Partial Heart Transplantation for Severe Pediatric Semilunar Valve Dysfunction (NCT05372757) | Clinical Trial Compass
CompletedNot Applicable
Partial Heart Transplantation for Severe Pediatric Semilunar Valve Dysfunction
United States5 participantsStarted 2023-05-03
Plain-language summary
The long-term goal of this research initiative is to develop a new valve replacement option for neonates, infants and young children. The central hypothesis is that transplantation of a freshly isolated heart valve will be associated with superior outcomes compared to currently available options, including preserved cadaver valves, bioprosthetic tissue valves, or mechanical valves. This new operation has been named "partial heart transplantation".
The proposed study is a single-center, nonrandomized single arm pilot trial of "partial heart transplantation" in neonates, infants and young children who require semilunar heart valve replacement. This "first in man" trial seeks to determine whether valve replacement using partial heart transplant is feasible and safe. Primary aims are survival one year and five years following the procedure. The hypothesis is that, when compared to historical controls who have undergone homograft valve replacement, those undergoing partial heart transplantation will have equal or superior survival one year and five years following the procedure. Secondary aims are to assess growth and function of the transplanted valve. The hypothesis is that when compared to historical controls who have undergone conventional valve replacement, those undergoing partial heart transplantation will have valve growth that corresponds with somatic growth and superior valve function 1 year following the procedure. Up to five patients will be enrolled in this trial over three years.
Who can participate
Age range
1 Day – 2 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Children less than 2 years of age who are referred for a cardiac operation that involves a primary semilunar valve replacement or children less than 2 years of age who are referred for a cardiac operation involves an initial replacement of a previously placed prior homograft, bioprosthetic, or mechanical valve in the aortic or pulmonary position.
* Deemed acceptable for partial heart transplantation based on the standard evaluation process used for orthotopic heart transplantation (see Appendix 1) Insurance approval.
* Written informed consent of both parents/guardians; if there is only one parent/guardian, consent from that individual will be adequate.
Exclusion Criteria:
* Absolute contraindications for orthotopic heart transplantation.
* Severe bilateral long segment pulmonary arterial hypoplasia
* Bilateral pulmonary vein stenosis
* \<34 weeks corrected gestational age
* Persistent acidosis with a pH \< 7.1
* Diagnosis of immune deficiency.
* Inability for the parent to understand English or Spanish.
* Failure to pass the following psychosocial evaluation:
* The candidate should reside within 4 hours traveling time from Medical University of South Carolina for a minimum of four to six months post-transplantation to assure careful follow-up
* The candidate's family should be capable of long-term supportive care of the child and be able to support the medical needs of the child in follow-up
* Parental (custodial) alcohol and/or substance abuse
* Doc…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The ability to perform the new operation in at least 1 patient per year for 3 years (averaged) after enrollment opens.
Timeframe: Duration of the study (3 years after enrollment opens).
2
Number of participants who achieve survival.
Timeframe: 6 months after all 5 subjects have been enrolled.
3
Number of participants who achieve survival.
Timeframe: 1 year after all 5 subjects have been enrolled.