EXTEND: A Prospective Study to Evaluate the Safety and Efficacy of GGTA1 KO Thymokidney XenoTransplantation in Patients With End-stage Renal Disease (ESRD)

NCT07224763 Phase 1/Phase 2 RECRUITING

The purpose of this study is to evaluate the safety and efficacy of the GGTA1 KO Thymokidney in patients with end-stage renal disease (ESRD) who are either not eligible for conventional allogeneic kidney transplantation (Group 1) or are on an Organ Procurement and Transplantation Network (OPTN) kidney transplant waitlist, but are more likely to die or go untransplanted within 5 years than receive a kidney transplant (Group 2). The study consists of xenotransplantation followed by a 24-week Post-transplant Follow-up Period (Part A) to evaluate the efficacy and safety objectives followed by a Long-term Follow-up Period (Part B) to evaluate participant survival, GGTA1 KO Thymokidney survival, and screening for zoonotic infections. Part B will continue for the lifetime of the participant or for 52 weeks following nephrectomy, if required.

Details

Lead sponsor	United Therapeutics
Phase	Phase 1/Phase 2
Status	RECRUITING
Enrolment	50
Start date	2026-03
Completion	2076-03

Conditions

• ESRD (End-Stage Renal Disease)
• Kidney Transplantation
• Xenotransplantation

Interventions

• GGTA1 KO Thymokidney

Primary outcomes

• Survival Rate of Patients with ESRD Receiving the GGTA1 KO Thymokidney at 24 Weeks Post Transplant — Day 0 (day of xenotransplantation) to 24 weeks post transplant
  Participant survival rate at 24 weeks post transplant.
• Survival Rate of the GGTA1 KO Thymokidney at 24 Weeks Post Transplant — Day 0 (day of xenotransplantation) to 24 weeks post transplant
  GGTA1 KO Thymokidney survival rate at 24 weeks post transplant. GGTA1 KO Thymokidney failure is defined as GGTA1 KO Thymokidney nephrectomy.
• Survival Time of Participants Receiving the GGTA1 KO Thymokidney — Day 0 (day of xenotransplantation) until death for any cause, assessed at least every 24 weeks after transplantation while the participant is alive, up to 50 years
  Participant survival post transplant. Participant survival is defined as time from xenotransplantation to death for any cause.
• Survival Time of the GGTA1 KO Thymokidney (Overall Survival) — Day 0 (day of xenotransplantation) until start of chronic dialysis, nephrectomy, or death, whichever occurs first, assessed at least every 24 weeks after transplantation while the participant is alive and the thymokidney is functional, up to 50 years
  Overall survival of the GGTA1 KO Thymokidney post transplant. Overall survival time of the GGTA1 KO Thymokidney is defined as time from xenotransplantation to GGTA1 KO Thymokidney nephrectomy or death, whichever occurs first.
• Survival Time of the GGTA1 KO Thymokidney (Death-censored Survival) — Day 0 (day of xenotransplantation) until start of chronic dialysis, nephrectomy, or death, whichever occurs first, assessed at least every 24 weeks after transplantation while the participant is alive and the thymokidney is functional, up to 50 years
  Death-censored survival GGTA1 KO Thymokidney. Death-censored survival time of the GGTA1 KO Thymokidney is defined as time from xenotransplantation to GGTA1 KO Thymokidney nephrectomy censored for death.
• GGTA1 KO Thymokidney Function Post Transplant (Endogenous GFR) — At 24 weeks post transplant
  Endogenous measured GFR (24-hour urine creatinine clearance) at 24 weeks post transplant.

Countries

United States