Clinical Trials

This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat Severe Aplastic Anemia.

This is an open-label study of the combination of atomoxetine and oxybutynin (ato-oxy) in children with Down syndrome and obstructive sleep apnea (OSA) documented by polysomnography (PSG).

Have you or your child been diagnosed with a blood disorder? If so, you (or they) may be able to participate in a clinical study where researchers are studying a type of infusion using cord blood. Participants can be of any age but must have a blood disorder diagnosed by their doctor. The study is taking place in locations in Alabama, Arizona, California, Colorado, Delaware, Florida, Georgia, Illinois, Indiana, Kentucky, Maryland, Massachusetts, Michigan, Minnesota, Mississippi, Missouri, Nebraska, New York, Ohio, Oklahoma, Oregon, Pennsylvania, South Carolina, Tennessee, Texas, Virginia, Washington, Washington, D.C., West Virginia, and Wisconsin.

Are you scheduled for a stem cell transplant with cord blood? This study will assess the safety and effectiveness of certain cord blood transplants. The study will help researchers learn the best methods for collecting, storing, and using cord blood in transplants. To participate in this study, you must have a disorder that compromises your body’s ability to make blood cells. This study is located in Bethesda, Maryland.

Do you or your child have severe asthma that is not easily controlled by inhaled corticosteroids? This study evaluates the use of five different asthma medicines to treat adults and adolescents with severe asthma. To participate in this study, you or your child must be 12 years old or older. This study takes place in multiple locations across the United States.

Have you or your child been diagnosed with cystic fibrosis, and would you like to support better diagnosis of lung infections? This study is exploring new ways to diagnose bacterial lung infections in people who have cystic fibrosis. The study will test whether analyzing a participant’s breath and sputum samples can help diagnose infections. Participants in this study must be at least 3 years old. This study is taking place in multiple locations in the United States.

Is your child an individual with Down syndrome and obstructive sleep apnea? This study is examining whether a combination medication therapy that includes atomoxetine and oxybutynin can help treat obstructive sleep apnea in children with Down syndrome. Participants in this study must be between 6 and 17 years old. This study takes place in Tucson, Arizona.

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.

Does your child have Down Syndrome? Did they have a complete atrioventricular septal defect (CAVSD) repair within the first year of their life? Researchers in this study are investigating how the
brain develops in children with Down Syndrome who have congenital heart disease. To participate, your child must be between 5 and 12 years old and have Down Syndrome. The study will enroll children who have congenital heart disease as well as children who do not have congenital heart disease. The study is taking place in locations in Delaware, Georgia, Indiana, Massachusetts, Michigan, Missouri, New York, Ohio, Pennsylvania, South Carolina, Texas, Utah, Washington, D.C., and Toronto, Canada