SICKLE CELL DISEASE

This study tests a gene therapy that edits a patient's own blood stem cells to produce more fetal hemoglobin, a healthy type that can replace the defective hemoglobin causing sickle cell disease or beta-thalassemia. Up to 10 people aged 13-40 with severe forms of these blood diso…

This study tests a gentler bone marrow transplant for children with sickle cell disease using a sibling donor. The goal is to cure the disease with fewer side effects by using low-dose radiation and medicines that calm the immune system. About 100 children will take part to see i…

This study tests a new 21-valent pneumococcal vaccine (PCV21) in 100 children aged 2 to 17 with sickle cell disease, who are at high risk for serious infections. Researchers will compare PCV21 to a licensed vaccine to see if it is safe and helps the body produce protective antibo…

This study tests a partial stem cell transplant for people with severe sickle cell disease or beta-thalassemia who need regular blood transfusions. The goal is to get more donor cells to stay in the body long-term, which could reduce disease complications. About 90 participants w…

This early-stage study tests a new drug called ITU512 in healthy people and those with sickle cell disease. The goal is to see if it is safe and can increase fetal hemoglobin, which may reduce sickle cell complications. About 161 participants will take part, starting with healthy…

This study looks at whether giving hydroxycarbamide twice a day works better than once a day for children with sickle cell disease. About 50 children aged 9 months to 11 years will take the medicine every day for 12 months. Researchers will measure drug levels in the blood and ch…

This study looks at whether a mobile app that gives patients with sickle cell disease easy-to-use guidelines can help them have fewer emergency room visits and hospital stays. About 287 adults with sickle cell disease will use the app along with a booklet, or just the booklet alo…

This study tests a program to help doctors and clinics in Nigeria prescribe hydroxyurea, a medicine that reduces pain and complications from sickle cell disease. Currently, less than 1% of patients in Africa use it, even though it works. The study will enroll 900 patients and tra…

This early-stage trial tests the safety of a one-time treatment using the patient's own stem cells, edited with CRISPR technology to produce more fetal hemoglobin. The goal is to reduce painful crises and other complications of severe sickle cell disease. Up to 25 participants ag…

This study tests a single dose of a CRISPR-based gene therapy (CTX001) in 26 adults with transfusion-dependent beta-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, which are edited to produce more fetal hemoglobin, potentially red…

This early-stage study tests a new gene therapy called nula-cel in 15 people with severe sickle cell disease. The treatment uses the patient's own blood stem cells to change the sickle hemoglobin into healthy adult hemoglobin. The main goals are to check safety and see if it can …

This study tests whether adding the drug ruxolitinib to a standard stem cell transplant can lower the chance of graft failure in children and young adults (ages 12-45) with severe sickle cell disease. About 24 participants will receive the treatment and be followed for 2 years. T…

This study tests a new medicine called etavopivat to see if it can lower the number of painful sickle cell crises in people aged 12 and older with sickle cell disease. About 408 participants will receive either the drug or a placebo by chance. The trial lasts about 2 years and al…

This study offers ongoing access to crizanlizumab for people with sickle cell disease who were already benefiting from the drug in a previous Novartis trial. About 130 participants will continue treatment while researchers monitor safety. The goal is to provide uninterrupted care…

This study tests a telehealth program called C-READY that aims to improve thinking skills and self-management in 120 teens with sickle cell disease. Participants will complete online exercises and coaching to help them prepare for transitioning from pediatric to adult healthcare.…

This study tests whether the drug motixafortide is safe for people with sickle cell disease and if it can increase the number of stem cells that can be collected from their blood. These stem cells could be used for a future transplant, such as gene therapy. The trial involves 15 …

This study tests a stem cell transplant from a healthy donor for people with serious blood disorders like sickle cell disease or thalassemia. The goal is to replace the faulty blood cells with healthy ones, reducing the need for lifelong transfusions. Participants will receive a …

This study tests whether losartan, a blood pressure medication, can reduce or prevent scarring in the heart muscle of people with sickle cell disease. About 24 participants aged 6 and older will take losartan for one year. Researchers will use MRI scans to measure changes in hear…

