Clinical Trials

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 or your child have sickle cell disease? Researchers are testing whether a type of gene therapy will prevent all severe vaso-occlusive pain events in individuals living with sickle cell disease. In this study, stems cells are collected from the patient, modified in a lab, and then the gene-modified cells are given back to the patient. Participants must be between 13 and 40 years old. The study is taking place at sickle cell and transplant centers in California, Georgia, Illinois, Massachusetts, and Wisconsin.

Are you or your child at least 13 years old and have sickle cell disease? Do you or your child have sickle cell disease or beta-thalassemia? Researchers are testing whether a new antibody therapy prior to stem cell transplantation improves the success of the stem cell transplant in individuals at high risk for complications. Participants must be 13 years old or older. The study is taking place at the National Institutes of Health (NIH) Clinical Center in Bethesda, Maryland. Patients who have the stem cell transplant must live within 1 hour of the NIH Clinical Center for 3 months after the transplant.
The stem cells for the transplant come from a fully matched donor, 4 years old or older. Donors may provide blood samples for research.

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 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.

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 or your child have sickle cell disease? Researchers are testing a type of red blood cell transfusion matching for individuals living with sickle cell disease who receive regular red blood cell transfusions. Participants must be 8 years old or older. The study is taking place at the Children's Hospital of Philadelphia.

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.