Arrival to the Embassy of France

By Course Director Professor Patrick Rossignol (Monaco, MON) on behalf of the scientific committee

Moderator: Prabir Roy-Chaudhury (Chapel Hill, USA)

Discussant (5 minutes):

Patient Perspective: Nichole Jefferson (Home Dialyzors United, USA)

Speakers (10 minutes each):

• Academic perspective: Glenn Chertow (Stanford, USA) - Anti-inflammatory therapies in ESKD

• Academic perspective: Jonathan Himmelfarb (Mount Sinai, USA) - Innovative Renal Replacement Approaches

Discussants (5 minutes each):

• Academic perspective: Ron Gansevoort (Groningen, NL) -ACE/ARB-SGLT-2i-MRA-GLP-1 in ESKD: What are the Right Endpoints?

• Academic perspective: Jennifer Flythe (University of North Carolina, USA) - Patient Centered Clinical Trial Endpoints for Device Studies

• Industry perspective: Ven Manda (President, Mozarc) - Creating and Innovation Substrate for Novel Dialytic Therapies

• Industry perspective: : Rob Kossman (Fresenius Medical Care, USA) - Implementation of New Therapies in ESKD:  of Patients, Policy, and Payment

• FDA-CDRH: Gema Gonzalez

• FDA-CDER: Aliza Thomson

• NIH: Kevin Abbott

• CMS: Abigail Ryan

Panel discussion with all the above

Moderator: Prabir Roy-Chaudhury (Chapel Hill, USA)

Discussant (5 minutes):

Patient Perspective: Caroline Wilkie (National Kidney Foundation, USA) - Vascular Access is my Lifeline.

Speakers (10 minutes each):

• Academic perspective: Joris Rotman (University of Leiden, NL). New Therapies for Vascular Access

• Academic perspective: Robert Lee (Michigan, USA). Clinical Trial Designs for Vascular Access Trials

Discussants (5 minutes each):

• Academic perspective: Vandana Niyyar (Emory, USA) - Choosing the Right End Point for the Right Device

• Academic perspective: Prabir Roy-Chaudhury (University of North Carolina, USA): Precision Medicine Approaches to Vascular Access Care

• Industry perspective: Andrea Acuna (BD, Phoenix, USA)

• Industry perspective: Anna Szafranski (Medtronic, Minneapolis St Paul, USA)

• Industry perspective: Mahesh Krishnan (Davita, Washington DC) - Implementation of Vascular Access Innovation into Dialysis Care Pathways

• NIDDK: Danny Gossett

• FDA: Gema Gonsalez or designee

• CMS: Abigail Ryan

Panel discussion with all the above

In the lobby of the Maison Française

Moderators: TBD

Speakers (10 minutes each):

• Investigator’s perspective: Lesley Inker (Boston, US)

• Speaker 2

Discussants (5 minutes each):

• Discussant 1: TBD

• Discussant 2: TBD

Discussion between NIDDK, FDA, EMA, Patient and Payer perspectives

In the lobby of the Maison Française

Moderator: Adeera Levin (Vancouver, CAN)

Speakers (10 minutes each):

• Pediatrician : Brad Warady  (Kansas City, US)

• Pediatrician : Howard Trachtman (Michigan, US)

• Academic perspective: Mathias Kretzler (Ann Arbor, USA)

• Academic perspective: Josef Coresh (Baltimore, USA)

Discussants (5 minutes each):

• Academic perspective: Alissa Forni (Miami, USA)

• Academic perspective: Morgan Grams (New York, USA)

• Academic perspective: Michelle Denburg (Philadelphia, USA)

• Industry perspective: Marvin Sinsakul (AstraZeneca, USA)

• Industry perspective: Gordana Atanackovic (Bayer, USA)

• Bayer discussant: TBD

• NIDDK: Debbie Gipson

• FDA: TBD

• CMS: TBD

Panel discussion with all the above

Moderator: Prabir Roy-Chaudhury (Chapel Hill, USA)

Discussant (5 minutes):

• Patient Perspective: Glenda Roberts (Mt. Sinai, USA) - Who Should we Test These Novel Therapies On?

Speakers (10 minutes each):

• Joseph Bonventre (Harvard, USA) - Kidney Regenerative Approaches

• Vineeta Kumar (University of Alabama, USA) - Clinical Trial Design for Xenotransplantation

Discussants (5 minutes each):

• Academic perspective: Robert Montgomery (New York University, USA) - From Conventional Dialysis and Transplant to Xenotransplants and Regenerated Kidneys: How do we decide who gets what…

• Academic perspective: Mark Lim (Washington, USA)

• Industry perspective: Martine Rothblatt, President and CEO United Therapeutics (Silver Spring, Maryland)

• Industry perspective: Matt Tector, CSO, Makana Therapeutics

• NIDDK: Eric Brunskill, Program Officer

• FDA: Celia Witten, Deputy Director, CBER

• Bioethics of Xenotransplantation: Robyn Shapiro, Health Sciences Law Group

Panel discussion with all the above

In the lobby of the Maison Française

Moderators: Glenn Chertow (Stanford, USA) & Vlado Perkovic (Sydney, AUS)

Defining the objectives of a clinical trial is crucial to align design, analysis, and interpretation. With the recent release of an addendum to the E9 guideline on 'Statistical principles in clinical trials' by the International Council of Harmonization (ICH), regulatory agencies require the specification of estimand(s) which precisely describe the treatment effect(s) of interest in the study protocol. In this session the integration of estimands into kidney disease trials from both clinical and statistical perspectives will be explored. Clinically, estimands allow for more accurate measurement of treatment effects by incorporating so-called intercurrent events such as treatment discontinuation or use of rescue medications. Statistically, estimands need to be aligned with analytical methods, ensuring that sensitivity analyses are robust and account for missing data. This approach enhances the interpretability of trial results, improving decision-making in nephrology research and regulation. 

