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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Partial Heart Transplantation Arm | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Partial Heart Transplantation | Procedure | The donor heart will be recovered and the new heart valve will be removed from this heart by the clinical team. The child will be scheduled for an emergent operation once the donor heart valve is deemed acceptable. The child will have general anesthesia during surgery. This is a state of unconsciousness, which is carefully controlled by the anesthesiologist with a mixture of very potent drugs, to prevent or lessen pain. The child will have a partial heart transplant using the donated heart valve to replace the dysfunctional heart valve. Expected hospital stay will range from weeks to months. The medical care after surgery is performed by the child's clinical team. This type of re-operation could similarly be required after a standard heart valve replacement. |
| Measure | Description | Time Frame |
|---|---|---|
| The ability to perform the new operation in at least 1 patient per year for 3 years (averaged) after enrollment opens. | This is how feasibility will be measured. This will be answered as "yes" or "no". | Duration of the study (3 years after enrollment opens). |
| Number of participants who achieve survival. | Safety will primarily be defined as patient survival or re-operation or transcatheter reintervention on the transplanted valve at 6-month intervals, starting 6 months after the first subject undergoes a valve transplant, and continuing until 1 year after all 5 subjected have been enrolled. | 6 months after all 5 subjects have been enrolled. |
| Number of participants who achieve survival. | Safety will primarily be defined as patient survival or re-operation or transcatheter reintervention on the transplanted valve at 6-month intervals, starting 6 months after the first subject undergoes a valve transplant, and continuing until 1 year after all 5 subjected have been enrolled. | 1 year after all 5 subjects have been enrolled. |
| Measure | Description | Time Frame |
|---|---|---|
| Valve annulus growth. | The valve annulus diameter will be measured using echocardiography in 2 planes at baseline (i.e., during postoperative hospitalization 5-10 days after surgery) and every 6 months following the operation. It is expected that the valve annulus diameter Z score will be relatively unchanged over time, which would reflect an absolute increase in valve annulus growth corresponding with an increase in body surface area. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Taufiek Rajab, MD | Medical University of South Carolina | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Medical University of South Carolina | Charleston | South Carolina | 29401 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36749770 | Derived | Rajab TK, Ochoa B, Zilinskas K, Kwon J, Taylor CL, Henderson HT, Savage AJ, Kavarana M, Turek JW, Costello JM. Partial heart transplantation for pediatric heart valve dysfunction: A clinical trial protocol. PLoS One. 2023 Feb 7;18(2):e0280163. doi: 10.1371/journal.pone.0280163. eCollection 2023. |
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| Every 6 months following the operation until child is old enough (18+ years) for mechanical prosthesis or date of death, whichever comes first. |
| The level of valve stenosis. | Mean valve stenosis will be assessed by transthoracic echocardiogram using pulse Doppler every 6 months following the operation. | Every 6 months following the operation until child is old enough (18+ years) for mechanical prosthesis or date of death, whichever comes first. |
| The level of valve regurgitation. | Mean valve regurgitation will be assessed with color Doppler echocardiography. | Every 6 months following the operation until child is old enough (18+ years) for mechanical prosthesis or date of death, whichever comes first. |