This study tests a drug called crizanlizumab to see if it can reduce the number of painful crises (called vaso-occlusive crises) in people with sickle cell disease. About 315 teens and adults who have frequent crises will receive either the drug or a placebo, and some may also ta…

This study tests whether giving a drug called daratumumab before a stem cell transplant can prevent a serious complication where the body attacks donor red blood cells. It includes children and young adults (ages 2-24) with sickle cell disease who have a matched sibling donor. Pa…

This study tests a stem cell transplant from a half-matched family donor for people with severe sickle cell disease. Participants receive low-dose radiation and chemotherapy before the transplant to help the new cells take hold. The main goal is to see if the donor cells successf…

This study tests whether early heart screening and treatment for iron buildup in the heart can prevent heart failure, irregular heartbeats, and hospital stays in adults with sickle cell disease. About 100 participants will receive regular heart imaging and iron management. The go…

This early-stage study tests a new drug called DISC-3405 in 24 adults with sickle cell disease. The main goal is to see if the drug is safe and how the body handles it. Researchers will also check if it improves blood health markers like hemoglobin. This is not a cure, but aims t…

This study tests whether giving immune-suppressing drugs before a stem cell transplant can reduce serious side effects like graft failure and graft-versus-host disease. It includes adults and children aged 2 to 50 with sickle cell disease or beta-thalassemia who have a half-match…

This study follows 50 people with sickle cell disease who received the gene therapy BEAM-101 in an earlier trial. Researchers will monitor them for 13 years to check for long-term side effects, new health problems, and how well the treatment controls their disease. The goal is to…

This study tests a stem cell transplant for children with sickle cell disease who have a family donor with a different blood type. The goal is to see if a milder conditioning regimen can safely allow the transplant to work. Up to 12 children will take part. If successful, this co…

This early-phase study tests the safety of a drug called panobinostat in 18 adults with sickle cell disease. The drug aims to boost fetal hemoglobin and reduce inflammation, which may help manage the disease. Participants must be at least 18, have a confirmed diagnosis, and have …

This expanded access program offers a special stem cell processing technique (CD34+ selection) to patients with severe combined immune deficiency, Fanconi anemia, dyskeratosis congenita, or sickle cell disease who are receiving a stem cell transplant. The method removes certain i…

This study looks at whether giving pregnant women with sickle cell disease regular blood transfusions can reduce serious complications like pain crises, strokes, and pregnancy problems. About 50 women will be randomly assigned to receive either repeated transfusions or standard c…

This early-stage trial tests a one-time infusion of the patient's own blood stem cells, which have been corrected using CRISPR gene editing to fix the sickle cell mutation. The goal is to reduce painful blockages in blood vessels (vaso-occlusive events) and improve quality of lif…

This study tests a new daily pill, etavopivat, in 27 children aged 12–16 with sickle cell disease who are at higher risk for a first stroke. The medicine aims to improve blood flow in the brain, measured by ultrasound, over one year. Participants may already be taking hydroxyurea…

This program provides crizanlizumab (SEG101) to people with sickle cell disease who have no other treatment options and cannot join a clinical trial. It is for patients with serious or life-threatening conditions where the potential benefit outweighs the risks. The goal is to off…

This study tests a low dose of tocilizumab, a drug used for arthritis and COVID-19, to treat acute chest syndrome in people with sickle cell disease. Researchers believe it may help improve oxygen levels and reduce the need for blood transfusions. The trial enrolls 200 adults age…

This study is testing whether alendronate, a drug used for osteoporosis, can help treat bone damage (osteonecrosis) in adults with sickle cell disease. About 30 participants will take the drug for 6 months to see if it reduces pain and improves quality of life. The study also che…

This study tests a daily pill called rilzabrutinib in people aged 10 to 65 with sickle cell disease. The goal is to see if it can lower the number of painful vaso-occlusive crises (VOCs) over 52 weeks. About 192 participants will receive either the drug or a placebo. Those who co…

This study looks at the long-term safety and effectiveness of etavopivat, an experimental pill for sickle cell disease and thalassemia. It includes 480 adults, teens, and children who have already benefited from etavopivat in a previous study. Participants will take the drug for …

This early-stage study tests a single injection of CS-101, made from a patient's own gene-edited blood stem cells, for severe sickle cell disease. The goal is to see if it is safe and can reduce or eliminate painful vaso-occlusive crises. The study involves 5 participants aged 12…

