United Therapeutics Corporation Announces FDA Clearance to Proceed with UHeart Xenotransplantation Clinical Trial - Seite 2

Safety and efficacy data will be reviewed frequently by an independent Data Monitoring Committee. After at least 12 weeks post-transplant of the first participant, United Therapeutics will provide data to FDA prior to initiating a second transplant. If safety and efficacy results from the first 2 participants are supportive, the study sample size will be increased to enable the study to support registration.

Efficacy Endpoints

Efficacy endpoints include participant survival rate, UHeart survival rate, UHeart function¹, change in quality of life in participants² at 24 weeks post-transplant, and participant exercise capacity³. Overall survival time of participants receiving a UHeart and overall survival time of the UHearts themselves are also efficacy endpoints.

Safety Endpoints

Safety endpoints include the incidence of adverse events and serious adverse events, all-cause mortality, and the incidence of arrythmias, thromboembolic and ischemic strokes, zoonotic infections, and opportunistic infections.

Key Participation Criteria

Key participation criteria include those ≥50 years of age, diagnosed with end-stage or advanced heart failure (HF) classified as American College of Cardiology/American Heart Association stage D and New York Heart Association Class IV, and no remaining therapeutic options. Participants will be screened using a crossmatch assay to assess expected immunological compatibility with the UHeart. Participants must not need multiple organ transplants; must not have had a prior solid organ transplant; must not have support with venoarterial ECMO; must not have severe medical co-morbidities, including but not limited to chronic liver disease, severe central vascular disease, severe neurologic diseases, 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 will be provided in a future listing on the clinicaltrials.gov website.

About Advanced Heart Failure

According to the Journal of Cardiac Failure, in the United States nearly 6.7 million adults 20 years of age or older have HF, and in 2023, HF was responsible for 14.6% of all causes of death according to a CDC report⁴. In 2023, only a fraction of Americans with HF were considered candidates and waitlisted for a human heart transplant (0.12% or 8,000 Americans), and only approximately 4,000 heart transplants were performed⁵.

Schreibe Deinen Kommentar