SICKLE CELL DISEASE

This early-stage study tests a new gene therapy called CS-206 for people with severe sickle cell disease. The treatment uses a patient's own blood stem cells, which are modified with a gene-editing tool to correct the disease. Five participants aged 12 to 18 will receive a single…

This study tests whether a high dose of plerixafor can safely collect enough stem cells from people with sickle cell disease for a future transplant. About 12 participants aged 18-25 will receive transfusions, take the drug, and have stem cells collected through a process called …

This early study tests a one-time treatment called KL003 in 3 people with severe sickle cell disease. The treatment uses the patient's own blood stem cells, which are modified in a lab to produce healthy red blood cells. The goal is to see if the modified cells can safely and suc…

This study tests an experimental drug called SIL-8301 (senicapoc) in about 105 adults with sickle cell disease who have severe anemia and few pain crises. Participants take the drug or a placebo daily for 24 weeks, while continuing their usual hydroxyurea treatment. The main goal…

This study tests a drug called iloprost to see if it can reduce pain and the need for strong painkillers in adults with sickle cell disease who are hospitalized for a painful crisis. Iloprost helps open blood vessels and reduce inflammation. About 144 participants will receive ei…

This study tests whether minocycline, an antibiotic that reduces brain inflammation, can safely prevent or reverse memory and thinking problems in adults with sickle cell disease. About 30 participants will receive either a low dose, high dose, or placebo for one year. Researcher…

This study will test whether a 4-week music therapy program can reduce chronic pain and improve daily life for 25 youth with sickle cell disease. Participants will be randomly assigned to music therapy or a control group. Researchers will also look at changes in immune cells and …

This study tests if shining a special light on the head (transcranial photobiomodulation) can improve thinking and brain oxygen levels in children with sickle cell disease. About 60 kids aged 8-17 will be randomly assigned to get real light therapy or a placebo. The goal is to se…

This study looks at whether giving ketamine within the first hour of a sickle cell pain crisis can lower the chance of being admitted to the hospital. About 90 children and young adults aged 6 to 24 will receive either ketamine or a placebo by mouth. The goal is to see if early t…

This study tests a training program for healthcare providers that teaches them to see each patient as an individual and understand their perspective. The goal is to improve communication with children who have sickle cell disease. About 30 providers from three hospitals will be r…

This study will look at how genetics and body processes affect the way Nigerian patients with sickle cell disease respond to the drug hydroxyurea. Researchers will study 100 patients to create a computer model that can help doctors choose the best dose for each person. The goal i…

This study will look at how often children with sickle cell disease have painful vaso-occlusive crises and what problems arise from them. Researchers will review medical records of 25 children aged 1-18 to find risk factors and complications like acute chest syndrome or stroke. T…