This study tests a new drug called anumigilimab in 63 adults with sickle cell disease to see if it is safe. Participants receive weekly injections for 64 weeks, starting with a low dose that increases to their personal maximum. The goal is to reduce painful crises that often requ…

This study tests a new gene therapy for people aged 12 to 35 with severe sickle cell disease who do not have a matching bone marrow donor. The treatment uses the patient's own blood stem cells, which are modified in a lab to produce healthy red blood cells and reduce sickling. Th…

This study tests a once-daily pill called etavopivat in children aged 6 months to 18 years with sickle cell disease. The main goals are to check the medicine's safety and how it moves through the body. Participants take the pill every day for 96 weeks and have regular check-ups. …

This study is testing a new oral medication called osivelotor for people with sickle cell disease. The main goals are to see if the drug can safely increase hemoglobin levels and reduce the frequency of painful vaso-occlusive crises (VOCs). The trial will involve nearly 400 adult…

This study is testing a special skin sensor to see if it can accurately measure hydration levels in people with sickle cell disease. Researchers will compare the sensor's readings with standard dehydration tests in 30 participants, including some who are having a pain crisis. The…

This study is testing a blood test that can check for genetic disorders in unborn babies without the risks of traditional invasive tests. The test looks at fetal DNA found in the mother's blood as early as 9 weeks into pregnancy. Researchers aim to make this safe, accurate diagno…

This study looks at whether a portable ultrasound done at the bedside can help doctors diagnose acute chest syndrome in children and young adults with sickle cell disease. About 30 hospitalized patients aged 0-25 will take part. Researchers will compare the ultrasound results to …

This study looks at whether a liquid medicine called Xromi (hydroxycarbamide) can safely prevent painful complications of sickle cell disease in children aged 9 months to 2 years. About 180 children will take the medicine and be compared to a group of similar children who did not…

This study tests whether a daily medicine called hydroxyurea can prevent strokes in children with sickle cell anemia who are at high risk. About 220 children in Nigeria will receive low or moderate doses of the drug and be monitored for strokes or mini-strokes. The goal is to see…

This study tests whether dronabinol, a drug similar to a compound in marijuana, can reduce chronic pain and inflammation in adults with sickle cell disease. About 60 participants will receive either dronabinol or a placebo to see if it improves pain scores and quality of life. Th…

This study looks at whether a smartphone-based mindfulness program is practical and helpful for teens and young adults (ages 15-39) with sickle cell disease who have chronic pain and sleep problems. Participants use a guided mindfulness app for about 8 weeks. The goal is to see i…

This study tests whether children with sickle cell disease (ages 8 to 17) can safely take a daily stimulant medication (methylphenidate) for 4 weeks to improve attention, thinking, and school performance. Researchers will check for side effects and measure changes in cognitive sk…

This study explores whether writing and playing relaxing songs can help reduce anxiety in adolescents aged 11-18 with sickle cell disease. About 30 participants will try songwriting and report how it affects their anxiety, mood, and pain. The goal is to see if this creative activ…

This study tests whether an intravenous amino acid called citrulline can help shorten painful sickle cell crises in hospitalized children and young adults (ages 4-21). Participants receive either citrulline or a placebo for 16 hours, and researchers track how long the pain lasts …

This study looks at whether following a Mediterranean diet can help reduce chronic pain in people with sickle cell disease. Researchers will compare pain levels when participants eat a Mediterranean diet versus their usual diet. The study involves 30 adults with sickle cell disea…

This study tests whether using a virtual reality headset, along with standard pain medicine, can help adults with sickle cell disease feel less pain during a painful crisis in the emergency department. About 30 participants will be split into three groups to compare pain scores a…

This study tests a group training program called I-STRONG for teens with sickle cell disease who have chronic pain. The program teaches mind and body skills to help improve daily functioning and reduce pain. About 155 teens and their parents will take part to see if the program h…

This study looks at whether music therapy is a practical way to improve quality of life for teens and adults (14+) with sickle cell disease. 90 participants will be split into three groups: in-person music therapy, a mix of in-person and virtual music therapy, or virtual health e…

