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

NCT06878560 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: GPK-KF-101
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 2

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.

Conditions

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

Keywords

10 GE Xenokidney; End-stage renal disease; ESRD; Xenotransplantation

Outcome Measures

Primary Outcomes (15)

• Survival Rate of Patients with ESRD Receiving the 10 GE Xenokidney at 24 Weeks Post Transplant

  Participant survival rate at 24 weeks post transplant.

  Day 0 (day of xenotransplantation) to 24 weeks post transplant

• Survival Rate of the 10 GE Xenokidney at 24 Weeks Post Transplant

  10 GE Xenokidney survival rate at 24 weeks post transplant. 10 GE Xenokidney failure is defined as 10 GE Xenokidney nephrectomy.

  Day 0 (day of xenotransplantation) to 24 weeks post transplant

• Survival Time of Participants Receiving the 10 GE Xenokidney

  Participant survival post transplant. Participant survival is defined as time from xenotransplantation to death for any cause

  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

• Survival Time of the 10 GE Xenokidney (Overall Survival)

  Overall survival of the 10 GE Xenokidney post transplant. Overall survival time of the 10 GE Xenokidney is defined as time from xenotransplantation to 10 GE Xenokidney nephrectomy or death, whichever occurs first.

  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

• Survival Time of the 10 GE Xenokidney (Death-censored Survival)

  Death-censored survival 10 GE Xenokidney. Death-censored survival time of the 10 GE Xenokidney is defined as time from xenotransplantation to 10 GE Xenokidney nephrectomy censored for death.

  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 Function Post Transplant (Endogenous GFR)

  Endogenous measured GFR (24-hour urine creatinine clearance) at 24 weeks post transplant.

  At 24 weeks post transplant

• 10 GE Xenokidney Function Post Transplant (Exogenous GFR)

  Exogenous measured GFR (nuclear medicine GFR) at 24 weeks post transplant.

  At 24 weeks post transplant

• Quality of Life in Participants Receiving the 10 GE Xenokidney by EuroQol 5-Dimension 5-Level (EQ-5D-5L)

  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).

  Baseline to 24 weeks post transplant

• Quality of Life in Participants Receiving the 10 GE Xenokidney by Standardized Outcomes in Nephrology Life Participant (SONG-LP)

  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).

  Baseline to 24 weeks post transplant

• Quality of Life in Participants Receiving the 10 GE Xenokidney by Kidney Transplant Questionnaire (KTQ)

  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.

  Baseline to 24 weeks post transplant

• Quality of Life in Participants Receiving the 10 GE Xenokidney by Patient Global Impression of Change (PGI-C)

  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).

  At 24 weeks post transplant

• Incidence of Treatment-Emergent Adverse Events (Safety of the 10 GE Xenokidney)

  Incidence of adverse events and serious adverse events; all-cause mortality.

  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 Proteinuria

  Incidence of proteinuria from Day 0.

  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

  Incidence of zoonotic infection from Day 0.

  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

  Incidence of opportunistic infection from Day 0.

  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

Study Arms (1)

10 GE Xenokidney

EXPERIMENTAL

Participants will receive 10 GE Xenokidney

Biological: 10 GE Xenokidney

Interventions

10 GE Xenokidney BIOLOGICAL

Porcine-derived kidney containing intentional genomic alterations for xenotransplantation

Also known as: Xenokidney, UKidney

10 GE Xenokidney

Eligibility Criteria

• Age: 40 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Provide voluntarily informed consent to participate in the study and for lifetime follow up.
• Have a diagnosis of ESRD at the time of informed consent.
• Hemodialysis dependent for a minimum of 6 months and has a functioning arterial venous fistula/graft or permanent catheter at the time of informed consent.
• to 70 years of age at the time of informed consent, or 40 to \<50 years of a age with a calculated panel reactive antibody (cPRA) of ≥99.9%.
• Live withing 3 hours travel time of the xenotransplant center.
• Female participants must be postmenopausal or permanently sterilized (eg, hysterectomy, bilateral oophorectomy, or bilateral salpingectomy). Male participants must agree to the use of a highly effective method of birth control, if the possibility of conception exists.
• Negative xeno-crossmatch at Screening and pre-transplant.
• Estimated Post Transplant Survival Calculator score \>20% (https://optn.transplant.hrsa.gov/data/allocation-calculators/epts-calculator/).
• Body mass index ≤35 kg/m2.
• Have completed or have initiated and plan to complete (meningococcal A, C, W, Y and meningococcal B vaccine series only) Centers for Disease Control and Prevention recommended courses of age and risk factor appropriate vaccinations.
• Seropositive (immunoglobulin G) for cytomegalovirus and Epstein-Barr virus.
• \. Ineligible for conventional allogeneic kidney transplantation due to medical reason(s) for any of the following:
• Ineligible for a living donor transplant.
• Ineligible for an OPTN kidney transplant waitlist (reason for ineligibility will be collected).
• Delisted from OPTN kidney transplant waitlist (reason for delisting will be collected).

You may not qualify if:

• Need for multiple organ transplants.
• Severe medical co-morbidities including, but not limited to:
• Chronic liver disease.
• Advanced cardiovascular disease.
• Severe peripheral vascular disease that limits technical ability to transplant the 10 GE Xenokidney.
• Severe neurologic diseases or conditions that would preclude meaningful recovery or informed consent.
• Oral steroid-dependent airway disorder or chronic pulmonary disease or requires chronic, intermittent or continuous supplemental oxygen.
• Pulmonary hypertension.
• Uncontrolled diabetes or sequelae of diabetes mellitus including severe non-proliferative diabetic retinopathy.
• Severe neurogenic bladder that requires intermittent catheterization.
• ESRD due to hereditary or structural kidney disease.
• Active or recently treated malignancy at the time of informed consent.
• Non-renal cause of hematological disorders associated with anemia (eg, thalassemia and sickle disease).
• Cannot discontinue chronic anticoagulation therapy (low-dose daily aspirin is permissible).
• History of major psychiatric disorders with a psychiatric hospitalization and/or suicidal ideation within 5 years of informed consent.

Sponsors & Collaborators

• United Therapeutics: lead

Study Sites (2)

Northwestern Memorial Hospital

Chicago, Illinois, 60611, United States

RECRUITING

New York University Langone Health

New York, New York, 10016, United States

RECRUITING

MeSH Terms

Conditions

Renal Insufficiency, Chronic; Kidney Failure, Chronic

Condition Hierarchy (Ancestors)

Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

United Therapeutics Global Medical Information

CONTACT

919-485-8350; clinicaltrials@unither.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This single group study will enroll participants simultaneously from either of two patient populations: (1) participants that are ineligible for conventional allogeneic kidney transplantation due to medical reason or (2) participants on an Organ Procurement and Transplantation Network kidney transplant waitlist that are more likely to die or go untransplanted within 5 years than receive a kidney transplant.

Study Record Dates

• First Submitted: March 3, 2025
• First Posted: March 17, 2025
• Study Start: October 29, 2025
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): October 1, 2075
• Last Updated: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)