United Therapeutics Corporation announced the first clinical xenotransplantation in its EXPAND study of the UKidney?? in patients with end-stage renal disease (ESRD). The successful transplant operation was performed at NYU Langone Health.

This achievement marks a transformative moment in transplant medicine. The EXPAND study offers new hope to the thousand s of Americans currently on the kidney transplant waiting list, many of whom may not survive long enough to receive a human organ. EXPAND (NCT06878560) is a multicenter, open-label, safety and efficacy study and is intended to support a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA).

The study is designed as a combination phase 1/2/3 trial (sometimes referred to as a "phaseless" study) to evaluate safety and efficacy seamlessly without moving through separate phase 1, phase 2, and phase 3 studies that are typically associated with conventional drug approvals. Study participants will receive a UKidney transplant followed by a 24-week post-transplant follow-up period, including the evaluation of all study endpoints and safety assessments. There will be a 12-week waiting period between the first and second transplants.

After the initial cohort reaches at least 12 weeks post-transplant, safety and efficacy data will be reviewed by an Independent Data Monitoring Committee to determine if the study should proceed to the next cohort. After reviewing the data from the first six transplants with the FDA, United Therapeutics plans to amend the study protocol to increase the study sample size to support a BLA and include additional transplant centers. Additional Key Participation Criteria: Additional key participation criteria include an age of 55 to 70 years old, a diagnosis of ESRD, and at least six months on hemodialysis.

Participants will be screened using a crossmatch assay to assess expected immunological compatibility with the UKidney. Participants must not need multiple organ transplants; must not have severe medical co-morbidities, including but not limited to advanced cardiovascular disease, severe peripheral vascular disease, severe neurological disease, chronic pulmonary disease, and uncontrolled diabetes; and must not have a history of medical noncompliance that may preclude adherence to the demands and requirements of xenotransplantation. Full inclusion and exclusion criteria for this study are available on the clinicaltrials.gov website.