This study tests whether acupuncture can reduce chronic pain in people with sickle cell disease. Researchers will also use brain scans and other tests to understand how acupuncture works. The study involves 120 participants aged 14 to 80 who have had sickle cell pain in the past …

This early-stage study tests a drug called fostamatinib in 25 adults with stable sickle cell disease to see if it is safe and tolerable. Participants take the pill twice daily for up to 6 weeks, with regular checkups. The goal is to find a safer way to prevent blood clots in peop…

This study tests a stress management and mindfulness program for adults with sickle cell disease. The program includes relaxation exercises, meditation, and communication skills. Researchers want to see if it can improve quality of life and reduce pain's impact on daily activitie…

This study looks at whether adding a small amount of ketamine to usual pain medicine (like morphine) can better relieve severe pain from sickle cell disease in children and young adults ages 5 to 20. About 120 participants will be randomly assigned to receive either standard pain…

This study tests a program called SickleFit that combines exercise and nutrition coaching for adults aged 35 and older with sickle cell disease. The goal is to see if the program is safe, doable, and liked by participants. About 40 people will take part, with some getting the pro…

This study looks at whether using virtual reality (VR) can help reduce pain in children and young adults with sickle cell disease who are having a painful crisis. About 76 participants, ages 6 to 25, will either get standard pain treatment alone or standard treatment plus VR. Res…

This study tests whether a digital cognitive behavioral therapy (CBT) program, with or without personalized peer support, can reduce pain, opioid use, and hospital visits in 470 teens and young adults (ages 16–30) with sickle cell disease and chronic pain. Participants will be ra…

This study tests a small device that stimulates nerves in the ear to see if it can safely and effectively reduce pain in children with sickle cell disease during a pain crisis. About 20 children hospitalized for a sickle cell pain crisis will use the device. The goal is to see if…

This study tests a program designed to help teens and young adults with sickle cell disease build resilience and cope better. Researchers want to see if the program is practical and helpful for participants. The goal is to reduce depression, anxiety, and pain interference.

This study tests whether a web-based mobile app helps 272 teens and young adults with sickle cell disease learn to manage their symptoms and care for themselves. Participants use the app or standard care, and researchers measure changes in self-management behaviors. The goal is t…

This study is creating a large database of health information from up to 200,000 people with blood disorders like hemophilia, sickle cell disease, and thrombosis. Researchers will use this data to better understand these conditions, improve treatments, and guide public health dec…

This study follows 3,000 people with non-cancer blood disorders (like hemophilia, sickle cell disease, and clotting issues) to see how well treatments work over time and what side effects may occur. Researchers collect safety data on approved therapies to understand long-term ris…

This study is testing two different daily doses of zinc (25 mg and 40 mg) in 34 people with sickle cell disease, aged 15 to 40. The goal is to find out which dose is best at improving bone health markers and is safe to take. Results will help design a larger future study on zinc'…

This study measures how long red blood cells survive in people with sickle cell disease and other inherited blood disorders. Researchers will label participants' own red blood cells with biotin (a vitamin) and track them over time. The goal is to compare cell survival between peo…

This study tests a program that helps teens and young adults (ages 15-25) with sickle cell disease work with their doctors to choose the best treatment. About 68 patients, their families, and doctors will try a shared decision-making toolkit that includes virtual reality or stand…

This study is for people with sickle cell disease or other rare anemias. Researchers want to use advanced blood tests and genetic analysis to better understand each person's condition. The goal is to predict disease severity and guide personalized treatment. About 200 participant…

This study looks at whether sickle cell disease affects the number of eggs in the ovaries of girls aged 10 to 18. Researchers will measure a hormone called AMH in participants and compare it to healthy girls of the same age and stage of puberty. The goal is to understand how the …

This study tests a new communication tool called 'Let's Get REAL' that helps children and teens (ages 8-17) and their families talk together about stem cell transplant or cellular therapy decisions. About 60 youth and their parents will try the tool and report how easy and helpfu…

This study tests new brain imaging tools to measure oxygen flow in children with sickle cell disease. Researchers will compare these tools with standard MRI scans. The goal is to find better ways to decide when treatment is needed and how well it works. The study includes healthy…

