Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
GGTA1 KO Thymokidney · 1 trial · 3 indications
Participant survival rate 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.
Participant survival post transplant. Participant survival is defined as time from xenotransplantation to death for any cause.
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.
Death-censored survival GGTA1 KO Thymokidney. Death-censored survival time of the GGTA1 KO Thymokidney is defined as time from xenotransplantation to GGTA1 KO Thymokidney nephrectomy censored for death.
Endogenous measured GFR (24-hour urine creatinine clearance) at 24 weeks post transplant.
Exogenous measured GFR (nuclear medicine GFR) at 24 weeks post transplant.
Proliferative responsiveness to source GGTA1 KO Pig versus third-party pig as measured by mixed lymphocyte reaction from baseline to 24 weeks post transplant.
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).
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).
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.
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).
Incidence of adverse events and serious adverse events; all-cause mortality.
Incidence of proteinuria from Day 0.
Incidence of zoonotic infection from Day 0.
Incidence of opportunistic infection from Day 0.
| Arm | Type | Description |
|---|---|---|
| GGTA1 KO Thymokidney | EXPERIMENTAL | Participants will receive GGTA1 KO Thymokidney |
| Name | Type | Description |
|---|---|---|
| GGTA1 KO Thymokidney | BIOLOGICAL | Porcine kidney containing an intentional genomic alteration and thymic tissue autograft for xenotransplantation |
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-v...
GGTA1 KO Thymokidney is an investigational xenotransplantation product being studied for patients with end-stage renal disease (ESRD). It is a genetically modified thymokidney designed to be transplanted into patients with ESRD as a potential alternative to standard kidney transplantation. The product is currently in Phase 1 clinical development.
GGTA1 KO Thymokidney is being developed by United Therapeutics Corporation, a biotechnology company traded on the NASDAQ under the ticker symbol UTHR. The company is conducting a Phase 1 clinical trial to evaluate the safety and efficacy of this investigational xenotransplantation product in patients with end-stage renal disease.
GGTA1 KO Thymokidney is in Phase 1 clinical development. The product is investigational and has not been approved by the FDA. A Phase 1 trial is currently recruiting participants in the United States to assess the safety and efficacy of the thymokidney in patients with end-stage renal disease.
GGTA1 KO Thymokidney is being evaluated in a Phase 1 clinical trial with the identifier NCT07224763. This trial is titled "Study to Evaluate the Safety and Efficacy of the GGTA1 KO Thymokidney in Patients With ESRD" and is currently recruiting participants in the United States. The study is an uncontrolled, open-label trial with a target enrollment of 50 patients aged 40 years and older.
GGTA1 KO Thymokidney is not FDA approved. It is an investigational product currently in Phase 1 clinical development. The ongoing trial, NCT07224763, is designed to evaluate the safety and efficacy of the thymokidney in patients with end-stage renal disease, but the product has not yet received regulatory approval for commercial use.