Optimal Pediatric Heart Transplant Immunosuppression With MicroRNAs (OPTIMA)

Inova Health Care Services

Status: Begins enrollment this month

Conditions: Cardiac Failure; Graft Rejection

Study type: Observational

Funder types: Other

Identifiers

NCT06532890

Enduring Hearts Awards#1248960 (Other Identifier)

INOVA-2024-197

Details and patient eligibility

About

This study aims to discover circulating microRNAs (associated with drug doses and levels) that can be used to characterize the overall immune state in pediatric heart transplant patients and predict patients that will go on to develop infection and rejection. MicroRNAs (miRs) are small, non-coding RNA molecules that regulate gene expression and serve as molecular biomarkers found in the circulation.

Full description

The study objectives will be accomplished in a prospective, multicenter observational, longitudinal cohort study that includes 100-150 Pediatric Heart Transplant (PHT) patients from the United States. Patients will be screened for eligibility and enrolled ~1 month after PHT. Study participation will last 24 months.

All patients will follow the center's standard of care surveillance schedule after transplant. Blood samples will be collected for miR evaluation at:

1. specified time intervals after transplant and
2. when a clinical event of interest occurs, including rejection, infection, or major change in immunosuppression.

Research samples will be collected and used to evaluate microRNA expression as well as other biomarkers related to heart transplantation and immunosuppression medications. Additional data collection will include demographics, medical history, medications, human leukocyte (HLA)/donor specific antibody (DSA) evaluations, endomyocardial biopsy (EMB), echocardiography, donor-derived cell-free DNA (dd-cfDNA), and other post-transplant events and testing.

This work will form the basis for a non-invasive, genomic blood test that can be used to monitor patients after heart transplant to mitigate complications of over-immunosuppression, such as infection, without increasing the risks of under-immunosuppression, such as rejection.

Enrollment: 150 estimated patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age ≤ 18 years at enrollment
• Receipt of orthotopic heart transplant (OHT) within the prior 1 month ± 2 weeks
• Planned follow-up at the transplant center for a minimum of one-year.
• Caregiver able and willing to comply with the study visit schedule, study procedures, and study requirements.

Exclusion criteria

• Recipient of a multi-organ transplant
• History of prior solid organ transplant before the index heart transplant
• Ongoing mechanical circulatory support or hemodynamic instability
• Active infection requiring either a) hospitalization b) treatment with antimicrobial therapy or c) reduction in immunosuppression
• History of rejection prior to enrollment
• Inability to collect specified blood volume at enrollment +/- 1 week