This study looks at how blood flow and metabolism affect brain development in children with sickle cell anemia. Researchers will use MRI scans and a special mask to measure brain blood vessel function. They will compare 120 children with sickle cell disease to healthy children to…

This study looks at how sleep problems might affect memory and thinking in teenagers (ages 12-18) with sickle cell disease. Participants will use a simple home device to track sleep and complete thinking tests during a clinic visit. The goal is to understand the link between nigh…

This study talks to patients and parents of children with rare diseases like sickle cell disease to learn what they think and want to know about gene therapy. Researchers will use these conversations to build an online platform with easy-to-understand information and decision aid…

This study aims to find out why some people with sickle cell disease develop severe, disabling pain while others do not. Researchers will track 70 participants aged 15 to 40 over up to 48 months using surveys and pain tests. The goal is to improve pain management and guide future…

This study collects biological samples like blood, saliva, urine, and fat tissue from people with blood disorders (such as sickle cell disease and thalassemia) and healthy volunteers. The goal is to learn more about these conditions and improve future treatments. Participants age…

This study looks at how diet and eating habits affect the health of adults with sickle cell disease. Researchers will interview participants about their food intake and behaviors, collect blood and urine samples, and have them complete surveys. The goal is to understand links bet…

This study talks to parents and teens with sickle cell disease to learn their views on genetic research and gene therapy. Researchers will use surveys and interviews to measure knowledge and trust, then create a web tool to help families discuss treatment options with doctors. Th…

This early-phase study is testing a new drug called pociredir in 24 people with sickle cell disease. The main goal is to see how the drug is processed by the body when taken with or without food. Researchers will also check for any side effects. This study does not aim to treat t…

This study follows 750 people with sickle cell disease to see if curative treatments like stem cell transplants improve or harm the heart, lungs, and kidneys over time. Researchers will compare those who received curative therapy with those on standard care. The goal is to help p…

This study looks at why heart damage happens in people with sickle cell disease. Researchers will use PET scans to measure blood flow to the heart during rest and stress. They will compare results from 40 young adults with and without sickle cell disease to see if reduced blood f…

This study is creating new ways to test exercise in children with cystic fibrosis, sickle cell disease, or long COVID. Current tests are designed for healthy athletes and don't work well for kids with these conditions. Researchers will track about 240 children over several years …

This study follows 10,000 people with sickle cell disease over many years to learn how the disease affects organs like the kidneys and lungs as patients grow up. Researchers collect data during regular clinic visits and also study blood samples to understand why some people respo…

This study looks at 100 adults with sickle cell disease who are in intensive care for severe pain or breathing problems. Researchers want to see if the kidneys filter morphine too quickly, making pain relief less effective. By measuring kidney function and morphine levels, they h…

This study collects blood samples from 250 adults with sickle cell disease, sickle cell trait, or healthy red blood cells. Researchers will use the samples to test thousands of compounds, including existing FDA-approved drugs, to find new treatments for sickle cell disease. Parti…

This study collects blood, bone marrow, and other samples from people with non-cancerous blood diseases and their family members. Researchers will analyze genes to find causes of these diseases and why some people get sicker than others. The goal is to improve future diagnosis an…

This study aims to find genetic differences that explain why some people with sickle cell disease develop immune reactions to blood transfusions. Researchers will compare the genes of 50 participants who have had transfusions, some who reacted and some who did not. The goal is to…

This study tests a school readiness program for children aged 3.5 to 6.5 years with sickle cell disease. The goal is to see if the program is practical and helpful for improving learning skills. About 36 children and their caregivers will take part. The study compares the program…

This study looks at how early childhood experiences—like stress, social factors, and breastfeeding—affect the health of children with sickle cell disease. Researchers will enroll 20 mothers and their infants in Birmingham, Alabama. The goal is to learn whether a community-based b…

This study looks at how blood flow and oxygen use in the brain change as children grow, and how these changes might affect development. Researchers will use MRI scans and sometimes measure blood flow while participants breathe in extra carbon dioxide. The goal is to better unders…

This study looks at whether infants and young children with sickle cell disease (SCD) get enough protection from the standard measles vaccine. Researchers will measure antibody levels in 130 children with SCD after they receive their routine measles shots. The goal is to understa…