Clinical Trials

Is your newborn in the neonatal intensive care unit at St. Louis Children’s Hospital? This study aims to better understand breathing problems and blood flow through the heart in premature newborns to better detect conditions such as pulmonary hypertension. To participate in this study, your newborn must have been born prematurely between 24 and 29 weeks’ gestation and be a patient in the Neonatal Intensive Care Unit at St. Louis Children’s Hospital. This study is located in St. Louis, Missouri.

Do you or a family member have pulmonary hypertension? This study is looking at how hormones contribute to pulmonary arterial hypertension and whether treatments for hormone imbalances or insulin resistance also improve pulmonary hypertension. To participate in this study, you must be 90 years old or younger and have pulmonary arterial hypertension or be a family member of someone who has this condition. This study is located in Nashville, Tennessee.

Este estudio explora si un medicamento llamado espironolactona puede mejorar la función cardíaca y pulmonar durante el ejercicio en personas con hipertensión pulmonar. Para participar en este estudio, debe tener entre 18 y 99 años y un tipo de hipertensión pulmonar conocido como hipertensión arterial pulmonar. Este estudio se lleva a cabo en Bethesda (Maryland).

Are you an adult with pulmonary arterial hypertension (PAH) and currently taking vasodilators? This study is testing the efficacy of a new medicine called famotidine for people with PAH. Participants in this study must be between 18 and 80 years old and willing to take famotidine or a placebo for 6 months. This study takes place in Seattle, Washington.

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 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.

This study is seeking to determine the safety and tolerability of dronabinol use in adults with SCD compared to placebo.

Are you at least 18 years old and have sickle cell disease and taking the medication Hydroxyurea? For this study, researchers testing are tool for measuring oxygen levels, blood flow, and the makeup of skin and muscle in patients with sickle cell disease. To participate in this study, you must be at least 18 years old and have sickle cell disease and also be taking the medication Hydroxyurea. This study is taking place at the National Institutes of Health Clinical Center in Bethesda, Maryland.