Reducing The Impact Of Chronic Kidney Disease (CKD): Opportunities For Randomized Clinical Trials
 - July 19–20, 2011 - Natcher Conference Center
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AGENDA

DAY 1:  Tuesday, July 19, 2011 
   
7:00 – 8:00 a.m. Registration
   
8:00 – 8:15 a.m. Opening Remarks and Objectives
Dr. Robert Star and Dr. Michael Flessner
   
8:15 – 9:45 a.m. State-of-the-Art Lectures
Moderator:  Dr. Glenn Chertow
  • Lessons from CRIC and CKid:  Defining Risk Factors Relevant to CKD Trials
    Dr. Harold Feldman and Dr. Susan Furth
  • Lessons from CKD Trials (TREAT, AASK, RENALL)
    Dr. Robert Toto
  • Why Good Trials Go Bad
    Dr. Tom Greene
   
9:45 – 10:00 a.m. BREAK
   
10:00 – 12:30 p.m.   Challenges in Clinical Trial Design (I) – Planning Studies for CKD
Moderator:  Dr. Laura Dember
  • Selection of Study Populations:  Prevention or Interventions to Slow Progression?
    Dr. Julie Lewis
  • Renal Outcomes for Phase III Trials
    Dr. Lawrence Appel
  • Biomarkers for CKD Progression or Risk of Progression
    Dr. Patrick Murray
  • Effect Size and Sample Size Calculations
    Dr. Glenn Chertow
  • Adaptive Designs to Increase Efficiency for Phase II/III Trials
    Dr. Michael Rosenblum
  • SPRINT:  High Risk CKD Subgroup Within a Larger Clinical Trial
    Dr. Alfred Cheung and Dr. David Reboussin
   
12:30 – 1:15 p.m. Lunch (Pre-Purchased Box Lunches in Breakout Rooms)
   
1:15 – 3:40 p.m. Therapies in Phase II/Pilot Studies
Moderator:  Dr. Flessner
  • Allopurinol
    Dr. Allesandro Doria
  • Antioxidants
    Dr. Jonathan Himmelfarb
  • Bicarbonate
    Dr.  Michal Melamed
  • Mineralocorticoid Receptor Blockers
    Dr. Robert Toto
  • Non-Pharmacologic Therapies:  Glucose Control, Weight Loss, and Sodium Restriction
    Dr. William Mitch
  • Pentoxifylline
    Dr. Robert Perkins
  • Pirfenidone
    Dr. Kumar Sharma
  • Vitamin D/Phosphate Binding/Dietary Intervention
    Dr. Geoff Block
   
3:45 – 5:15 p.m.

Breakout Sessions
Purpose:  To discuss the critical design elements that are required to bring a medication or maneuver to a Phase III trial. 

Topics for Individual Breakout Sessions:

Group 1
Trial in Children:  Agent To Prevent Progression
Moderators:  Dr. Susan Furth, Dr. Russell Chesney, Dr. James Chan, Dr. Frederick Kaskel, Dr. Lynne Yao, and Dr. David Reboussin

Group 2
Trial in Adults with Bicarbonate 
Moderators:  Dr. Michal Melamed, Dr. Donald Wesson, and Dr. Tom Greene

Group 3
Trial in Adults with Pentoxifylline
Moderators:  Dr. Robert Perkins and Dr. Linda Fried, NIH Biostatistician

Group 4
Trial in Adults with Mineralocorticoid Receptor Blocker
Moderators:  Dr. Robert Toto, Dr. Tom Hostetter, and Dr. Michael Rosenblum

Group 5
Trial in Adults with Phosphate Control/Vitamin D: 
Moderators:  Dr.  Myles Wolf, Dr. Rajnish Mehrota, Dr. Ravi Thadani, and Dr. Glenn Chertow, NIH Biostatistician

Each Group Will Consider the Following Issues:

  1. Define Trial Question:  Preliminary Data?

  2. Renal Endpoints for Phase III CKD Trial  
    1. Proteinuria as an endpoint? Are there other surrogate endpoints?
    2. Creatinine (or cystatin C)-based endpoints:  Doubling? Slope?
    3. Are CKD staging tools feasible endpoints?
    4. What are the obstacles that have prevented the design and execution of outcome studies for renal-specific endpoints?

