Precision Guided Tacrolimus Dosing in Pediatric Heart Transplant
-
- STATUS
- Recruiting
-
- End date
- Apr 30, 2025
-
- participants needed
- 16
-
- sponsor
- University of Utah
Summary
Immunosuppressive therapy is required to prevent organ rejection, however, dosing of immunosuppressive agents is complicated by patient-specific differences impacting the body's absorption and elimination of these agents. The goal of this research proposal is to clinically validate an innovative precision medicine strategy for dosing the immunosuppressant tacrolimus in pediatric heart transplant, which will in turn lead to improvements in long-term transplant survival outcomes. The strategy and techniques used in this project can be extended to improve drug therapy across multiple pediatric diseases requiring chronic therapy.
Description
Of the more than 400 children annually receiving a heart transplant, the likelihood that the child dies within five years (25%) is equivalent to the potential that the child lives >20 years post-transplant. The success of the transplant, and thus long-term survival of the child, is well known to correlate with achieving adequate immunosuppression to prevent rejection, especially in the period immediately following transplant. Attaining adequate immunosuppression is complicated by several factors, many of which impact the pharmacokinetics (PK) of the immunosuppressive agent. These factors include age of the patient, renal and hepatic function, concomitant medications, patient genetics, and the time post-transplant. Frequent, invasive drug monitoring is used clinically to assess how these factors impact an individual child's PK, and in turn the dose required to achieve and maintain adequate immunosuppression. However, this empirical "guess and check" approach often results in the child spending substantial time receiving immunosuppressive therapy that is either ineffective or unsafe, decreasing the longevity of the transplant organ and thus, long-term patient survival. A more desirable approach would be to guide dosing using a precision medicine approach, wherein patient specific factors are used a priori to predict the dose most likely to achieve adequate immunosuppression. Developing a precision medicine platform for immunosuppression following pediatric heart transplant has the potential to not only improve outcomes in heart transplant recipients, but across all transplant types. This proposal aims to: 1) determine the impact of CYP3A5 genetic variation on tacrolimus concentrations in children, 2) prospectively validate the clinical utility of a PK-guided precision medicine platform in pediatric heart transplant patients receiving tacrolimus-based immunosuppression; and 3) identify and correlate biomarkers of transplant outcomes and tacrolimus safety/effect with the PK of tacrolimus, in order to establish the tacrolimus concentrations required for safe and effective immunosuppression in pediatric heart transplant recipients. The overall objective of this study is to improve the long-term survival of children with heart transplant via optimization of the child's immunosuppressive therapy. Importantly, the interdisciplinary nature of this mentored research proposal will facilitate my progress towards an independent career developing precision medicine tools to improve health outcomes in children.
Details
| Condition | Heart Transplant Failure and Rejection |
|---|---|
| Age | 1years - 18years |
| Treatment | Tacrolimus Dosing Support Tool |
| Clinical Study Identifier | NCT04380311 |
| Sponsor | University of Utah |
| Last Modified on | 19 February 2024 |
How to participate?
,
You have contacted , on
Your message has been sent to the study team at ,
What happens next?
- You can expect the study team to contact you via email or phone in the next few days.
- Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.
You are contacting
Primary Contact
Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
Learn moreIf you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
Learn moreComplete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.
Learn moreSimilar trials to consider
Not finding what you're looking for?
Sign up as a volunteer to stay informed
Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.
Sign up as volunteerStudy AnnotationsStudy Notes
Notes added here are public and can be viewed by anyone. Notes added here are only available to you and those who you share with.
Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!
No annotations made yet
Add a private note
- Select a piece of text from the left.
- Add notes visible only to you.
- Send it to people through a passcode protected link.
Study Definition
WikipediaAdd a private note
- Select a piece of text.
- Add notes visible only to you.
- Send it to people through a passcode protected link.