Clinical Trials and Studies

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).

Do you have sickle cell disease? Researchers are testing whether a drug that is often used to treat nausea caused by chemotherapy can also treat chronic pain and improve quality of life in adults living with sickle cell disease. Participants must be between 18 and 80 years old. The study is taking place at Mount Sinai Hospital in New York City.

Are you at least 18 years old and have sickle cell disease? People with sickle cell disease often have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant. Researchers in this study are trying to learn how and why this happens. To participate in this study, you must be at least 18 years old and have sickle cell disease. You must also have a history of problems with your heart, lungs, or other criteria that the study doctor will discuss with you. This study is taking place at the National Institutes of Health Clinical Center in Bethesda, Maryland.

Do you or your child have sickle cell disease, and would you be willing to receive sickle cell care through telemedicine? Researchers are trying to learn more about how two different types of telemedicine can improve care for children living with sickle cell disease. Participants include caregivers (18 years old and older) who have children between 0 to 21 years old with sickle cell disease and adolescents and young adults with sickle cell disease. The study is taking place at Riley Hospital for Children Comprehensive Sickle Cell Disease Clinic in Indianapolis, Indiana.

Do you have sickle cell disease? People with sickle cell disease are at risk of developing blood clots. Researchers are studying a medicine that may prevent blood clots in patients with sickle cell disease and has less risk of bleeding. To participate in this study, you should be 18 to 65 years old and have sickle cell disease. This study is located in Bethesda, Maryland.

Do you have sickle cell disease and problems in your hip or shoulder joint? Researchers are studying a medicine to treat osteonecrosis (painful bone condition due to lack of blood flow) in patients with sickle cell disease. To participate in this study, you should be 18 to 80 years old and have sickle cell disease and hip or shoulder problems. This study is recruiting in Sacramento, California.

Do you have sickle cell disease) and have you had severe pain in the past 12 months? The goal of this study is to identify factors that predict severe pain outcomes in patients with sickle cell disease to improve pain management. To participate in this study, you should be 15 to 40 years old and have sickle cell disease. This study is recruiting in Durham, North Carolina.

Do you or your child have sickle cell disease? Researchers are looking at how heart-, lung-, and kidney-related health outcomes compare between those living with sickle cell disease who have standard treatment versus those who have stem cell transplant. Participants must be between 4 and 65 years old. The study is taking place at sickle cell and transplant centers in Washington, D.C., Georgia, Tennessee, and Maryland, including at the National Institutes of Health Clinical Center in Bethesda, Maryland.

Do you take hydroxyurea for sickle cell disease? This study looks at the use of hydroxyurea comparing two types of interventions to encourage and monitor adherence with daily hydroxyurea. One group will receive telephone calls and texts and submit videos showing daily hydroxyurea administration and another group will receive texts with health tips. Children and adolescents up to 25 years old may be able to participate in this study if they have a diagnosis of sickle cell disease and are taking hydroxyurea. This study is located in Chicago, Illinois, and Providence, Rhode Island.