Sickle cell disease

This trial tests a gene therapy called YOLT-204 in people aged 14 to 35 with sickle cell disease or transfusion-dependent thalassemia. The goal is to see if a single intravenous dose can safely boost fetal hemoglobin levels, potentially reducing or eliminating the need for blood …

This pilot trial tests a gene-editing approach for sickle cell disease. Participants receive their own blood stem cells that have been edited to increase fetal hemoglobin, a healthy type of hemoglobin that prevents sickling. The goal is to cure the disease with a one-time treatme…

This early-stage study tests a one-time gene therapy (CS-206) in 5 teenagers with severe sickle cell disease. The treatment uses the patient's own blood stem cells, modified with a gene-editing tool, to help produce healthy red blood cells. The goal is to reduce painful blockages…

This early study tests a gene therapy called KL003 in just 3 people with severe sickle cell disease. The therapy uses the patient's own stem cells, modified to produce healthy hemoglobin, to reduce painful crises and the need for transfusions. The main goals are to see if the tre…

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. Twelve patients aged 18-25 will receive the drug and have their stem cells collected through a process called apheresis. The goal …

This study investigates whether urine tests for two biomarkers (NAG and KIM-1) and special MRI scans can detect early kidney damage in children with sickle cell disease. The goal is to find a way to identify kidney problems before they become serious, potentially allowing earlier…

This study tests whether combining point-of-care screening with community-based strategies can help identify infants with sickle cell disease early and connect them to care. Researchers will screen up to 6,750 newborns in Mozambique and track their health outcomes. The goal is to…

Adults with sickle cell disease often face severe pain crises that require hospital treatment with intravenous morphine. This trial compares standard morphine treatment with combinations that add paracetamol, ketamine, or nefopam. Researchers want to see if these combinations eas…

This trial tests whether a smartphone-based mindfulness program can help adolescents and young adults with sickle cell disease manage chronic pain and sleep problems. Participants are randomly assigned to either use the mindfulness app for about 8 weeks or receive usual care. The…

This study tests a virtual reality app called Amani designed to help adults with sickle cell disease manage pain, emotional distress, and social isolation. Researchers will enroll 40 adults to see if the app is easy to use and helpful for improving coping skills and peer support.…

This study explores whether using a virtual reality headset can help reduce pain for adults with sickle cell disease during a painful crisis. Participants will try different types of headset experiences—like watching a 2D video or exploring a 3D world—during their infusion center…

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 take either a low dose, high dose, or placebo for one year. Researchers w…

This study tests if iloprost, a drug that widens blood vessels and reduces inflammation, can help adults with sickle cell disease during painful vaso-occlusive crises. About 144 participants hospitalized for a crisis will receive either iloprost or a placebo, plus standard care. …

This study tests whether giving ketamine within an hour of pain onset can prevent hospital admission for children and young adults with sickle cell disease. 90 participants aged 6-24 will receive either ketamine or a placebo by mouth. If admitted, they can still receive standard …

This study tests whether shining a special light on the head (transcranial photobiomodulation) can improve oxygen use and thinking skills in children with sickle cell disease. The brain needs steady oxygen, but sickle cell can reduce blood flow. Researchers will randomly assign 6…

This study tests whether a computer simulation can help pharmacists and pharmacy students better understand the pain struggles of sickle cell disease patients. About 200 healthcare professionals will use a point-of-view simulation and then complete surveys to see if their misconc…

This study looks at how genetic differences affect the way Nigerian sickle cell patients respond to hydroxyurea, a common treatment. Researchers will monitor 100 patients to build a computer model that predicts the best dose for each person. The goal is to reduce side effects and…

This study tests a 90-minute virtual training for healthcare providers that teaches them to see each patient's unique perspective and communicate more clearly. The goal is to improve how doctors talk to children with sickle cell disease. Thirty providers from three hospitals will…

This study looks at how different types of anemia—like iron deficiency or thalassemia—can change HbA1c levels, a common test for diabetes, in people who do not have diabetes. Researchers will compare HbA1c results between 100 non-diabetic adults with various anemias and a healthy…

This study will review medical records of 25 children with sickle cell disease to see how often they experience painful vaso-occlusive crises and what complications arise. Researchers hope to identify risk factors that could lead to better care. No new treatments are being tested…