Friday, May 30, 2025 10:00am-4:00pm ET
Master Protocols for Rare Diseases: A Framework for Batten Disease
Conference Co-chairs: Jennifer Vermilion, Erika Fullwood Augustine
Conference Speakers: Melanie Quintana, Sabrina Paganoni, Scott Plotkin, Sara Calvert, Ineka Whiteman, Raymond Wang
Master protocols: Platform Trials, Basket Trials, Umbrella Trials.
Dive into sessions on Master Protocols—featuring biostatistics insights and real-world platform trial case studies—and explore the Batten Disease pipeline with leaders from the Batten Disease Support and Research Association. After the sessions, join a breakout group to brainstorm next steps, followed by a full group summary.
Topics throughout the day included:
- Master Protocol Design and Case Examples
- Designing a Master Protocol Trial in Batten Disease
- Breakout Sessions: Umbrella Trial Development and Basket Trial Development