Study to Evaluate the Safety and Efficacy of the GGTA1 KO Thymokidney in Patients With ESRD
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age40-70
SponsorUnited Therapeutics
About this trial
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
This trial does not accept healthy volunteersQualifiers
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.
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%.
Disqualifiers
Need for multiple organ transplants.
Chronic liver disease.
Advanced cardiovascular disease.
Severe peripheral vascular disease that limits technical ability to transplant the GGTA1 KO Thymokidney.
Trial design
Design model
Single group
Treatments tested in this trial
GGTA1 KO Thymokidney
Biological/VaccinePorcine kidney containing an intentional genomic alteration and thymic tissue autograft for xenotransplantation
Treatment groups
50 Participants
are divided into 1 treatment groupGroup A: GGTA1 KO ThymokidneyExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Survival Rate of Patients with ESRD Receiving the GGTA1 KO Thymokidney at 24 Weeks Post Transplant
Participant survival rate at 24 weeks post transplant.
Time frame
Day 0 (day of xenotransplantation) to 24 weeks post transplant2
Survival Rate of the GGTA1 KO Thymokidney at 24 Weeks Post Transplant
GGTA1 KO Thymokidney survival rate at 24 weeks post transplant. GGTA1 KO Thymokidney failure is defined as GGTA1 KO Thymokidney nephrectomy.
Time frame
Day 0 (day of xenotransplantation) to 24 weeks post transplant3
Survival Time of Participants Receiving the GGTA1 KO Thymokidney
Participant survival post transplant. Participant survival is defined as time from xenotransplantation to death for any cause.
Time frame
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 years4
Survival Time of the GGTA1 KO Thymokidney (Overall Survival)
Overall survival of the GGTA1 KO Thymokidney post transplant. Overall survival time of the GGTA1 KO Thymokidney is defined as time from xenotransplantation to GGTA1 KO Thymokidney nephrectomy or death, whichever occurs first.
Time frame
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 yearsSponsors and contacts
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Source ClinicalTrials.gov