NCT07224763 Study to Evaluate the Safety and Efficacy of the GGTA1 KO Thymokidney in Patients With ESRD
| NCT ID | NCT07224763 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | United Therapeutics |
| Condition | ESRD (End-Stage Renal Disease) |
| Study Type | INTERVENTIONAL |
| Enrollment | 50 participants |
| Start Date | 2026-03 |
| Primary Completion | 2029-03 |
Trial Parameters
Eligibility Fast-Check
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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.
Eligibility Criteria
Inclusion Criteria for all Participants (Groups 1 and 2): 1. Provide voluntarily informed consent to participate in the study and for lifetime follow-up. 2. Have a diagnosis of ESRD at the time of informed consent. 3. 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. 4. 50 to 70 years of age at the time of informed consent, or 40 to \<50 years of age with a calculated panel reactive antibody (cPRA) of ≥99.9%. 5. Evidence of thymic involution on chest computed tomography (CT) scan with a thymic region of interest score of ≤1. 6. Live within 3 hours travel time of the xenotransplant center. 7. 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. 8. Negative xeno-crossmatc