily electronic diaries for real-time data collection and engaged local imaging facilities for protocol compliance. Targeted outreach through patient advocacy networks and virtual engagement strategies were employed to ensure safety monitoring and maintain participant retention. Ultimately, the trial successfully enrolled 40 participants across 19 states.