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 |
|---|---|---|
| Chair | Alain Romero, PharmD | KHI Board of Directors Liaison; Independent Biotechnology and Pharmaceutical Consultant |
| Chair | Santosh Saraf, MD | Project Chair, University of Illinois Chicago |
| Member | Kirk Campbell, MD | Mount Sinai |
| Member | Jeff Lebensburger, DO | University of Alabama at Birmingham |
| Member | Vimal Derebail, MD, MPH | University of North Carolina |
| Member | Kenneth Ataga, MD | University of Tennessee |
| Member | Jonathan Sorof, MD | Independent Biotechnology and Pharmaceutical Consultant |
Deliverables:
- Framework for clinical/regulatory pathways for SCKD drugs in development
- Manuscripts
If you have any questions, please contact KHI at khi@asn-online.org.