SICKLE CELL DISEASE

This study is testing if adding a drug called daratumumab before a bone marrow transplant from a matched sibling can safely cure sickle cell disease. It focuses on patients who have been excluded from this curative treatment because their blood type doesn't perfectly match their …

This early-stage trial is testing a new gene therapy called nula-cel for people with severe sickle cell disease. The treatment involves collecting a patient's own blood stem cells, modifying them in a lab to produce healthy hemoglobin, and then returning them to the patient. The …

This study is testing a stem cell transplant from a partially matched family donor as a potential cure for people with severe sickle cell disease. It aims to see if the donor cells successfully take hold in the patient's body by 60 days after the transplant. The trial is for pati…

This study is testing a modified stem cell transplant procedure for children and adults with severe sickle cell disease or beta-thalassemia. The goal is to cure these genetic blood disorders by replacing a patient's faulty blood-making cells with healthy ones from a half-matched …

This early-stage study is testing a potential one-time treatment for severe sickle cell disease. Doctors will collect a patient's own blood stem cells, edit a specific gene in the lab to boost protective fetal hemoglobin, and then return the cells. The goal is to see if this sing…

This study is testing a single treatment using the patient's own stem cells that have been genetically modified with CRISPR technology. The goal is to see if this one-time therapy can eliminate the need for lifelong blood transfusions in beta-thalassemia patients and stop painful…

This study aims to see if a new 21-valent pneumococcal vaccine (PCV21) is safe and helps the body build protective antibodies in children and teens with sickle cell disease. It will compare the new vaccine to an already licensed vaccine. About 100 participants aged 2 to 17 will r…

This study is testing a less toxic bone marrow transplant method for children with severe sickle cell disease. It uses lower doses of radiation and specific medications to try to reduce dangerous complications while still aiming to cure the disease. The research involves 100 chil…

This early-stage study is testing the safety of a new, one-time gene-editing treatment for people with severe sickle cell disease or beta-thalassemia. Doctors take a patient's own blood stem cells, edit a specific gene in the lab to boost healthy fetal hemoglobin, and then return…

This program provides expanded access to a special method of preparing stem cells for transplant. It is designed for patients with severe immune deficiencies, sickle cell disease, and other conditions where a standard transplant carries high risks. The process aims to reduce a da…

This early-stage study is testing the safety of an oral drug called panobinostat in adults with sickle cell disease who haven't responded well to standard treatment. Researchers want to find the highest dose patients can tolerate without severe side effects. The drug aims to incr…

This early-stage study is testing a new drug called ITU512 for sickle cell disease. The main goal is to see if it's safe and how it works in the body, particularly its ability to increase a protective type of hemoglobin that can reduce sickling of red blood cells. The study will …

This study is testing an investigational drug called tebapivat to see if it can improve anemia in people with sickle cell disease. About 56 participants will be randomly assigned to receive either tebapivat or a placebo (an inactive substance) to compare their effects. The main g…

This study is testing whether an investigational drug called rilzabrutinib can reduce the frequency of painful vaso-occlusive crises (VOCs) in people with sickle cell disease. The trial will involve 192 participants aged 10 to 65 who experience 2-10 painful crises per year. Parti…

This study is testing if taking zinc supplements can help improve bone health in people with sickle cell disease. It will enroll 34 participants, aged 15-40, to compare the effects of two different daily doses of zinc over 12 weeks. The goal is to find the best dose to use in a l…

This study aims to see if a daily oral medication called hydroxyurea can effectively prevent first strokes in children with sickle cell disease in Nigeria. It will involve 220 children aged 5-12 who are at high risk for stroke. The goal is to confirm this treatment works well in …

This study is testing a new gene therapy called DREAM01 for people with severe sickle cell disease who don't have a matched sibling donor for a bone marrow transplant. Doctors take a patient's own blood stem cells, genetically modify them in a lab to produce healthy hemoglobin, a…

This study tests whether regular heart scans and iron-reducing treatment can prevent heart damage and reduce hospital visits in adults with sickle cell disease. Participants will receive heart imaging every 3 months for a year along with medications to remove excess iron. The goa…

This study is testing the safety of an experimental drug called anumigilimab in adults with sickle cell disease. Over 64 weeks, researchers will monitor side effects while participants receive injections of the drug or a placebo. The goal is to see if the treatment is safe and if…

This study is testing a daily oral medication called etavopivat in adolescents with sickle cell disease. Researchers want to understand how safe the drug is, how it moves through the body, and whether it helps reduce complications like pain crises. About 50 participants will take…

