Critical Path Institute held a free virtual workshop, Design of Clinical Trials in New-Onset Type 1 Diabetes: Regulatory Considerations for Drug Development, June 15-16, 2021.
The purpose of this scientific workshop was to discuss the existing evidence regarding the role of C-peptide in clinical trials intended to support regulatory decision making, unique regulatory considerations from FDA and EMA and next steps for the T1D drug development community.
The preliminary agenda included:
- Welcome and Introductory Remarks
- Session I: Regulatory Framework for Clinical Investigations in New/Recent Onset T1D
- Session II: Scientific Framework: The rationale for C-peptide preservation and use as a clinical trial endpoint
- Session III: Establishing/Confirming Clinical Benefit
- Session IV: Overall Issues of Study Design
View the available sessions below.
Agenda – Day 1
| Time (EST) | Title | Presenter |
| 10:00 | Welcoming Remarks and Housekeeping | Inish O’Doherty, C-Path |
| 10:05 | FDA Introductory Remarks | Ilan Irony, FDA |
| 10:15 | Patient Perspective Opening Remarks: Unmet Need | Aaron Kowalski, JDRF |
| 10:25 | Session I: Regulatory Framework for Clinical Investigations in New/Recent Onset T1D | |
| 10:25 | FDA perspective | Kristen Pluchino, FDA |
| 10:45 | EMA perspective | Peter Mol, EMA |
| 11:05 | Break: 20 minutes | |
| 11:25 | Session II: Scientific Framework: The rationale for C-peptide preservation and use as a clinical trial endpoint | Session Co-chairs:
Chantal Mathieu, INNODIA + Patricia Beaston, FDA |
| 11:30 | C-Peptide as Primary Endpoint & Natural History | Kevan Herold, Yale University |
| 12:00 | Islet Transplantation: Relationship of C-peptide and clinically meaningful outcomes | Michael Rickels, University of Pennsylvania |
| 12:20 | Differential Rates of C-Peptide Decline | Carla Greenbaum, Benaroya Research Institute |
| 12:40 | C-Peptide as a Primary Endpoint | Stephen Gough, Novo Nordisk |
| 13:00 | Panel Discussion | Moderators: Session II co-chairs
Panelists: Panelists: Session II speakers + Mark Peakman, Sanofi |
| 13:40 | Day 1 Closing Remarks | |
Agenda – Day 2
| Time (EST) | Title | Presenter |
| 10:00 | Day 2 Opening Remarks | |
| 10:10 | Session III: Establishing/Confirming Clinical Benefit |
Session Co-chairs: Lisa Yanoff, FDA + Colin Dayan, Cardiff University |
| 10:15 | Perspective from people living with T1D: Clinically meaningful measures |
Chantal Mathieu, INNODIA; Marjana Marinac, JDRF; Kyle Jacques Rose, INNODIA PAC; Melissa Schwaber |
| 10:45 | FDA Perspective: Clinical endpoints and validated surrogates | Lauren Wood Heickman, FDA |
| 10:55 | EMA Perspective: Clinical endpoints and validated surrogates | Carine de Beaufort, EMA |
| 11:05 | General considerations for trial design for confirmatory endpoints | Allison Goldfine, Novartis Institutes of Biomedical Research |
| 11:20 | Additional Clinical Outcomes: Considerations and current limitations | Joe Hedrick, Janssen |
| 11:35 | Panel Discussion: Discuss relevant perspectives |
Moderators: Session III co-chairs Panelists: Session III speakers |
| 12:15 | Break: 20 Minutes | |
| 12:35 | Session IV: Overall Issues of Study Design |
Session Co-chairs: Inish O’Doherty, C-Path + Peter Gottlieb, University of Colorado |
| 12:40 | Ethical Considerations of Trial Design | Donna Snyder, FDA |
| 13:00 | Statistical Considerations for Trial Design and Feasibility | Tee Bahnson, Benaroya Research Institute |
| 13:15 | Considerations for Trial Design and Feasibility | Regine Bergholdt, Novo Nordisk |
| 13:30 | Final Panel Discussion/Open Comment |
Moderators: Session IV co-chairs Panelists: Panelists: Session IV speakers + Francisco Leon, Provention Bio |
| 14:30 | Workshop Closing Remarks | Lisa Yanoff, FDA |
