Clinical Trials

The goal of the study is to improve the quality of heart CT scans by using new methods to create the images.

This is randomized, double blind, study of dronabinol as a palliative agent in the treatment of pain, inflammation, and other complications of sickle cell disease (SCD).

his trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein (HbF and red blood cells expressing HbF)and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice.

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 long-term goal of this line of research is to identify the most effective components of SCD interventions while also identifying effective implementation strategies informed by a systematic and rigorous work done by the members of the SCDIC consortium to improve the quality of life of people with SCD, a life-threatening hematological disorder. This study aims to collect detailed information about the planned interventions, the implementation strategies and the control conditions from each site.

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and episodically transfused patients. The objective of the study is to determine how specific genetic and non-genetic factors in donors and recipients may impact RBC survival after transfusion - in short, what factors on both the donor and recipient side may improve the efficacy of the transfusion.

This study is a randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).

This is a study for parents of young children with Sickle Cell Trait (SCT) identified by newborn screening who are referred and present for in person SCT education at the Institution.

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.