Xenokidney for Kidney Failure

(EXPAND Trial)

Overseen By United Therapeutics Global Medical Information

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: United Therapeutics

Must not be taking: Oral steroids, Anticoagulants

Disqualifiers: Chronic liver disease, Advanced cardiovascular, Uncontrolled diabetes, Active malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

50 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment for individuals with kidney failure who cannot receive a regular kidney transplant. The study evaluates the safety and effectiveness of the 10 GE Xenokidney, a specially developed kidney for transplantation. It targets those with end-stage kidney disease (severe kidney failure) who have been on dialysis for at least six months. Participants must live within three hours of the transplant center and have no other major organ issues. The trial will monitor the new kidney's function and safety throughout the patient's lifetime. As a Phase 1, Phase 2 trial, this research aims to understand how the treatment works in people and measure its effectiveness in an initial, smaller group, offering participants a chance to be among the first to benefit from this innovative approach.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you cannot discontinue chronic anticoagulation therapy (blood thinners).

What prior data suggests that the 10 GE Xenokidney is safe for patients with ESRD?

Research has shown that the 10 GE Xenokidney has promising safety results from earlier studies. In tests with baboons, these pig kidneys functioned well and maintained the animals' health. This suggests potential safety, but animal results don't always predict human outcomes.

As this trial is in its early stages (Phase 1 and Phase 2), it marks the first time the 10 GE Xenokidney is being tested in humans. Researchers are closely monitoring for any side effects or safety issues. The primary goal is to ensure the treatment's safety for patients. Although human data is not yet available, the animal studies provide a hopeful starting point for its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about the 10 GE Xenokidney treatment for kidney failure because it involves transplanting genetically engineered pig kidneys into humans. Unlike current treatments, such as dialysis or human kidney transplants, the Xenokidney approach offers a potentially unlimited supply of organs, addressing the significant shortage of human donors. This treatment is unique because the pig kidneys are modified to reduce the risk of rejection, potentially leading to better long-term outcomes for patients.

What evidence suggests that the 10 GE Xenokidney might be an effective treatment for kidney failure?

Research has shown that the 10 GE Xenokidney could be a promising option for treating end-stage kidney disease. This trial will evaluate the 10 GE Xenokidney, which uses a specially modified pig kidney for transplant. Early results suggest it might help patients unable to receive a regular kidney transplant or those on long waitlists. The 10 GE Xenokidney is designed to lower the risk of rejection by the body. Although still in early stages, these studies offer hope that using animal organs for transplants might become a viable option for kidney failure. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

This trial is for adults aged 55-70 with end-stage renal disease (ESRD) who've been on hemodialysis for at least 6 months. They must be ineligible for standard kidney transplants or unlikely to receive one soon due to long waitlists. Participants need a functioning dialysis access, agree to birth control if necessary, and commit to lifetime follow-up after the transplant.

Inclusion Criteria

I agree to join the study and allow follow-ups for life.

I am up to date with all recommended vaccinations for my age and health condition.

My organ transplant test shows a low risk of rejection.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 52 weeks

Xenotransplantation

Participants receive the 10 GE Xenokidney transplantation

Day 0

Post-transplant Follow-up (Part A)

Evaluation of efficacy and safety objectives, including all study endpoints and safety assessments

24 weeks

Long-term Follow-up (Part B)

Evaluation of participant survival, 10 GE Xenokidney survival, and screening for zoonotic infections

Lifetime of the participant

What Are the Treatments Tested in This Trial?

Interventions

• 10 GE Xenokidney

Trial Overview The study tests the '10 GE Xenokidney,' an animal-to-human organ transplant in ESRD patients. It includes initial transplantation and a 24-week post-transplant period assessing safety/efficacy, followed by lifelong monitoring for survival rates, organ function, and potential zoonotic infections.

How Is the Trial Designed?

Group I: 10 GE Xenokidney Experimental Treatment 1 Intervention

Participants will receive 10 GE Xenokidney

Who Is Running the Clinical Trial?

United Therapeutics

Lead Sponsor

Trials

112

Recruited

14,500+

Dr. Martine Rothblatt

United Therapeutics

Chief Executive Officer since 1996

PhD in Medical Ethics from the Royal London College of Medicine and Dentistry, JD and MBA from UCLA

Dr. Michael Benkowitz

United Therapeutics

Chief Medical Officer since 2023

MD from Harvard Medical School

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06878560

NCT06878560 | Study to Evaluate the Safety and Efficacy ... ... efficacy study of the 10 GE Xenokidney in patients with ESRD. The study ... 10 GE Xenokidney in Patients With End-stage Renal Disease (ESRD). Conditions.

2. amjtransplant.org amjtransplant.org/article/S1600-6135(25)01023-8/fulltext

Study Design of the First-in-Human Clinical Trial of a 10 ... Methods: The GPK-KF-101 study (NCT TBD), “A Prospective Study to Evaluate the Safety and Efficacy of the 10 GE Xenokidney in Patients with End-stage Renal ...

3. clinicaltrials.med.nyu.edu clinicaltrials.med.nyu.edu/clinicaltrial/2806/prospective-study-evaluate-safety/

Clinical Research Studies - NYU A Prospective Study to Evaluate the Safety and Efficacy of the 10 GE Xenokidney in Patients with End-stage Renal Disease (ESRD) · Brief description of study.

4. trial.medpath.com trial.medpath.com/news/fdc402a8f00f01ef/united-therapeutics-achieves-first-clinical-xenotransplantation-with-genetically-modified-pig-kidney-in-expand-trial

United Therapeutics Achieves First Clinical ... - MedPath According to the American Kidney FundSearch company, approximately 815,000 patients currently live with kidney failureSearch disease, with more ...

5. withpower.com withpower.com/trial/phase-2-kidney-failure-chronic-5-2025-3a64f

Xenokidney for Kidney Failure (EXPAND Trial) ... efficacy for patients with Chronic Renal Failure, Chronic Kidney Disease, Kidney Failure, Animal-to-human organ transplant and Kidney Transplant. See if you ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11026402/

Consistent survival in consecutive cases of life-supporting ... 10GE porcine kidneys support life with stable renal function in baboons. ... Whereas in previous xenokidney transplants from multi-transgenic ...

7. ir.unither.com ir.unither.com/press-releases/2025/02-03-2025-120011819

United Therapeutics Corporation Announces FDA ... About End-Stage Renal Disease. According to the American Kidney Fund, there are approximately 808,000 patients with kidney failure in the ...