Study to Evaluate the Safety and Efficacy of the GGTA1 KO Thymokidney in Patients With ESRD

Not Applicable

Recruiting

• Conditions: ESRD (End-Stage Renal Disease); Kidney Transplantation; Xenotransplantation
• Interventions: Biological: GGTA1 KO Thymokidney

• Registration Number: NCT07224763
• Lead Sponsor: United Therapeutics

Brief Summary

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.

Detailed Description

This is a Phase 1/2/3, multicenter, open-label, safety and efficacy study of the GGTA1 KO Thymokidney in patients with ESRD. The study will be comprised of the following:

* A Screening Period up to 52 weeks.

* Part A consists of the GGTA1 KO Thymokidney transplantation followed by a 24-week Post-transplant Follow-up Period, including the evaluation of all study endpoints and safety assessments.

* Part B is a Long-term Follow-up Period that extends for the lifetime of participants who received the GGTA1 KO Thymokidney or for 52 weeks following nephrectomy, if required, including but not limited to documentation of participant survival, GGTA1 KO Thymokidney survival, and screening for zoonotic infections.

There will be 2 groups of participants enrolled in the study. Group 1 will be participants deemed ineligible for conventional allogeneic kidney transplantation due to medical reason(s). Group 2 will be participants on an OPTN kidney transplant waitlist but who are more likely to die or go untransplanted within 5 years than receive a kidney transplant.

For the purpose of the primary analysis, the end of the study is defined as the date of the final visit of the final participant in Part A of the study.

Study Timeline

• Status Verified: 2025-11
• Study First Submitted: 2025-11-04
• Study First Submitted QC: 2025-11-04
• Last Update Submitted: 2025-11-04
• Study First Posted: 2025-11-05
• Last Update Posted: 2025-11-05
• Study Start: 2026-03-01
• Primary Completion: 2029-03
• Study Completion: 2076-03-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
GGTA1 KO Thymokidney	GGTA1 KO Thymokidney	Participants will receive GGTA1 KO Thymokidney

Primary Outcome Measures

Name	Time	Method
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 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 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 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.
GGTA1 KO Thymokidney Function Post Transplant (Exogenous GFR)	At 24 weeks post transplant	Exogenous measured GFR (nuclear medicine GFR) at 24 weeks post transplant.
GGTA1 KO Thymokidney Function Post Transplant (Proliferative Responsiveness)	At 24 weeks post transplant	Proliferative responsiveness to source GGTA1 KO Pig versus third-party pig as measured by mixed lymphocyte reaction from baseline to 24 weeks post transplant.
Quality of Life in Participants Receiving the GGTA1 KO Thymokidney by EuroQol 5-Dimension 5-Level (EQ-5D-5L)	Baseline to 24 weeks post transplant	Change in the EQ-5D-5L from baseline to 24 weeks post transplant. The EQ-5D-5L questionnaire assesses health-related quality of life across 5 categories (Mobility, Self-Care, Usual Activities, Pain/Discomfort, and Anxiety/Depression).; Each category has 5 levels, ranging from 1 (no problems) to 5 (extreme problems or inability to perform the activity). Higher scores indicate a worse health outcome (more mobility issues, greater pain, more anxiety, etc).
Quality of Life in Participants Receiving the GGTA1 KO Thymokidney by Standardized Outcomes in Nephrology Life Participant (SONG-LP)	Baseline to 24 weeks post transplant	Change in SONG-LP from baseline to 24 weeks post transplant. The SONG-LP assesses participation in different life activities over the past month. The minimum score (worst outcome) is 4 (if all responses are "1" - Never) and the maximum score (best outcome) is 20 (if all responses are "5" - Always). Higher scores indicate a better health outcome (greater ability to participate in activities).
Quality of Life in Participants Receiving the GGTA1 KO Thymokidney by Kidney Transplant Questionnaire (KTQ)	Baseline to 24 weeks post transplant	Change in KTQ from baseline to 24 weeks post transplant. Each question in the KTQ is scored on a 1 to 7 scale with: 1 = worst outcome (eg, "A very great deal of trouble or distress" / "All of the time") and 7 = best outcome (eg, "No trouble or distress" / "None of the time"). Higher scores indicate a better outcome, meaning less distress, fewer symptoms, and better well-being.
Quality of Life in Participants Receiving the GGTA1 KO Thymokidney by Patient Global Impression of Change (PGI-C)	At 24 weeks post transplant	PGI-C at 24 weeks post transplant. The PGI-C assesses a patient's perception of improvement or worsening over time. The minimum score (best outcome) is 1 ("Very Much Improved") and the maximum score (worst outcome) is 7 ("Very Much Worse"). Higher scores indicate a worse health outcome (greater worsening).
Incidence of Treatment-Emergent Adverse Events (Safety of the GGTA1 KO Thymokidney)	Baseline until last visit, assessed at least every 24 weeks after transplantation while the participant is alive and the thymokidney is functional, up to 50 years, or for 1 year after nephrectomy if required	Incidence of adverse events and serious adverse events; all-cause mortality.
Incidence of Proteinuria	Day 0 (day of xenotransplantation) until last visit, assessed at least every 24 weeks after transplantation while the participant is alive and the thymokidney is functional, up to 50 years, or for 1 year after nephrectomy if required	Incidence of proteinuria from Day 0.
Incidence of Zoonotic Infection	Day 0 (day of xenotransplantation) until last visit, assessed at least every 24 weeks after transplantation while the participant is alive and the thymokidney is functional, up to 50 years, or for 1 year after nephrectomy if required	Incidence of zoonotic infection from Day 0.
Incidence of Opportunistic Infection	Day 0 (day of xenotransplantation) until last visit, assessed at least every 24 weeks after transplantation while the participant is alive and the thymokidney is functional, up to 50 years, or for 1 year after nephrectomy if required	Incidence of opportunistic infection from Day 0.

Trial Locations

Locations (1)

New York University Langone Health; 🇺🇸 New York, New York, United States