KHI Current Project

Sickle Cell Kidney Disease (SCKD): Natural History and Clinical Trial Endpoints

Kidney disease is a common complication of sickle cell disease. Nearly one-third of young SCD patients develop kidney disease, with this increasing to 68% in older patients. 12% of people with SCD develop irreversible kidney damage. Most SCD clinical trials exclude patients with kidney disease. The purpose of this project is to develop a framework for clinical/regulatory pathways for SCKD for drugs approved or in development. The goal is to improve management of SCKD and support organ preservation.

The goals of this project are to:

  • Characterize the natural history of SCKD and identify those at high risk of progression to CKD
  • Identify and support choice of endpoints, metrics, and effect sizes, including for albuminuria and GFR or others
    • Address measurement issues related to albuminuria and GFR
  • Recommend trial populations based on biomarker (eGFR microalbuminuria and other markers, eGFR predictability balancing risk and feasibility).

Steering Committee Members

Role Name Organization
ChairAlain Romero, PharmDKHI Board of Directors Liaison; Independent Biotechnology and Pharmaceutical Consultant
ChairSantosh Saraf, MDProject Chair, University of Illinois Chicago
MemberKirk Campbell, MDMount Sinai
MemberJeff Lebensburger, DOUniversity of Alabama at Birmingham
MemberVimal Derebail, MD, MPHUniversity of North Carolina
MemberKenneth Ataga, MDUniversity of Tennessee
MemberJonathan Sorof, MDIndependent Biotechnology and Pharmaceutical Consultant


  1. Framework for clinical/regulatory pathways for SCKD drugs in development
  2. Manuscripts

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