NCT07053462

CRISPR-Edited HLA Donor Kidney Transplant to Reduce Rejection Risk

Recruiting now Phase 1, PHASE2 Last updated 8 July 2025

What this trial tests

Phase 1, PHASE2 trial testing Ex Vivo CRISPR-Cas9 Gene Editing of Donor Kidney in End-Stage Renal Disease in 90 participants. Currently enrolling.

Timeline

1 June 2025

Primary endpoint
18 December 2027

28 December 2028

Quick facts

Lead sponsor	AMERICAN ORGAN TRANSPLANT AND CANCER RESEARCH INSTITUTE LLC
Phase	Phase 1, PHASE2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	90
Start date	1 June 2025
Primary completion	18 December 2027
Estimated completion	28 December 2028
Sites	1 location across China

Drugs / interventions tested

• Ex Vivo CRISPR-Cas9 Gene Editing of Donor Kidney — full drug profile →
• Kidney Transplantation with Standard Care

Conditions studied

• End-Stage Renal Disease — all drugs for End-Stage Renal Disease →
• End Stage Renal Disease on Dialysis — all drugs for End Stage Renal Disease on Dialysis →
• End Stage Renal Disease With Renal Transplant — all drugs for End Stage Renal Disease With Renal Transplant →
• Kidney Transplant Rejection — all drugs for Kidney Transplant Rejection →

Sponsor

AMERICAN ORGAN TRANSPLANT AND CANCER RESEARCH INSTITUTE LLC

Who can join

Adults 16 to 85, any sex, with End-Stage Renal Disease or End Stage Renal Disease on Dialysis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.