SICKLE CELL DISEASE

This early-stage study is testing a new method to safely collect a patient's own blood stem cells, which is a necessary first step for a future gene therapy. The goal of that future therapy is to provide a one-time cure for sickle cell disease. This small pilot study will check i…

This is an early safety study testing a one-time gene therapy that aims to cure sickle cell disease. Doctors collect a patient's own blood stem cells, use a modified virus to insert a corrective gene, and then return the cells after chemotherapy. The goal is to see if this approa…

This study is testing a new combination of drugs designed to make a curative stem cell transplant safer, especially for sickle cell patients who already have damage to organs like their heart, lungs, or kidneys. The goal is to help the patient's body accept donor cells without se…

This was a Phase 3 trial designed to see if a single dose of a gene therapy called exa-cel could cure severe sickle cell disease in adolescents and adults. The treatment aimed to permanently modify a patient's own blood stem cells to stop the disease. The study was withdrawn befo…

This study tests whether monthly automated blood exchange transfusions can reduce hospital visits and prevent deaths in adults with sickle cell disease who are at high risk of heart and lung complications. Researchers will compare the transfusion approach against standard care in…

This study is following 4 patients for 15 years after they received an experimental gene therapy for sickle cell disease. The therapy involves editing a patient's own blood stem cells to produce fetal hemoglobin, which can help control the disease. Researchers are monitoring long…

This study is testing a new oral medication called epeleuton in adults with sickle cell disease. The main goals are to see how the drug moves through the body, how safe it is, and if it can help control the disease by reducing markers of inflammation and pain crises. About 30 par…

This clinical trial is testing whether an oral medication called mitapivat can help people with sickle cell disease. Researchers want to see if the drug increases healthy hemoglobin levels in the blood and reduces painful crises compared to a placebo. The study involves 286 parti…

This study is testing whether a new oral medication called etavopivat can help people with sickle cell disease. It aims to increase healthy hemoglobin levels in the blood and reduce the frequency of painful vaso-occlusive crises. The trial involves 450 participants aged 12 to 65 …

This study aims to see if a proven team-based care program can be successfully used at other hospitals to save the lives of pregnant women with sickle cell disease and their babies in Ghana. It will enroll 500 pregnant women with sickle cell disease at three hospitals to receive …

This study tracks the long-term safety and effectiveness of a one-time CRISPR gene therapy called CTX001 in people with sickle cell disease or beta-thalassemia. It follows 160 children and adults who have already received the therapy in earlier studies. The main goal is to monito…

This study is checking if the sickle cell disease drug voxelotor can help patients walk farther and improve their physical function. It will follow 6 adults with sickle cell disease for 6 months, measuring how far they can walk in 6 minutes before and after treatment. The goal is…

This early-stage study is testing a new way to prepare patients for a stem cell transplant to treat severe sickle cell disease. The goal is to use a more targeted form of radiation to reduce damage to healthy organs and lower the risk of a serious transplant complication. Researc…

This early-stage study is testing a new type of stem cell transplant for adults with severe sickle cell disease. It uses a half-matched family donor and a milder chemotherapy regimen to prepare the body, aiming to reduce transplant risks. The main goals are to see if the procedur…

This study is testing the long-term safety and effects of a drug called mitapivat for adults with stable sickle cell disease. It is an extension for 15 people who benefited from an earlier short-term study. Participants will take the pill twice daily for up to two years while res…

This study is testing a one-time treatment called CTX001 in children with severe sickle cell disease who don't respond well to the standard medication, hydroxyurea. The treatment uses the patient's own modified stem cells (a type of gene therapy using CRISPR technology) to try to…

This study is testing a new, one-time treatment for severe sickle cell disease. Doctors take a patient's own blood stem cells, edit their genes in a lab, and then return them to the patient. The goal is to see if this single treatment can safely and significantly reduce the numbe…

This study is testing whether a new drug called crovalimab, when added to a patient's usual sickle cell treatments, can help prevent the severe pain episodes known as vaso-occlusive crises. It will involve about 90 participants with sickle cell disease who have experienced severa…

This small, early-stage study is testing whether a drug called etavopivat can improve blood flow and oxygen delivery to the brain in children and young adults with sickle cell disease. Researchers will use special light-based scanners to measure brain blood flow in about 12 parti…

This study aims to prevent strokes in young Nigerian adults with sickle cell anemia, where strokes affect 10% of patients. Researchers will measure how common strokes and silent brain injuries are in 250 participants aged 16-25, then test whether regular blood transfusions or a d…

This study is testing if teaching mothers about healthy, locally available foods can prevent malnutrition and help young children with sickle cell disease grow better. Community health workers will provide this education to mothers and compare the results to standard doctor advic…

This study is testing whether acupuncture can help manage the chronic pain experienced by people with sickle cell disease. Researchers are comparing traditional needle acupuncture to a needle-free laser version over 10 sessions. They will also use brain scans and other tests to t…

This small pilot study is testing whether a guided prayer and relaxation program can help adults with sickle cell disease manage their daily pain and stress. Researchers want to see if this non-drug approach, used alongside regular pain medication, can improve sleep and potential…

This remote study aimed to provide holistic education and pastoral counseling to help people manage stress and improve wellness in daily life. It was designed for specific unmarried Alaska Medicaid recipients who met religious and lifestyle criteria. The study was withdrawn befor…

This study is testing whether adding flaxseed, a plant-based source of omega-3 fatty acids, to the diet can help reduce pain and inflammation in children with sickle cell disease. Researchers will enroll 30 children, aged 5 to 18, to see if a flaxseed-rich diet is more acceptable…

This study is exploring whether following a Mediterranean diet for four weeks can help reduce the chronic pain experienced by adults with sickle cell disease. Researchers are comparing pain levels while participants eat the Mediterranean diet to their pain levels while eating the…

This small, remote pilot study is testing whether a type of counseling called Acceptance and Commitment Therapy (ACT) can help adults with sickle cell disease who have trouble sleeping. Participants will have weekly video coaching sessions for 8 weeks and wear a wrist device to t…

This study tests an online education program designed to help adults with sickle cell disease or trait make informed decisions about having children. The program provides information about genetic risks, reproductive options, and pregnancy safety. Researchers want to see if this …

This study aims to better understand how inflammation and blood clotting interact during painful episodes (vaso-occlusive crises) in adults with sickle cell disease. Researchers will collect blood samples from 25 hospitalized patients during their pain crisis, while they're in cr…

This study is testing a 'smart' pill bottle to see if it's a good way to measure how consistently teens with sickle cell disease take their daily medication (hydroxyurea). The bottle tracks when it's opened and sends the data to researchers. The goal is to find a more accurate me…

This study aims to understand why people with sickle cell disease sometimes struggle to take their daily medication. Researchers will interview patients to identify common barriers like cost, forgetfulness, or side effects. They will then work with patients to design and test per…

This study aims to understand the relationship between cholesterol, fats, and complications in adults with sickle cell disease. Researchers will measure blood fat levels in 116 patients over four years, both when they are well and when they experience painful crises or other comp…

This study is collecting blood and urine samples from adults with and without sickle cell disease to help researchers develop better laboratory tests. The samples will be used to study how inflammation and blood vessel function relate to sickle cell disease and other conditions. …

This study aims to understand stroke risk in adults with sickle cell disease, as current prevention guidelines are based on children's data. Researchers will follow 102 adults for several years, tracking who experiences strokes and monitoring brain health. The data collected will…