  3. Non-Renal Endpoints for Phase III CKD Trials
    1. Non-renal endpoints (CVD, mortality, hospitalization)
    2. Which should be used in either Phase II or Phase III studies?
    3. Should these be primary or secondary endpoints in Phase III studies?

  4. Patient Selection and Recruitment
    1. Inclusion/exclusion criteria  (Early disease vs. late disease)
    2. Impact of patient selection on design of study
    3. Should a trial be restricted to proteinuric disease or have a stratum with a target proportion with proteinuria? If yes, how would proteinuria be defined?
    4. Is the recruitment strategy feasible?
    5. Biomarkers for risk stratification
    6. Incorporation of Protocolized Adjunctive Care (Will this agent be added to ACEI or ARB or be substituted for the current standard of care?)

  5. Adverse Events/Pilot and Feasibility Issues:  Dose Selection

  6. Sample Size and Power
   
5:15 – 6:00 p.m. Preliminary Reports From the Breakout Sessions and General Discussion 
   
6:00 p.m. Adjournment
   
DAY 2:  Wednesday, July 20,  2011
   
7:00 – 8:00 a.m. Registration
   
8:00 – 9:00 a.m. Government-Academic-Industry Partnerships
Moderator:  Dr.  Michael Flessner
  • Regulatory Issues:  FDA Viewpoint
    Dr. Aliza Thompson
  • Public Private Partnerships/Collaborative Opportunities/An NIH View
    Dr. Barbara Mittleman
  • Intellectual Property – An NIDDK View
    Ms. Anna Amar
   
9:00 – 9:30 a.m. Break
   
9:30 – 12:00 p.m. Ongoing Industry Trials/Research and Development
Moderator:  Dr. David Warnock 
  • Bardoxolone:  BEACON Trial:
    Dr. Paul Audhya and Dr. Colin Meyer, Reatta Pharmaceuticals
  • CTP-499, a First-in-Class Clinical Development Candidate for Diabetic Nephropathy
    Dr. James Shipley, CoNCERT Pharmaceuticals
  • TBD
    Dr. Thomas Daniel, Celgene Corporation
  • Atrasentan
    Dr. Dennis Andress, Abbott
  • AST-120/EPPIC Program
    Dr. Alan Glicklich, Mitsubishi Tanabe Pharma America
  • ALTITUDE Trial
    Dr. Marc Pfeffer
  • Development of Acthar for Treatment of Diabetic Nephropathy
    Dr. David Young, Questcor Pharmaceuticals

Industry Panel Discussion:  Speaker Panel and Moderator

   
Noon – 3:00 p.m.

Breakout Sessions (Working Lunch) Continued from Day One

Topics for Individual Breakout Sessions:

Group 1
Trial in Children:  Agent To Prevent Progression
Moderators:  Dr. Susan Furth, Dr. Russell Chesney, Dr. James Chan, Dr. Frederick Kaskel, Dr. Lynne Yao, and Dr. David Reboussin

Group 2
Trial in Adults with Bicarbonate 
Moderators:  Dr. Michal Melamed, Dr. Donald Wesson, and Dr.  Tom Greene

Group 3
Trial in Adults with Pentoxifylline
Moderators:  Dr.  Robert Perkins and Dr. Linda Fried, NIH Biostatistician

Group 4
Trial in Adults with Mineralocorticoid Receptor Blocker
Moderators:  Dr. Robert Toto, Dr. Tom Hostetter, and Dr. Michael Rosenblum

Group 5
Trial in Adults with Phosphate Control/Vitamin D 
Moderators:  Dr. Myles Wolf, Dr. Rajnish Mehrota, Dr. Ravi Thadani, and Dr. Glenn Chertow, NIH Biostatistician

   
3:00 – 5:00 p.m. Reports From the Breakout Sessions:  Discussion and Next Steps
   
5:00 – 5:15 p.m.

Summary
Dr. Robert Star and Dr. Michael Flessner

   
5:15 p.m. Adjournment
   
NIH NIDDK DHHS