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 study tests whether red blood cells processed to be low in oxygen (hypoxic) last longer in the body than standard cells for people with sickle cell anemia who need regular transfusions. About 48 participants aged 7 and older will receive both types of blood in a crossover de…

This trial compares a new gene therapy to standard care in people aged 12 to 35 with severe sickle cell disease. The gene therapy uses a patient's own blood stem cells, modified to produce a therapeutic form of hemoglobin and reduce the sickling hemoglobin. The study measures how…

This trial investigates whether continuous positive airway pressure (CPAP) can help adults with sickle cell disease recover faster from acute chest syndrome, a serious lung complication. Participants who need extra oxygen are randomly assigned to receive either standard oxygen al…

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 study will test 120 babies in Côte d'Ivoire for sickle cell disease using a simple point-of-care test. Babies who test positive will start comprehensive care early, including medicines and vaccines. Researchers want to see if this approach reduces serious health problems and…

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 early-phase trial will test a single dose of a new drug called YOLT-204 in 18 children and teens with transfusion-dependent β-thalassemia or sickle cell disease. The goal is to see if it is safe and can raise fetal hemoglobin levels enough to reduce the need for blood transf…

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 a bedside lung ultrasound, done within six hours of arriving at the emergency department, can help diagnose acute chest syndrome in adults with sickle cell disease experiencing a vaso-occlusive crisis. Acute chest syndrome is a serious lung complication t…

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 short educational videos or interactive games can empower children with sickle cell disease. The goal is to help kids aged 12 to 18 understand their condition and build skills for self-care and managing pain. Researchers will measure changes in self-effic…

This trial tests whether a single dose of buprenorphine, given as a film inside the cheek, is safe and helps control pain in adolescents and young adults with sickle cell disease who are hospitalized for a pain crisis. Participants receive one dose of buprenorphine instead of sta…

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 will test whether eating flaxseed, a source of omega-3 fatty acids, can help reduce pain and improve daily life for children with sickle cell disease. Twenty children aged 5 to 18 will take part. The main goal is to see if it's possible for them to eat flaxseed regular…

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 tests whether a low-dose ketamine injection can relieve pain in children with sickle cell disease during a pain crisis better than standard opioid medications. Researchers will compare how quickly pain improves and whether fewer children need to be admitted to the hosp…

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 awake prone positioning (lying on the stomach while awake) can help sickle cell patients with severe acute chest syndrome in the ICU. The approach aims to improve oxygen levels and breathing by evening out air flow in the lungs. Fifteen adults will be enr…

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 will look at medical records of 350 children with sickle cell disease in France who had serious bacterial infections between 2020 and 2025. The goal is to find out which bacteria are causing these infections today, especially with new vaccines available. The results co…

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…

This study examines how regular blood transfusions (exchange transfusions) affect thinking speed in adults with severe sickle cell disease. Researchers will test 85 participants remotely using a simple symbol-matching task and other cognitive assessments at different points in th…