Transplanting heart valves from donors into patients who need them

Allogeneic Valve Transplantation

This study is testing if heart valves from donors can help kids and young adults who need valve replacements by providing a solution that grows with them and reduces the need for more surgeries later on.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 4 (estimated)
• Ages: 30 Days to 50 Years
• Sex: All
• Sponsor: Boston Children's Hospital (Academic / other)
• Locations: 1 site (Boston, Massachusetts)
• Trial ID: NCT06387446 on ClinicalTrials.gov

What this trial studies

This study involves the transplantation of heart valves from organ donors into pediatric and young adult patients who require valve replacement. The focus is on patients whose heart valves may not grow with them, offering a potential solution that could reduce the need for multiple surgeries over their lifetime. The trial will evaluate the outcomes of these valve transplants, including any side effects experienced by the patients. The goal is to provide a durable valve option that can accommodate growth in younger patients.

Who should consider this trial

Good fit: Ideal candidates include pediatric patients aged 30 days to less than 18 years and adult patients aged 18 to 50 years who require aortic, pulmonary, mitral, or tricuspid valve replacement.

Not a fit: Patients under 30 days old or those with irreversible multisystem organ failure or progressive systemic diseases may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could provide a long-lasting heart valve solution for children and young adults, minimizing the need for repeated surgeries.

How similar studies have performed: While this approach is innovative, similar studies involving valve transplantation have shown promise, suggesting potential for success.

Eligibility criteria

Inclusion Criteria

* Patients requiring aortic, pulmonary, mitral or tricuspid valve replacement.
* Pediatric patients 30 days or older and \< 18 years, and adult patients 18-50 yrs.

  o Particularly patients with significant growth potential
* Insufficient options are available for valve replacement

  o Patients at risk of immunogenic bio-prosthetic valve failure
* Discussion with patient /family - option for durable valve rather than traditional prosthesis
* ABO compatible
* Patient and family able to travel to BCH within 48 hour time frame or within our organ procurement organization
* Patients that are able to maintain follow-up at BCH during the duration of the study
* Patients that are able to provide medical record authorization for 5 year follow-up

Exclusion Criteria

* Age \< 30 days
* Irreversible multisystem organ failure; or additive effects of the multiple systems affected making transplant survival unlikely
* Progressive systemic disease with early mortality (genetic/metabolic, idiopathic, syndromic)
* Morbid obesity (BMI\>35)
* Diabetes mellitus with evidence of end-organ damage
* Severe chromosomal, neurologic or syndromic abnormalities
* Immunocompromised condition (DiGeorge, SCID etc.)
* Active infection
* History of endocarditis
* HIV or chronic hepatitis B or C infection
* Malignancy within 5 years prior to transplant
* Severe renal or liver failure
* Inadequate social support for post-transplant management
* Recent history of illicit drug, tobacco or alcohol abuse despite trials/assistance to stop behavior
* Evidence of large stroke with high risk for hemorrhagic conversion
* Heart transplant recipient
* Patients that are unable to follow-up during the initial 6 month window
* Patients that are unable to provide medical record authorization for 5 years

Where this trial is running

Boston, Massachusetts

• Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)

Study contacts

• Principal investigator: Sitaram Emani, MD — Boston Children's Hospital
• Study coordinator: Sitaram Emani, MD

Conditions Valve Heart Disease; Valve Disease, Heart; Valve Disease, Aortic