Speakers (10 minutes each):

• Statistician’s perspective: Tim Friede (Göttingen, GER)

• Academic perspective: Jule Pinter (Würzburg, GER)

Discussants (5 minutes each):

• Regulators’ perspective: NN (FDA), NN (EMA or European national regulatory agency)

  • Industry statistician’s perspective:  Fred Yang (KBP, US)

• Industry statistician’s perspective:  Svenja Seide (Boehringer Ingelheim, GER)

• Industry statistician’s perspective: Melanie Wright (Novartis)

Panel discussion with all the above

7:00 PM - Dinner & adjourn

In the Lobby of la Maison Française

Moderators: Vlado Perkovic (Sydney, AUS) & Hiddo Heerspink Lambers (Groningen, NED)

Speaker (15 minutes):

• Academic perspective: Jürgen Floege (Aachen, GER)

• Academic perspective: Jonathan Barratt (Leicester, GBR)

• Academic perspective: Dana Rizk (Birmingham, USA )

Discussants (5 minutes each):

• Industry perspective: TBD (Novartis)

• Industry perspective: Radko Komers (Travere, USA)

• Industry perspective: Kerry Cooper (Vera Therapeutics, USA)

• Industry perspective: Eric Rowinsky (Biocity Biopharmaceutics)

• Industry perspective: Jay Garg (Genentech)

• NIDDK: Ivonne Schulman

• FDA: TBD

• CMS: TBD

Panel discussion with all the above

In the lobby of la Maison Française

Moderators: Vlado Perkovic (Sydney, AUS) & Hiddo Heerspink Lambers (Groningen, NED)

Having established relevant endpoints for IgAN (with already several drugs approved), and now FSGS,  membranous nephropathy coming next, must  specific endpoints be considered for each  glomerular disease vs generic core outcomes ? Potentially implemented in platform trials ?

Discussant (5 minutes ):

• Patient’s perspective: Josh Tarnoff (NephCure, USA)

Speakers (10 minutes each):

• Insights from the PARASOL initiative: Brad Rovin (Colombus, USA)

Discussants (5 minutes each):

• Academic perspective: Mathias Kretzler (Ann Arbor, USA)

• Academic perspective: Anna Greka (Boston, USA)

• KHI perspective: Uptal Patel (KHI, USA)

• Academic perspective: Jerome Rossert (Boston, USA)

• Industry perspective: Kerry Cooper (Vera Therapeutics, USA)

• Industry perspective: David Fuller (Dimerix)

• Industry perspective: Dominik Steubl (Boehringer-Ingelheim)

• Industry perspective: Jay Garg (Genentech)

• NIDDK: Ivonne Schulman

• FDA: TBD

• CMS: TBD

Panel discussion with all the above

Moderators: Adeera Levin (Vancouver, CAN) & Christoph Wanner (Würzburg, GER)

Speakers (10 minutes each):

• Platform Trial: Sradha Kotwal (Sydney, AUS)

• Academic perspective: Meg Jardine (Sydney, AUS)

• Adaptive trial: Apol K1mediated disease - Ogo Egbuna (Vertex, USA)

Discussants (5 minutes each):

• Academic perspective: Vlado Perkovic (Sydney, AUS)

• Academic perspective: Brad Rovin (Colombus, USA)

• Academic perspective: Melissa West (Washington, USA)

• How industry and academia are working together in platform trials: Heather Ascani (Michigan, USA)

• Academic perspective: Hiddo Heerspink (Groningen, NED)

• Insights from Covid platform trials: Yves Rosenberg (NHLBI, USA)

•  AKI (TBD)

•  Oncology trials : (TBD)

• Academic Funder’s perspective: Harold Feldman (PCORI, USA)

• Industry perspective: Martin Cowie (Astra-Zeneca)

• NIDDK perspective: Ivonne Schlumann

• Regulator’s perspective: TBD

• Payer’s perspective: TBD

Panel discussion with all of the above

In the lobby of la Maison Française

Moderator: Mathieu Legrand (San Francisco, USA)

Summary of the literature Review:

• Natalja Stanski (Cincinnati, USA)

• Dana Fuhrman (Pittsburgh, USA)

• Bruno Garcia (Brussels, BEL)

• Lui Forni (London, GRB)

• Ravindra Mehta (San Diego, USA)

• Marlies Osterman (London, GRB)

• Sean Bagshaw (Edmonton, CAN)

• Matthieu Legrand (San Francisco, USA)

• Patrick Rossignol (Monaco, MON)

• Jay Koyner (Chicago, USA)

• D’Arcy Duquette (Calgary, CAN)

Final words by Professor Patrick Rossignol and co-directors, and adjourn