SICKLE CELL DISEASE

This early-phase study tests a gene therapy approach for sickle cell disease. The treatment uses a modified virus to add a gene that boosts fetal hemoglobin, which can reduce sickling and may cure the condition. Ten participants with severe sickle cell disease will receive their …

This study looks at the long-term safety of a daily pill called pociredir in adults with sickle cell disease who have already seen benefits from it in a previous study. About 50 participants will take the drug for up to 48 months. The goal is to monitor side effects and check if …

This study tests a single dose of BEAM-101, a treatment made from a patient's own gene-edited stem cells, for people aged 12 to 35 with severe sickle cell disease. The goal is to reduce or eliminate painful vaso-occlusive crises. Participants will be followed for safety and how w…

This study tests an oral medication called etavopivat in 450 people with sickle cell disease. The goal is to see if it can raise hemoglobin levels and reduce the number of painful vaso-occlusive crises (blocked blood vessels). Participants are randomly assigned to receive the dru…

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

This study tests a single dose of a gene-edited stem cell treatment (CTX001) in 13 children with severe sickle cell disease who cannot take or did not benefit from hydroxyurea. The goal is to see if the treatment can prevent severe pain crises and hospital stays for at least a ye…

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

This study aimed to test the drug mitapivat in people with sickle cell disease who also have kidney damage (nephropathy). The goal was to see if mitapivat could reduce protein in the urine, a sign of kidney stress. The trial was withdrawn before any participants were enrolled, so…

This early-phase trial 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 along with a drug called alemtuzumab. The goal is to reduce …

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 family donor and a milder chemotherapy to prepare the body, along with removing certain immune cells from the donor cells to…

This study tests a drug called mitapivat in 286 people with sickle cell disease to see if it can raise hemoglobin levels and reduce painful sickle cell crises. Participants will receive either the drug or a placebo, and researchers will monitor safety and effectiveness over time.…

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 follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). Researchers will monitor participants for years to check for side effects, new cancers, and how well the thera…

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. The trial involves 25 participants aged 13…

This study tests a new stem cell transplant method for adults with severe sickle cell disease. Participants receive stem cells from a half-matched relative after low-dose radiation and immune-suppressing drugs. The goal is to see if the transplant can control the disease without …

This study looks at whether voxelotor, a drug that helps red blood cells stay healthy, can improve physical function in people with sickle cell disease. Six adults will take the drug for 6 months, and their walking ability will be measured before and after treatment. The goal is …

This study tests the safety and benefits of hydroxyurea, a medicine that helps control sickle cell disease, in children aged 3 to 10 in Africa. The goal is to make this treatment more widely available, especially for kids diagnosed early through newborn screening. The trial invol…

This study follows 4 people with sickle cell disease who received a one-time treatment of gene-edited stem cells (OTQ923). The goal is to check for any delayed side effects and see how long the therapy's benefits last. Participants must have already completed at least one year of…

This study tests a new drug, tebapivat, to see if it can improve anemia in people with sickle cell disease. About 59 adults will receive either tebapivat or a placebo pill. The main goal is to measure increases in hemoglobin and check for side effects.

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 aimed to test a single dose of exa-cel, a gene therapy, in people with severe sickle cell disease who have the HbSC genotype. The goal was to see if it could safely raise fetal hemoglobin levels to reduce complications. However, the study was withdrawn before enrolling…

This Phase 3 study tests whether adding automated red blood cell exchange to usual care reduces hospital visits and deaths in adults with sickle cell disease who are at high risk. About 173 participants will be randomly assigned to receive either standard care alone or standard c…

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 with SCD. Community health workers will provide special nutr…

This study tests whether acceptance and commitment therapy (ACT), a type of talking therapy, can help adults with sickle cell disease who have trouble sleeping. Participants will have weekly video chats with a coach for 8 weeks and wear a wrist device to track sleep. The goal is …

This study looks at whether a moderate exercise program can help children with sickle cell disease improve their muscle strength, balance, and ability to walk and move. Twenty children aged 6 to 17 will take part in a targeted training program. The goal is to see if the program i…

This study tests whether a simple, self-administered acupressure routine can reduce pain for people with sickle cell disease. Three hundred adults will learn to apply pressure to specific points on their own body every other day. The goal is to see if this drug-free method eases …

This study tests whether acupuncture can reduce pain in people with sickle cell disease. Researchers will use brain scans and other tests to understand how acupuncture works. The study involves 60 participants aged 14 to 80 who have chronic pain or recent pain crises.

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

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 these barriers and find out what kind of text-message reminders or tips they would prefer. The…

This study looks at whether a special pill bottle that records each time it's opened can accurately track how often teens with sickle cell disease take their medication, hydroxyurea. About 36 teens aged 12 to 17 will use the bottle for two months. The goal is to see if this devic…

This study follows 102 adults with sickle cell disease to understand how often silent and overt strokes occur. Researchers use MRI scans and neurological exams to track strokes over time. The goal is to gather the data needed to design future clinical trials for stroke prevention…

This study was designed to track how long specially treated red blood cells survive in people with sickle cell disease who receive regular blood exchanges. It planned to enroll adults with sickle cell disease who had been on exchange therapy for at least three months. However, th…

This study was designed to see if holistic education could help people manage stress at work and in daily life. It was meant for unmarried Alaska Medicaid recipients aged 22 and older. The study was withdrawn before any participants were enrolled, so no results are available.

This study collects blood and other samples from 2,000 adults with sickle cell disease and healthy volunteers. Researchers will use the samples to study how blood vessels work and what goes wrong in diseases like sickle cell. The goal is to better understand these conditions, not…

This study looks at how different types of fats in the blood (like cholesterol) are linked to complications in adults with sickle cell disease. Researchers will follow 116 patients in the French West Indies over several years, checking their fat levels during normal times and dur…

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 blood flow tests to find hidden damage and check for risk factors like high blood pressure. The goal…

This study tests a web-based program called CHOICES to help adults with sickle cell disease or trait learn about their reproductive health. The goal is to see if the program improves knowledge about genetic risks, safe contraception, and pregnancy options. 506 men and women aged …

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…