This study is checking the safety and real-world effectiveness of a liquid medicine called Xromi for very young children with sickle cell disease. It will follow about 180 children under 2 years old for two years, comparing those taking the medicine to similar children who are no…

This early-stage study is testing if a drug called motixafortide is safe for adults with severe sickle cell disease. Researchers want to see if the drug helps the body release more stem cells into the bloodstream so they can be collected. These collected cells could potentially b…

This is an early safety study to see if a pill called fostamatinib is safe and tolerable for adults with stable sickle cell disease. Up to 25 participants will take the drug at home for 6 weeks, with visits every 2 weeks to check for side effects. The main goal is to find a safe …

This study is testing whether a mobile phone app, combined with educational booklets, can help adults with sickle cell disease better manage their condition. The goal is to see if using these tools leads to fewer trips to the emergency room and hospital. Researchers will compare …

This study is testing a new oral medication called osivelotor for people with sickle cell disease. The main goals are to see if the pill can safely increase hemoglobin levels and reduce the number of painful crises (VOCs) that require medical care. The trial involves nearly 400 p…

This study is testing a donor stem cell transplant to treat people with severe inherited blood disorders like sickle cell disease and thalassemia, who currently depend on regular blood transfusions. The goal is to replace the patient's faulty blood-making system with a healthy do…

This study is testing whether an investigational drug called crizanlizumab can reduce the frequency of painful vaso-occlusive crises in people with sickle cell disease. It will involve about 315 adolescents and adults who experience frequent pain crises, comparing the drug agains…

This study is testing whether losartan, a blood pressure medication, can reduce or stabilize heart scarring in people with sickle cell disease. Researchers will follow 24 patients aged 6 and older for one year to see if the drug is safe and effective. The goal is to prevent heart…

This program provides eligible sickle cell disease patients with access to an investigational drug called crizanlizumab. The goal is to help prevent or reduce the frequency of painful vaso-occlusive crises (VOCs) in patients aged 12 and older who continue to experience these cris…

This study will follow patients for 15 years after they received an experimental gene-editing treatment (BEAM-101) for sickle cell disease or similar blood disorders. The main goal is to monitor their long-term health and safety, and see how well the treatment continues to work o…

This large, late-stage study is testing whether a new oral drug called etavopivat can reduce the number of painful blockages in blood vessels (called vaso-occlusive crises) in people aged 12 and older with sickle cell disease. Participants will be randomly assigned to receive eit…

This study allows sickle cell disease patients who benefited from crizanlizumab treatment in previous Novartis trials to continue receiving the medication. It's designed for patients who have completed earlier studies but don't yet have access to commercially available treatment.…

This study is testing a new daily pill called etavopivat to see if it can lower the high risk of stroke in children with sickle cell disease. It will involve about 27 children aged 12-16 who have fast blood flow in their brain arteries, a key warning sign for stroke. The main goa…

This study is testing if giving a medicine called hydroxyurea twice a day is safe and effective for children with sickle cell disease. About 50 children, aged 9 months to 11 years, will take the medicine this way for one year. Researchers will check how the body processes the dru…

This early-stage study is testing a new drug called DISC-3405 in 24 adults with sickle cell disease. The main goal is to check if the drug is safe and how the body processes it. Researchers will measure changes in blood markers to see if the drug affects the disease.

This study aims to understand the long-term safety and effectiveness of etavopivat, an investigational drug for sickle cell disease and thalassemia. It will follow about 480 adults, adolescents, and children who have already taken etavopivat in a previous study and benefited from…

This study is testing if a weekly pill called alendronate is safe and tolerable for adults with sickle cell disease who have a painful bone condition called osteonecrosis (bone death). Researchers will enroll about 30 participants to take the pill for 6 months to see if it helps …

This early-stage study is testing the safety of a new gene editing treatment for severe sickle cell disease. Researchers will collect a patient's own blood stem cells, edit them in the lab using CRISPR technology to boost protective fetal hemoglobin, and then infuse them back. Th…

This study is testing a small, non-invasive skin sensor to measure hydration in people with sickle cell disease. The goal is to see if the sensor can help doctors give the right amount of IV fluids during painful crises, avoiding the risks of giving too much or too little. Resear…

This study is evaluating a new, non-invasive blood test to diagnose serious inherited genetic disorders in unborn babies. The test analyzes tiny bits of the baby's DNA found in the mother's blood, aiming to provide a safe and accurate diagnosis as early as 9 weeks into pregnancy.…

This study aims to find out if giving regular blood transfusions during pregnancy can prevent health crises and pregnancy complications in women with sickle cell disease. Researchers will compare two groups: one receiving scheduled transfusions and another receiving standard care…

