Clinical Trial

Disease: End-Stage Renal Disease, (NCT07053462)

Disease info:

End-stage renal disease (ESRD) is the final stage of chronic kidney disease, where kidney function has declined to the point that the kidneys can no longer function on their own.

Chronic kidney disease occurs when kidneys are damaged and can no longer function efficiently. The kidneys can no longer remove armful wastes from the body and these begin to accumalate causing blood pressure to rise, retention of excess fluid and failure to make enough red blood cells. The symptoms of kidney failure vary from person to person and may change as kidney failure progresses. These symptoms include fatigue, drowsiness, changes in urination, dry and itchy skin, headaches, unexplained weight loss, nausea, pain in bones, skin and nail changes and bruising.

For ESRD when kidneys have lost all function, treatment options are limited to dialysis or a kidney transplant.

Source: End Stage Renal Disease (ESRD) | Johns Hopkins Medicine & Kidney Failure: MedlinePlus

 

Frequency:
According to the National Center for Chronic Disease Prevention and Health Promotion, about 30 million people in the U.S. are estimated to have chronic kidney disease.
Official title:
Phase 1/2, Single-Arm, Open-Label Trial to Evaluate the Safety, Feasibility, and Immunogenicity of Ex Vivo CRISPR-Cas9 Gene-Edited Donor Kidneys (Knockout of HLA-A, HLA-B, and CIITA) in Human Renal Transplant Recipients
Who:

Contact 

Name: Andrew R Linehan

Phone: +1 (302) 615-8388

Email: clinical-trials@aotcri.org

Sponsor:

AMERICAN ORGAN TRANSPLANT AND CANCER RESEARCH INSTITUTE LLC

Partners:
Locations:

Changping, China

Peking University Health Science Center (PKUHSC), Beijing, Changping, China, 102206
 

Study start:
Jun. 1, 2025
Enrollment:
90 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene knockout
Gene:
Gene knockout of HLA-A and HLA-B and CIITA
Delivery method:
- Ex-vivo
Trial link:
https://clinicaltrials.gov/study/NCT07053462?term=crispr&rank=2
IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

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.

Last updated: Aug. 2, 2025
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