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To gain a better understanding of the kidney patient therapeutic journey and to define candidate indicators for precision medicine pathway, target and trial readiness. This workshop will identify kidney precision medicine clinical trial barrier and potential solutions and to identify concepts relative to advancing precision medicine clinical trials not yet considered.
Meeting Objectives
Bring in the patient's voice to advance treatments for kidney disease.
Define candidate indicators for precision medicine pathways, targets, and trial readiness.
Identify kidney precision medicine clinical trial barriers and potential solutions.
Identify concepts relative to advancing precision medicine clinical trials not yet considered.
Background
Progressive kidney disease affects how individuals feel, function, and survive. Therapies for individuals with kidney disease have only modest efficacy; both risk for severe toxicity and selection of treatments are not well informed by precise assessment of disease mechanism(s) active at the time of decision-making. In this standard of care context, many people with chronic kidney disease experience a series of therapies with limited efficacy and, ultimately, progress to kidney failure. Scientific advances are increasing awareness and improving the ability to assess molecular mechanisms of kidney disease and elucidation of associated therapeutic targets.
Organizing Committee
External Members
Joseph Bonventre
Harvard University; Brigham and Women’s Hospital
Glenn M. Chertow
Stanford University
Paul T. Conway
American Association of Kidney Patients
Rasheed Gbadegesin
Duke University
Melanie Joy
University of Colorado
Matthias Kretzler
University of Michigan
Mark Lim
Kidney Health Initiative
Kathleen Liu
University of California-San Francisco
Aliza Thompson
U.S. Food and Drug Administration
NIDDK
Debbie Gipson, Kevin Abbott, Kevin Chan, Raquel Greer, Paul Kimmel, Susan Mendley, Afshin Parsa, Tracy Rankin, Cindy Roy, Ivonne Schulman
Registration Deadline
March 17, 2024
Agenda
Day One—March 18, 2024
9:30 a.m. – 9:45 a.m.
Welcome and Workshop Framing: Preparing for ^More^ Kidney Precision Medicine Trials
Debbie Gipson, M.D., M.S., National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH)
9:45 a.m. – 9:50 a.m.
Welcome
Robert Star, M.D., Director, Division of Kidney, Urologic & Hematologic Diseases, NIDDK, NIH
9:50 a.m. – 10:30 a.m.
Kidney Patient Panel: Sharing Kidney Treatment Experiences
Moderators: Sandra Amaral, MD, Children’s Hospital of Philadelphia and Glenn Chertow, MD, Stanford University
Panelists: Patients and Family
10:30 a.m. – 11:30 a.m.
Scientific Considerations for Kidney Precision Medicine Trials
Moderators: Rasheed Gbadegesin, MD, Duke University and Javier Neyra, MD, MSCS, University of Alabama - Birmingham
Acute Kidney Injury (AKI) Endophenotypes, Mechanisms, and Targets Pavan Bhatraju, M.D., University of Washington
Chronic Kidney Disease (CKD) Endophenotypes, Mechanisms, and Targets Matthias Kretzler, M.D., University of Michigan
Interventions: Drug Screening, Development, and Optimization Hassan Ali, Ph.D., University of Miami
* Q&A follows each presentation
11:30 a.m. – 11:45 a.m.
Break
11:45 a.m. – 12:30 p.m.
Preparing the Precision Trials Toolbox
Moderators: Joseph Bonventre, MD, PhD, Brigham & Women’s Hospital and Ian de Boer, MD, MS, University of Washington
Honing Precision Trial Design Components: Population, Biomarkers, and Endpoints H. J. Lambers Heerspink, Pharm.D., Ph.D., University of Groningen
A Laboratorian’s Perspective on Assay Development Fit for Purpose in Precision Medicine Trials Chris Karlovich, PhD, Director, Molecular Characterization Laboratory, Frederick National Laboratory for Cancer Research
Questions
12:30 p.m. – 1:10 p.m.
Lunch and Networking
1:15 p.m. – 2:30 p.m.
Perspectives from Regulatory, Industry, and Patient Advocacy
FDA Panel Perspectives on Precision Medicine Trials Moderators: Aliza Thompson, FDA and Carla Nester, MD, University of Iowa
Michael Pacanowski, Pharm.D., MPH, Division of Translational and Precision Medicine, Center for Drug Evaluation and Research (CDER); Irene Tebbs, PhD, Center for Devices and Radiologic Health, Rekha Kambhampati, M.D., Division of Cardiology and Nephrology Division, CDER
Industry Panel Perspectives on Precision Medicine Trials Moderators: Uptal Patel, MD, HiBio and Kidney Health Initiative and Danny Gossett, PhD, NIDDK
Jerome Rossert, MD, PhD, AstraZeneca, Matthew Breyer, MD, Janssen, Susan Limb, MD, Maze Therapeutics
Advocacy Perspective on Balancing Aspiration and Uncertainty in Precision Medicine Trials Moderators: Mark Lim, PhD and Kevin Chan, MD, M.Sci
Paul Conway, American Association of Kidney Patients
2:30 p.m. – 2:45 p.m.
