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)TitlePresenter
10:00Welcoming Remarks and HousekeepingInish O’Doherty, C-Path
10:05FDA Introductory RemarksIlan Irony, FDA
10:15Patient Perspective Opening Remarks: Unmet NeedAaron Kowalski, JDRF
10:25Session I: Regulatory Framework for Clinical Investigations in New/Recent Onset T1D
10:25FDA perspectiveKristen Pluchino, FDA
10:45EMA perspectivePeter Mol, EMA
11:05Break: 20 minutes
11:25Session II: Scientific Framework: The rationale for C-peptide preservation and use as a clinical trial endpointSession Co-chairs:

Chantal Mathieu, INNODIA + Patricia Beaston, FDA

11:30C-Peptide as Primary Endpoint & Natural HistoryKevan Herold, Yale University
12:00Islet Transplantation:  Relationship of C-peptide and clinically meaningful outcomesMichael Rickels, University of Pennsylvania
12:20Differential Rates of C-Peptide DeclineCarla Greenbaum, Benaroya Research Institute
12:40C-Peptide as a Primary EndpointStephen Gough, Novo Nordisk
13:00Panel DiscussionModerators: Session II co-chairs

Panelists: Panelists: Session II speakers + Mark Peakman, Sanofi

13:40Day 1 Closing Remarks

Agenda – Day 2

Time (EST)TitlePresenter
10:00Day 2 Opening Remarks
10:10Session III: Establishing/Confirming Clinical Benefit

Session Co-chairs:

Lisa Yanoff, FDA + Colin Dayan, Cardiff University

10:15Perspective from people living with T1D: Clinically meaningful measures

Chantal Mathieu, INNODIA;

Marjana Marinac, JDRF;

Kyle Jacques Rose, INNODIA PAC;

Melissa Schwaber

10:45FDA Perspective: Clinical endpoints and validated surrogatesLauren Wood Heickman, FDA
10:55EMA Perspective: Clinical endpoints and validated surrogatesCarine de Beaufort, EMA
11:05General considerations for trial design for confirmatory endpointsAllison Goldfine, Novartis Institutes of Biomedical Research
11:20Additional Clinical Outcomes: Considerations and current limitationsJoe Hedrick, Janssen
11:35Panel Discussion: Discuss relevant perspectives

Moderators: Session III co-chairs

Panelists: Session III speakers

12:15Break: 20 Minutes
12:35Session IV: Overall Issues of Study Design

Session Co-chairs:

Inish O’Doherty, C-Path + Peter Gottlieb, University of Colorado

12:40Ethical Considerations of Trial DesignDonna Snyder, FDA
13:00Statistical Considerations for Trial Design and FeasibilityTee Bahnson, Benaroya Research Institute
13:15Considerations for Trial Design and FeasibilityRegine Bergholdt, Novo Nordisk
13:30Final Panel Discussion/Open Comment

Moderators: Session IV co-chairs

Panelists: Panelists: Session IV speakers + Francisco Leon, Provention Bio

14:30Workshop Closing RemarksLisa Yanoff, FDA
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