SICKLE CELL DISEASE

This early-phase study tests a gene therapy for sickle cell disease. The approach uses a patient's own blood stem cells, modified to boost fetal hemoglobin and reduce sickle hemoglobin, potentially curing the condition. Ten participants with severe sickle cell disease will receiv…

This study tests a drug called mitapivat in people with sickle cell disease. The goal is to see if it can raise hemoglobin levels and reduce painful sickle cell crises compared to a placebo. About 286 people aged 16 and older will take part. The trial has two phases: first to fin…

This study looks at the long-term safety of a daily pill called pociredir for adults with sickle cell disease who have already taken it and seen benefits in a previous study. About 50 participants will take the drug once a day for up to 48 months. Researchers will monitor side ef…

This study tests a new combination of drugs and low-dose radiation to make stem cell transplants safer for people with sickle cell disease, even those with organ damage. Six participants aged 16 and older will receive a transplant from a family donor. The goal is to see if the ne…

This study tests a drug called tebapivat in 59 people with sickle cell disease to see if it can improve anemia (low red blood cells) better than a placebo. Participants take either tebapivat or a dummy pill, and researchers check for changes in hemoglobin levels and any side effe…

This Phase 3 trial tests whether automated red blood cell exchange (a type of blood transfusion) plus standard care can lower the number of sickle cell disease flare-ups that require emergency care or cause death over 12 months. It involves 173 adults with sickle cell disease who…

This early study tests a new way to prepare the body for a stem cell transplant in people with sickle cell disease. Instead of full-body radiation, doctors use targeted radiation to the bone marrow and lymph nodes, plus a drug called alemtuzumab, to reduce damage to healthy organ…

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who already received an experimental CRISPR gene therapy called CTX001. Researchers will track participants for years to check for long-term side effects, new cancers, and how well the treatme…

This study aimed to test a single dose of exa-cel (CTX001) in adolescents and adults with a severe form of sickle cell disease called HbSC. The treatment was designed to increase fetal hemoglobin to reduce pain crises and other complications. However, the study was withdrawn befo…

This study tests an oral drug called etavopivat in 450 people with sickle cell disease. The goal is to see if it can raise hemoglobin levels and reduce painful blockages in blood vessels. Participants take the pill or a placebo for up to a year, and researchers track how often pa…

This study tests a new type of stem cell transplant for people with severe sickle cell disease who don't have a fully matched donor. It uses a half-matched relative as a donor and a milder chemotherapy to prepare the body, along with removing certain immune cells from the donated…

This early-stage trial tests a one-time treatment called BEAM-101 for people with severe sickle cell disease. The therapy uses the patient's own blood stem cells, which are gene-edited to produce healthy red blood cells and reduce painful blockages. The study aims to see if it is…

This study follows 4 people with sickle cell disease who already received a gene-edited cell therapy called OTQ923. Researchers will check for delayed side effects, such as new cancers or blood disorders, and measure how long the therapy's benefits last. The goal is to understand…

This study looks at the long-term safety of a drug called mitapivat for adults with stable sickle cell disease. Participants, who must have benefited from the drug in a prior study, take mitapivat tablets twice daily for up to 48 weeks, with an option to continue for another 48 w…

This study tests a gene therapy that modifies a patient's own blood stem cells to increase fetal hemoglobin, a healthy type that prevents sickling. The goal is to reduce or eliminate severe pain crises in people with sickle cell disease. About 25 participants aged 13 to 40 with s…

This study tests the safety and benefits of hydroxyurea, a daily medication, for children aged 3 to 10 with sickle cell anemia in Africa. The goal is to make this treatment more widely available to reduce pain and complications. Children take the drug long-term to control the dis…

This study aimed to see if the drug mitapivat could improve kidney function in people with sickle cell disease and kidney damage. It planned to enroll about 0 participants aged 16 and older with specific types of sickle cell disease. The trial was withdrawn before any participant…

This study tests a stem cell transplant from a half-matched family donor to treat sickle cell disease in adults. The goal is to see if the new, gentler transplant method is safe and can stop severe complications like strokes. Participants receive low-dose radiation and immune-sup…

This study tests a one-time gene-editing treatment (CTX001) in children with severe sickle cell disease who cannot take hydroxyurea. The treatment uses the child's own blood stem cells, modified with CRISPR-Cas9, to produce healthy red blood cells. The goal is to prevent severe p…

This study looks at whether voxelotor, a drug that helps red blood cells stay healthy, can improve physical function in adults with sickle cell disease. Six participants will take the drug for 6 months and have their walking ability tested. The goal is to see if the drug helps th…

