Partial Heart Transplantation for Severe Pediatric Semilunar Valve Dysfunction

NCT ID: NCT05372757

Last Updated: 2024-09-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-05-03
• Study Completion Date: 2023-11-30

Brief 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.

Conditions

Semilunar Heart Valve Replacement

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Partial Heart Transplantation Arm

Group Type: EXPERIMENTAL

Partial Heart Transplantation

Intervention Type: 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.

Interventions

Partial Heart Transplantation

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.

Intervention Type: PROCEDURE

Eligibility Criteria

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
• Documented parental (custodial) child abuse or neglect
• Parent (custodian) with cognitive/psychiatric impairment severe enough to limit comprehension of medical regimen
• Evidence of sepsis
• Hepatitis B surface antigenemia
• HIV positivity

• Minimum Eligible Age: 1 Day
• Maximum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Medical University of South Carolina

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Taufiek Rajab, MD

Role: PRINCIPAL_INVESTIGATOR

Medical University of South Carolina

Locations

Medical University of South Carolina

Charleston, South Carolina, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 36749770 (View on PubMed)

Other Identifiers

00114653

Identifier Type: -

Identifier Source: org_study_id