EXTEND: A Prospective Study to Evaluate the Safety and Efficacy of GGTA1 KO Thymokidney XenoTransplantation in Patients With End-stage Renal Disease (ESRD)
United Therapeutics
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.
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 limite…
Eligibility
- Age range
- 40–70 years
- Sex
- All
- Healthy volunteers
- No
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 t…
Interventions
- BiologicalGGTA1 KO Thymokidney
Porcine kidney containing an intentional genomic alteration and thymic tissue autograft for xenotransplantation
Location
- New York University Langone HealthNew York, New York