This early-stage study tests whether a drug called plerixafor can safely collect enough stem cells from people with sickle cell disease for a future gene therapy. Five participants will receive up to two injections of plerixafor. The goal is to see if this method is safe and work…

This study tests a special care program for pregnant women with sickle cell disease in Ghana. The program uses a team of doctors and nurses to prevent serious problems like acute chest syndrome and to monitor baby growth with ultrasound. About 500 women will take part to see if t…

This study tests a new way to do stem cell transplants for people with sickle cell disease. Instead of needing a perfectly matched donor, it uses a half-matched family donor (haploidentical). Before the transplant, patients get two rounds of immune-suppressing therapy to help the…

This study tests whether a personalized dosing approach for hydroxyurea can lower the need for blood transfusions in children with sickle cell anemia. Researchers will compare transfusion rates before and during treatment in 100 children aged 1 to 10 years. The goal is to improve…

This study looks at whether teaching mothers about nutritious local foods can prevent malnutrition in children with sickle cell disease (SCD) in northern Nigeria. Researchers will enroll 148 children aged 6 to 18 months and compare their growth and diet to those receiving standar…

This study looks at whether a moderate exercise program focused on strength, balance, speed, and agility can improve movement and endurance in children with sickle cell disease. Twenty children aged 6 to 17 will participate. The goal is to see if the program is safe and helpful f…

This study looks at whether acupuncture can reduce pain in people with sickle cell disease. Researchers will use brain scans and other tests to understand how acupuncture works. About 60 people aged 14 to 80 with sickle cell disease and chronic pain will take part.

This study looks at whether a plant-based omega-3 supplement from flaxseed can reduce pain and improve daily life for children with sickle cell disease. About 30 children aged 5 to 18 will take flaxseed and report their pain levels and quality of life. The goal is to find a more …

This study looks at whether acceptance and commitment therapy (ACT) can improve sleep in adults with sickle cell disease. Participants will have weekly video chats with a coach for 8 weeks and wear a wrist device to track sleep. The goal is to see if the therapy is helpful and ea…

This study tests whether a simple, self-administered acupressure routine can reduce pain in adults with sickle cell disease. Three hundred participants will apply pressure to specific points on their own bodies every other day. The goal is to see if this non-drug approach can low…

This study tests a web-based program to improve reproductive health knowledge in adults with sickle cell disease or trait who plan to have a child within two years. The program uses online quizzes and reminders to help participants understand genetic risks and parenting options. …

This study aimed to see if holistic education could help people manage stress and related conditions like anxiety, fatigue, and PTSD. It was designed for unmarried Alaska Medicaid recipients aged 22 and older who agreed to a smoke-free, drug-free, and alcohol-free lifestyle. The …

This study was designed to see how long pathogen-reduced red blood cells survive in adults with sickle cell disease who receive regular red cell exchange therapy. Researchers planned to use special markers to track the cells. However, the study was withdrawn before enrolling any …

This study collects blood and other samples from up to 2000 adults with sickle cell disease and healthy volunteers. Researchers will use the samples to study how inflammation affects blood vessels in sickle cell and related diseases. The goal is to better understand these conditi…

This study follows 102 adults with sickle cell disease to track silent and overt strokes using brain scans and exams. The goal is to understand how often strokes happen and what risk factors matter, so future trials can test ways to prevent them. No new treatments are given; part…

This study looks at whether an electronic pill bottle (AdhereTech) can accurately measure how often teens with sickle cell disease take their medication, hydroxyurea. About 36 teens aged 12-17 will use the device for two months. Researchers will compare the device's data to surve…

This study looks at how common strokes and silent brain injuries are in young adults (ages 16-25) with sickle cell anemia in northern Nigeria. Researchers will use brain scans and ultrasound to check for damage and also look at other risk factors like high blood pressure. The goa…

This study looks at fat levels in the blood of adults with sickle cell disease living in the French West Indies. Researchers want to see if these fat levels are linked to complications like pain crises, strokes, or organ damage. The goal is to better understand the disease, not t…

This study looks at why people with sickle cell disease sometimes struggle to take their daily medications. Researchers will interview and survey 70 patients aged 12 to 70 to understand their challenges and find out what kind of text-message reminders they would prefer. The goal …

This study looks at how platelets and inflammation work together during painful episodes in adults with sickle cell disease. Researchers will analyze blood samples from 25 participants to understand these processes better. The goal is to gather knowledge, not to test a new treatm…