Actively Recruiting
Study to Evaluate the Safety and Efficacy of the GGTA1 KO Thymokidney in Patients With ESRD
Led by United Therapeutics · Updated on 2025-12-02
50
Participants Needed
1
Research Sites
2609 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Study to Evaluate the Safety and Efficacy of the GGTA1 KO Thymokidney in Patients With ESRD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Provide voluntary informed consent for participation and lifetime follow-up
- Diagnosed with end-stage renal disease (ESRD) at consent
- Hemodialysis dependent for at least 6 months with a functioning arterial-venous fistula, graft, or permanent catheter
- Age 50 to 70 years, or age 40 to under 50 years with calculated panel reactive antibody (cPRA) ≥99.9%
- Evidence of thymic involution on chest CT scan with thymic region of interest score ≤1
- Live within 3 hours travel time of the xenotransplant center
- Female participants must be postmenopausal or permanently sterilized; males must agree to use effective birth control if conception is possible
- Negative xeno-crossmatch at screening and pre-transplant
- Estimated Post Transplant Survival Calculator score >20%
- Body mass index ≤35 kg/m2
- Completed or initiated age- and risk-appropriate meningococcal vaccinations
- Seropositive for cytomegalovirus and Epstein-Barr virus
- Group 1: Ineligible for conventional kidney transplant due to medical reasons (including ineligibility for living donor transplant, OPTN waitlist, or delisting)
- Group 2: On OPTN kidney transplant waitlist with no approved living donors and higher likelihood of death or no transplant within 5 years than receiving a transplant
You will not qualify if you...
- Need for multiple organ transplants
- Severe medical co-morbidities including chronic liver disease, advanced cardiovascular disease, severe peripheral vascular disease limiting transplant, severe neurologic diseases, steroid-dependent airway disorders, chronic pulmonary disease, pulmonary hypertension, uncontrolled diabetes or severe diabetic complications, severe neurogenic bladder requiring catheterization
- ESRD due to hereditary or structural kidney disease
- Active or recently treated malignancy
- Non-renal hematological disorders causing anemia such as thalassemia or sickle cell disease
- Inability to discontinue chronic anticoagulation therapy (except low-dose aspirin)
- History of major psychiatric disorders with hospitalization or suicidal ideation within 5 years
- Active tuberculosis treatment, prophylaxis, or positive TB test during screening
- Positive tests for hepatitis B or C, low hepatitis B surface antibody titer unless nonresponder to vaccine, or positive for HIV
- Inability to perform activities of daily life independently
- History of medical noncompliance that could affect adherence to study demands, including recent substance use disorder, lack of social support, or untreated psychological conditions
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
New York University Langone Health
New York, New York, United States, 10016
Actively Recruiting
Research Team
U
United Therapeutics Global Medical Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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