Clinical Trial

CRISPR-Edited HLA Donor Kidney Transplant to Reduce Rejection Risk

Recruiting Phase 1/2
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Summary
This clinical trial investigates the transplantation of donor kidneys that have been genetically modified ex vivo using CRISPR-Cas9 genome editing to reduce immunogenicity and transplant rejection. Donor kidney grafts will have key human leukocyte antigen (HLA) genes disrupted - specifically, knockout of HLA class I heavy chains HLA-A and HLA-B, along with disabling HLA class II expression by targeting the CIITA gene (a master regulator of HLA-DR/DQ/DP). Approximately 90 adult end-stage renal disease patients will receive a CRISPR-edited donor kidney transplant. The primary objectives are to assess the safety and feasibility of this novel intervention, while secondary objectives evaluate the reduction in immune responses (immunogenicity), graft function, and the practicality of implementing ex vivo gene-edited organ transplantation in humans. By knocking out major donor HLA molecules, the trial aims to reduce T-cell and antibody-mediated recognition of the graft, potentially lowering rejection rates and reliance on high-dose immunosuppressants. Safety, including any off-target effects or unanticipated immune reactions, will be closely monitored, and transplant outcomes will be tracked for one year post-transplant.
Trial Details
NCT Number NCT07053462
Lead Sponsor AMERICAN ORGAN TRANSPLANT AND CANCER RESEARCH INSTITUTE LLC
Conditions End-Stage Renal Disease, End Stage Renal Disease on Dialysis, End Stage Renal Disease With Renal Transplant, Kidney Transplant Rejection, Kidney Tumor, Kidney Failure, Kidney Ischemia
Enrollment 90 participants
Start Date 2025-06-01
Primary Completion 2027-12-18 (estimated)
Study Completion 2028-12-28 (estimated)
Updated on ClinicalTrials.gov 2025-07-08