Break
2:45 p.m. – 3:55 p.m.
Breakouts: Assess Current State of Kidney Precision Trial Preparation and Opportunities for Advancement
All Participants
Patient and Clinician Community
Facilitators: Kevin Fowler, Voice of the Patient and Paul Kimmel, MD, NIDDK
Disease Mechanisms-Biomarkers-Endpoints
Facilitators Joseph Bonventre, MD, PhD, Brigham & Women’s Hospital, Marcus Pezzolesi, PhD, MPH, University of Utah, and Jason Conage-Pough, PhD, NIDDK
Defining Evidentiary Thresholds for Precision Trial Activation
Facilitators Matthias Kretzler, MD, University of Michigan and Rasheed Gbadegesin, MD, MBBS, Duke University
Concurrent Breakouts for Virtual Attendees
Patient and Clinician Community
Facilitators: Paul Conway, AAKP and Kirk Campbell, MD, Mount Sinai
What do we know and how do we know it? Defining evidentiary thresholds for mechanisms, biomarkers and endpoints for precision trial activation
Facilitator: Laura Mariani, MD, MS, University of Michigan and Kim Reidy, MD, Albert Einstein College of Medicine
4:10 p.m. – 4:45 p.m.
Breakout Reports
Moderator: Kerry Willis, National Kidney Foundation
4:50 p.m. – 5:00 p.m.
Wrap-Up and Directions for Day 2
Day Two—March 19, 2024
9:00 a.m. – 9:10 a.m.
Welcome
9:10 a.m. – 9:30 a.m.
Precision Medicine Trial Program Exemplar
Moderator: Kathleen Liu, MD, University of California-San Francisco and Afshin Parsa, MD, NIDDK
Speaker: I-SPY: Laura Esserman, MD, University of California at San Francisco
9:30 a.m. – 9:45 a.m.
Q&A
9:45 a.m. – 11:00 a.m.
With a Little Help from Our Friends
Moderator: Melanie Joy, PharmD, PhD, University of Colorado and Susan Mendley, MD, NIDDK
Polycystic Kidney Disease: Academician and Biotech Partnership to Advance from Mechanisms to Trials
Vishal Patel, MD, The University of Texas Southwestern Medical Center
Testing the Right Drug and Right Dose, Pharmacology and Pharmacogenetics
Julie A Johnson, PharmD, The Ohio State University
Making Timely Matches in Precision Clinical Trial Platforms
Latifa Jackson, PhD, Director of Bioinformatics, Center for Biomedical Informatics and Information Technology (CBIIT), NCI and Brent Coffey, MS, MBA, Essex
* Q&A follows each presentation
11:00 a.m. – 11:15 a.m.
Break
11:15 a.m. – 12:30 p.m.
Breakouts: Identifying Opportunities and Potential Challenges to Kidney Precision Medicine Trials
All Participants
AKI Precision Trial Teams, Components, and Capacity
Facilitators: Kathleen Liu, MD, Chirag Parikh, MD, PhD, Johns Hopkins School of Medicine, Ivonne Schulman, MD, NIDDK
CKD Precision Trials Teams, Components, and Capacity
Facilitators: Mark Lim, PhD, Kidney Health Initiative and Prabir Roy-Chaudhury, MD, PhD, University of North Carolina
Drug and Dose Optimization {Hybrid}
Facilitators: Melanie Joy, PharmD, PhD, Julie Johnson, PhD, and Anna Sadusky, PhD, NIDDK
Concurrent Breakouts for Virtual Attendees
AKI Trials Teams, Components and Capacity
Facilitator: F. Perry Wilson, MD, MSCE, Yale University, Edward Siew, MD, MSCI, Vanderbilt University and Jenna Norton, PhD, NIDDK
CKD Trial Teams, Components and Capacity
Facilitators: James Lash, MD, University of Illinois-Chicago and Raquel Greer. MD, NIDDK
12:30 p.m. – 1:15 p.m.
Lunch and Networking
1:15 p.m. – 2:15 p.m.
Breakout Reports
Moderator: Glenn Chertow, MD
2:15 p.m. – 2:30 p.m.
Summary and Next Steps
Robert Star and Debbie Gipson, NIDDK
2:30 p.m.
Adjournment
Event Logistics
Registration
Registration Closed
Registration ended
Location
Natcher Conference Center NIH Main Campus Building 45 Bethesda,MD
This is a hybrid workshop. Virtual participation is available. For those attending via webinar, the link will be distributed via email prior to the date of the event.