This study is testing a 4-week telehealth program called C-READY, designed to help teenagers with sickle cell disease learn the skills they need to manage their own health as they transition to adult care. The program involves one-on-one video sessions with a therapist to work on…

This study is testing whether a special group training program can help teens with sickle cell disease better manage their chronic pain. The program, delivered via video calls, teaches skills to strengthen the mind and body. Researchers want to see if this training, when added to…

This study is testing a small, non-invasive device placed on the ear to see if stimulating nerves there can help reduce pain in children hospitalized with sickle cell disease. The goal is to find a safe alternative to strong pain medications like opioids. Researchers will measure…

This study is testing if a daily ADHD medication called methylphenidate is safe and practical for children with sickle cell disease who have trouble with focus, memory, and schoolwork. Over 4 weeks, 30 children will take the medication once a day to see if they can stick with it …

This study is testing whether a special mobile app can help teenagers and young adults with sickle cell disease better manage their condition. Researchers want to see if using the app teaches helpful self-care skills, reduces pain interference, and improves quality of life. The s…

This study is testing whether using a virtual reality (VR) headset can help reduce pain for adults with sickle cell disease who come to the emergency room with a severe pain crisis. It will add VR sessions to the usual pain medications to see if it makes patients feel better. Res…

This study is testing whether acupuncture can help manage the chronic pain that often comes with sickle cell disease. Researchers will compare traditional needle acupuncture to a needle-free laser version in about 120 participants. They will also use brain scans and other tests t…

This study is testing whether adding virtual reality (VR) to standard pain treatment helps children and young adults with sickle cell disease during painful crises. Participants receive either standard care alone or standard care plus a 15-minute VR session designed to distract f…

This study is testing whether music therapy is a practical way to help improve quality of life for teens and adults with sickle cell disease. Researchers will compare three different programs: in-person music therapy, a mix of in-person and virtual music therapy, and a standard h…

This study is testing if a therapy app on a phone can help teens and young adults with sickle cell disease manage their chronic pain. It will see if adding one-on-one support from a peer who also has the disease makes the app more helpful. The goal is to reduce pain, lower the ne…

This study is testing if adding a low dose of the drug ketamine to standard opioid pain medicine works better for children and young adults with sickle cell disease during a severe pain crisis. Researchers will compare pain levels, opioid use, and hospital stays between two group…

This study is testing whether a resilience-building program can help teenagers and young adults with sickle cell disease better manage their condition. The program focuses on providing mental health support to help participants cope with depression, anxiety, and pain. Researchers…

This study is testing a virtual summer program designed to help preschool children with sickle cell disease get ready for school. Researchers want to see if an 8-week online program for parents and children improves school readiness skills like early math, reading, and self-contr…

This study is testing whether a smartphone-based mindfulness program is practical and helpful for teens and young adults with sickle cell disease. Participants will use the app for about 8 weeks to try to manage their chronic pain and sleep problems. The main goal is to see if pe…

This study is testing whether dronabinol, a medication derived from cannabis, can help reduce chronic pain and inflammation in adults with sickle cell disease. Researchers will compare dronabinol to a placebo (inactive pill) in 60 participants to see if it improves pain levels an…

This study is testing whether a songwriting activity can help teenagers with sickle cell disease feel less anxious and better manage their emotions. Researchers want to see if participants find the creative process practical, enjoyable, and helpful for their mental well-being. Th…

This study is testing whether a 6-week group program teaching stress management and mindfulness skills can improve quality of life for people with sickle cell disease. 50 adults will be randomly assigned to either start the program right away or wait 6 weeks. The program includes…

This study aims to understand why standard morphine doses often fail to relieve severe pain in adults with sickle cell disease during a crisis. Researchers will measure how quickly patients' kidneys filter morphine to see if faster filtration leads to underdosing. The goal is to …

This study is collecting biological samples like saliva, blood, urine, and tissue from volunteers to help scientists better understand blood disorders like sickle cell disease, thalassemia, and malaria. Researchers need samples from both people with these conditions and healthy i…

This study aims to understand how blood flow and oxygen delivery affect brain development in children and young adults with sickle cell disease. Researchers will compare 120 participants—some with sickle cell and some healthy—using MRI scans to measure brain blood flow while brea…

This study aims to better understand how blood flow and oxygen delivery affect brain development in children and young adults. Researchers will use MRI scans to measure brain activity in healthy participants and in those with conditions like sickle cell disease or a history of EC…

