Myotonic Dystrophy Patient Centered Therapy Development Meeting
September 17, 2015
Ritz-Carlton Pentagon City, Arlington, VA
Goal: Explore myotonic dystrophy (DM) heterogeneity and the development of meaningful and measureable DM clinical endpoints, dientifying biomakrers nad patient-reported outcmes to guide and advance the discovery of new therapies and design of clinical trials for the care and cure of patients wtih myotonic dystrophy.
Note: as additional presentations become available, MDF will upload them and link them below.
Agenda
Time
Presentation
8:30 AM
Welcome: Molly White - Chief Executive Officer, MDF
8:35 AM
Introduction of Chair - Woodie Kessel, MD, MPH, MDF Vice Chair
8:40 AM
Meeting Overview - Stephen Spielberg, MD, PhD, Editor-in-Chief of Therapeutic Innovation & Regulatory Science Journal
8:45 AM
FDA Perspective - Richard Moscicki, MD, Deputy Center Director for Science Operations, CDER, FDA
9:00 AM
Patient Perspectives and Disease Overview - Sarah Clarke, MD
Living with DM Short Film
9:15 AM
Disease Symptoms & Pathology Overview - Nicholas Johnson, MD, University of Utah
9:30 AM
Session 1:
- PRISM-1 Study - Richard Moxley, MD, University of Rochester
- Christopher Project Data - Katharine Hagerman, PhD, Stanford University
- MDF Benefit-Risk Survey Results - Sharon Hesterlee, PhD, MDF
- Patient-Centered Research - Pujita Vaidya, MPH, FDA
10:30 AM
Discussion Panel: Session 1
11:00 AM
Break
11:15 AM
Session 2: DM Trial Endpoint Selection
- University of Rochester Natural History Study Data - Charles Thornton, MD, University of Rochester
- University of Sherbrooke Natural History Data, OMMYD Process & SOPs - Cynthia Gagnon, PhD, University of Sherbrooke
- Myotonic Dystrophy Health Index (MDHI) - Chad Heatwole, MD, University of Rochester
- Endpoint Selection & Validation - Nikunj Patel, PhD, FDA
12:15 PM
Discussion Panel: Session 2
12:45 PM
Lunch
1:30 PM
Session 3: FDA Case Studies
- Trial Design for a Slow Progressing, Heterogeneous Disease - Ronald Farkas, PhD, Lead Medical Officer & Clinical Team Leader, Division of Neurology Products, CDER, FDA
2:00 PM
Discussion Panel: Session 3
2:30 PM
Session 4: Candidate DM Biomarkers
- The Big Picture: DM Biomarker Development - John Day, MD, PhD, Stanford University
- Current Thinking: DM Biomarkers for Clinical Trials - John Carulli, PhD, Biogen Idec
- Biomarkers in Drug Development - Shashi Amur, PhD, FDA
3:00 PM
Discussion Panel: Session 4
3:30 PM
Summary and Next Steps Discussion - Steven Spielberg, MD, PhD
4:00 PM
Adjourn