VIEW NOW | Design of Clinical Trials in New-Onset Type 1 Diabetes: Regulatory Considerations for Drug Development

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.

Workshop Summary

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

 

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