SICKLE CELL ANEMIA

This study tests a stem cell transplant using cells from mismatched unrelated or partially matched family donors for people with severe sickle cell disease and other transfusion-dependent anemias. The approach removes certain immune cells (T-cells) from the donated stem cells to …

This study tests whether alendronate, a drug used for osteoporosis, can help adults with sickle cell disease who have bone damage (osteonecrosis). Thirty participants will take a weekly pill for 24 weeks. The main goals are to see if the treatment is feasible and safe, and to mea…

This study tests a one-time treatment called CTX001, which uses CRISPR gene editing to modify a patient's own stem cells to produce more fetal hemoglobin. The goal is to reduce or eliminate the need for blood transfusions in people with transfusion-dependent beta-thalassemia or s…

This study collects blood samples from 250 adults with sickle cell disease, sickle cell trait, or healthy red blood cells. Researchers will use these samples to screen thousands of compounds, including FDA-approved drugs, to find ones that stop red blood cells from sickling. The …

This study looks at how the drug hydroxyurea affects women with sickle cell disease and their babies during pregnancy and breastfeeding. Researchers will review medical records from 200 women to measure drug exposure and compare health outcomes. The goal is to better understand t…

The SAFE study is a long-term research project following 250 adolescents and young adults with sickle cell anemia. It aims to understand how the medicine hydroxyurea affects growth, puberty, and the ability to have children. Researchers will also compare pregnancy outcomes betwee…

This study looks at whether children with sickle cell disease (SCD) develop enough antibodies after getting the measles vaccine. Researchers will enroll 130 children aged 6 months to 6 years who are due for their measles shots. Blood samples will be taken a few weeks after each d…