SICKLE CELL DISEASE

This study tests a new treatment called KL003 for people with severe sickle cell disease. The treatment uses a patient's own blood stem cells, modified with a healthy gene, to help produce normal red blood cells. Three participants aged 12 to 50 will receive a single infusion. Th…

This early-stage study tests a one-time treatment called CS-206 for people aged 12 to 18 with severe sickle cell disease. The therapy uses gene editing to modify the patient's own blood stem cells, aiming to reduce painful crises and hospital stays. Only 5 participants will be en…

This study tests an experimental drug called SIL-8301 (senicapoc) in people with sickle cell disease who have low hemoglobin and few pain crises. About 105 participants aged 16-35 will take either the drug or a placebo daily for 24 weeks, while continuing their usual hydroxyurea …

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 study tests whether giving ketamine early (within one hour of arriving at the hospital) can help children and young adults with sickle cell disease avoid being admitted for pain. Participants aged 6 to 24 will receive either ketamine or a placebo by mouth. If they are admitt…

This study tests whether the antibiotic minocycline can safely stop or reverse thinking and memory problems in adults with sickle cell disease. The drug may reduce brain inflammation that causes cognitive decline. Thirty participants will take either a low dose, high dose, or pla…

This study tests if shining a special light on the head (transcranial photobiomodulation) can improve brain function in children with sickle cell disease. Sickle cell disease can reduce oxygen to the brain, causing thinking and learning problems. The study will randomly assign 60…

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 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 a training program for healthcare providers to improve how they talk with children who have sickle cell disease. The training teaches doctors to see each patient as an individual and understand their perspective. 30 providers from three hospitals will be randomly…

This study aims to make hydroxyurea therapy safer and more effective for 100 Nigerian patients with sickle cell disease. Researchers will analyze genetics, drug levels, and patient responses to create a personalized dosing guide. The goal is to reduce painful crises and improve q…

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…