Clinical Trials

Is your child undergoing heart surgery for congenital heart disease? This study aims to help researchers better understand the developing heart and what controls the strength of its beats by comparing it to adult hearts. Researchers will study small pieces of the heart that are removed as a normal part of surgery or repair for children with congenital heart disease. To participate in this study, your child must be undergoing cardiopulmonary bypass surgery and be 18 years old or younger. The study is located in Atlanta, Georgia.

Do you or your child have a heart defect that requires a procedure called extracardiac conduit–total cavopulmonary connection (EC-TCPC)? This study is evaluating the effectiveness of a new type of graft called a tissue-engineered vascular graft for EC-TPC. Participants of this study will have this procedure and several follow-up assessments with magnetic resonance imaging (MRI) to test the performance of the graft. This study takes place in Columbus, Ohio.

Sickle Cell Disease (SCD) impairs oxygen transport to tissue and causes endothelial injury. Thus, therapeutic interventions aim to improve both, but there is an unmet need for biomarkers to determine when intervention is necessary and evaluate the effectiveness of the chosen intervention in individual patients. This study proposes to monitor SCD and its treatment through their impact on cerebral hemodynamics, as the brain is one of the most vulnerable and consequential targets of the disease. Specifically, this study will optimize quantitative magnetic resonance imaging (MRI) and advanced optical spectroscopy techniques such as frequency-domain near-infrared and diffuse correlation spectroscopies (FDNIRS-DCS) to monitor 1) cerebral oxygen transport with measures of cerebral blood flow (CBF), cerebral oxygen extraction fraction (OEF) and cerebral metabolic rate of oxygen consumption (CMRO2) and 2) endothelial function with cerebrovascular reactivity (CVR).

The main goal of this study is to determine whether the treatment will lead to a complete absence of severe vaso-occlusive events (VOEs) in patients with severe SCD.

Are you or your child at least 13 years old and have sickle cell disease? In this stidy, researchers are trying to find out if a new medication improves the success of a blood stem cell transplant. People 13 years old or older who are eligible for a blood stem cell transplant to treat sickle cell disease may be able to participate. Healthy family members over age 13 who are matched to transplant recipients are also needed to donate blood for the study. This study is taking place at the National Institutes of Health Clinical Center in Bethesda, Maryland.

The purpose of this study is to learn more about how the use of two different types of telemedicine (distance medical care) can address barriers to receiving comprehensive sickle cell care, and whether care can be improved.

This is a pilot study to evaluate the feasibility and safety of providing RH genotype matched D+ Red Blood Cells (RBCs) to chronically transfused patients with sickle cell disease (SCD) who type D+ but have formed anti-D and are currently transfused with D- RBC (Red Blood Cell) units.

The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD.

Do you or your child have sickle cell disease and have a stem cell donor? This study is exploring how to improve bone marrow transplant procedures so the body better accepts donor stem cells. Participants in this study must be at least 4 years old. This study is located in Bethesda, Maryland.

Do you or your child have sickle cell disease or sickle cell trait? This study is interested in understanding pain crises and lung complications that occur in sickle cell disease patients and patients who have other red blood cell disorders. To participate in this study, you must be at least 2 years old with known or suspected sickle cell disease, sickle cell trait, or other red blood cell disorders. Participants will have a physical exam and receive standard medical care for sickle cell disease, including routine follow-up tests and procedures. This study is located in Washington, D.C., and Bethesda, Maryland.