Clinical Trials in Pancreatitis

Event Details

Meeting Objectives

• Identify gaps and recommendations
• Use workshop recommendations to inform possible future funding initiatives by NIDDK
• Publish a conference summary

Background

Recurrent acute and chronic pancreatitis are characterized by pain, disability, and late-stage complications. No U.S. Food and Drug Administration (FDA)–approved drugs currently exist to treat these diseases, but interest in developing therapeutic strategies and clinical trial guidelines has increased, both among the investigative community and in the FDA. This 1-day workshop will feature discussions to help attendees understand the principles and requirements of clinical trials for treatment approaches for these diseases.

Organizing Committee

Co-chairs

Philip Hart, The Ohio State University
Tonya Palermo, University of Washington

Federal

Dana Andersen, NIDDK
Jose Serrano, NIDDK
Aynur Unalp-Arida, NIDDK
Erica Lyons, FDA

Non-Federal

Vernon Chinchilli, The Pennsylvania State University
Zobeida Cruz-Monserrate, The Ohio State University
Evan Fogel, Indiana University
Chris Forsmark, University of Florida
Linda Martin, Mission: Cure
Søren Olesen, Aalborg University
Stephen Pandol, Cedars-Sinai Medical Center
Emily Perito, University of California, San Francisco
Anna Phillips, University of Pittsburgh
Hanno Steen, Harvard University
Temel Tirkes, Indiana University
David Whitcomb, University of Pittsburgh
Ying Yuan, MD Anderson Cancer Center

Registration Deadline

June 30, 2022

Agenda

July 20, 2022

8:00 a.m. – 9:50 a.m.

Session One: Introduction and Patient Perspectives
Keynote Lecture—Key Considerations in Clinical Trials
Patient Perspectives—The National Pancreas Foundation–U.S. Food and Drug Administration (FDA) Patient-Focused Drug Development Voice of the Patient Report
Considering Participant Burden to Optimize Recruitment

9:50 a.m. – 10:10 a.m.

Break

10:10 a.m. – 11:30 a.m.

Session Two: Outcomes and Endpoints in Clinical Trials
Defining Appropriate Patient/Subject Populations
Outcome Measures in Chronic Pancreatitis
Outcome Measures—A Regulatory Perspective

11:30 a.m. – 12:30 p.m.

Lunch

12:30 p.m. – 1:50 p.m.

Session Three: Considerations for Clinical Trial Design
Proof-of-Concept and Feasibility Trials
Trials to Assess Efficacy of Invasive Treatments
Approach to Complex or Innovative Trial Design

1:50 p.m. – 3:35 p.m.

Session Four: Other Considerations and Opportunities
Design and Conduct of Clinical Trials in Children
FDA Partnership, Scientific Public–Private Partnerships
Ethical Considerations in Clinical Trials
Opportunities for Investigators, including Early-Stage Investigators

3:35 p.m. – 3:55 p.m.

Break

3:55 p.m. – 4:45 p.m.

Session Five: Panel Discussion