This study aims to understand why some people with sickle cell disease develop immune responses after blood transfusions. Researchers will compare the genes of 50 patients who have had transfusion reactions with those who haven't. The goal is to identify genetic factors that migh…

This study aims to collect blood, saliva, and health information from people with blood diseases and their family members to build a research library. The goal is to discover unknown genetic causes and understand why disease severity varies between people. This is a research-only…

This study aims to develop better ways to measure exercise and fitness in children with chronic health conditions. Researchers will follow 240 children and teens with cystic fibrosis, sickle cell disease, or long-term effects from COVID-19, along with healthy children, for 3-4 ye…

This study aims to understand the long-term effects of curative therapies like stem cell transplants and gene therapy on people with sickle cell disease. Researchers will track 750 participants to see if these cures improve or worsen heart, lung, and kidney health over time compa…

This is a long-term observational study that will follow 10,000 people with sickle cell disease from birth through their entire lives. The goal is to collect detailed health information over time to understand how the disease progresses, how it damages organs, and how treatments …

This study aims to find out what factors predict whether someone with sickle cell disease will develop severe, long-lasting pain. Researchers will follow 70 participants, aged 15-40, for up to four years, using surveys and pain sensitivity tests. The goal is to gather information…

This study aims to understand if sickle cell disease affects ovarian health in young females. Researchers will compare hormone levels in 440 girls with sickle cell to healthy peers of the same age and puberty stage. The goal is to learn how the disease and its treatments might in…

This study is testing a new way to help teenagers and young adults with sickle cell disease understand their treatment options and make decisions with their doctors and families. Researchers are comparing a virtual reality education tool to standard printed and video materials to…

This study aims to understand how sleep and breathing problems might affect brain function, mood, and pain in teenagers with sickle cell disease. Researchers will mail sleep-monitoring devices to 65 participants (ages 12-18) to use at home for three nights. Later, participants wi…

This study aims to understand if providing community-based breastfeeding support to mothers of infants with sickle cell disease can improve health outcomes. Researchers will compare 20 mother-infant pairs, with some receiving support and others being observed. They will track how…

This study aims to understand how diet, eating habits, and nutrition affect the overall health of adults living with sickle cell disease. Researchers will observe 80 participants, collecting information through interviews, surveys, and lab tests to look for connections between wh…

This study is testing a new family communication tool called 'Let's Get REAL' to help children and teenagers be more involved in decisions about their stem cell or cellular therapy treatments. Researchers will work with 24 young patients (ages 8-17) and their parents to see if th…

This study aims to understand why people with sickle cell disease often develop heart problems. Researchers will compare how blood flows to the heart during stress in three groups: young adults with sickle cell and heart stiffness, those with sickle cell without stiffness, and he…

This study aims to develop better tools to understand and predict the course of rare inherited blood disorders like sickle cell disease. Researchers will combine detailed blood cell analysis, genetic testing, and patient health information to create personalized profiles for 200 …

This study aims to collect long-term safety and effectiveness data on treatments for various non-cancerous blood disorders, including hemophilia, von Willebrand disease, and platelet disorders. It will follow up to 3,000 participants for at least 15 years to see how newer, FDA-ap…

This study aims to understand how long red blood cells survive in people with sickle cell disease, thalassemia, and other inherited blood disorders. Researchers will label a small sample of a participant's own blood cells with a safe vitamin (biotin) and track them over several m…

This study aims to find out if children with sickle cell disease develop good protection after receiving their measles vaccines. Researchers will check blood samples from about 130 children before and after their routine measles shots to measure their immune response. The goal is…

This is an early safety study to see how the body absorbs and processes a new tablet called pociredir in people with sickle cell disease. It will enroll about 24 participants to test the drug when taken on an empty stomach and after a meal. The main goal is to measure the drug's …

This small pilot study aims to test new brain imaging methods to monitor sickle cell disease. Researchers want to see if advanced scans can measure how well oxygen reaches the brain and how treatments affect this process. The study involves children with sickle cell disease, incl…

This study aims to understand the attitudes and concerns of parents and adolescents with sickle cell disease regarding genetic research and clinical trials. Researchers will conduct interviews and surveys with families to learn about their knowledge, trust in healthcare providers…

This study aims to find new drug treatments for sickle cell disease by collecting small blood samples from volunteers. Researchers will test these samples against thousands of existing compounds to see if any can prevent the red blood cell damage that causes the disease. The stud…

This study aims to understand what patients and families think and need to know when considering gene therapy for rare diseases like sickle cell. Researchers will interview patients, parents, and healthcare workers about their experiences and concerns. Using what they learn, the …