Study to Evaluate the Safety and Efficacy of the 10 GE Xenokidney in Patients with ESRD

Phase 1

Not yet recruiting

• Conditions: ESRD (End-Stage Renal Disease); Kidney Transplantation; Xenotransplantation
• Interventions: Biological: 10 GE Xenokidney

• Registration Number: NCT06878560
• Lead Sponsor: United Therapeutics

Brief Summary

The purpose of this study is to evaluate the safety and efficacy of the 10 GE Xenokidney in patients with 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, 10 GE Xenokidney survival, and screening for zoonotic infections. Part B will continue for the lifetime of the participant.

Detailed Description

This is a Phase 1/2/3, multicenter, open-label, safety and efficacy study of the 10 GE Xenokidney in patients with ESRD. The study will be comprised of the following:

* A Screening Period up to 52 weeks.

* Part A consists of the 10 GE Xenokidney 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 10 GE Xenokidney, including but not limited to documentation of participant survival, 10 GE Xenokidney 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 are more likely to die or go untransplanted within 5 years than receive a kidney transplant.

Study Timeline

• Status Verified: 2025-03
• Study First Submitted: 2025-03-03
• Study First Submitted QC: 2025-03-12
• Last Update Submitted: 2025-03-12
• Study First Posted: 2025-03-17
• Last Update Posted: 2025-03-17
• Study Start: 2025-06
• Primary Completion: 2028-06-01
• Study Completion: 2028-06-01

Recruitment & Eligibility

• Status: NOT_YET_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
10 GE Xenokidney	10 GE Xenokidney	Participants will receive 10 GE Xenokidney

Primary Outcome Measures

Name	Time	Method
Survival Rate of Patients with ESRD Receiving the 10 GE Xenokidney 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 10 GE Xenokidney at 24 Weeks Post Transplant	Day 0 (day of xenotransplantation) to 24 weeks post transplant	10 GE Xenokidney survival rate at 24 weeks post transplant. 10 GE Xenokidney survival is defined as no requirement for chronic dialysis (3 times per week) or nephrectomy.
Survival Time of Participants Receiving the 10 GE Xenokidney	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
Quality of Life in Participants Receiving the 10 GE Xenokidney 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 10 GE Xenokidney)	Baseline until last visit, assessed at least every 24 weeks after transplantation while the participant is alive and the xenokidney 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.
Survival Time of the 10 GE Xenokidney	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 xenokidney is functional, up to 50 years	10 GE Xenokidney survival post transplant. Survival time of the 10 GE Xenokidney is defined as time from xenotransplantation to the start of chronic dialysis (3 times per week), nephrectomy, or death, whichever occurs first
10 GE Xenokidney Function Post Transplant	Baseline to 24 weeks post transplant	Change in measured glomerular filtration rate (24 hour creatinine clearance) from baseline to 24 weeks post transplant.
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 xenokidney is functional, up to 50 years, or for 1 year after nephrectomy if required	Incidence of proteinuria from Day 0.
Quality of Life in Participants Receiving the 10 GE Xenokidney 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 10 GE Xenokidney 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 10 GE Xenokidney 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.
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 xenokidney 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 xenokidney is functional, up to 50 years, or for 1 year after nephrectomy if required	Incidence of opportunistic infection from Day 0.