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. The treatment involves a stem cell transplant using the patient'…

This study tests a bone marrow transplant from a healthy brother or sister to cure sickle cell disease in children. The approach uses lower doses of radiation and drugs to reduce side effects while still aiming for a high cure rate. About 100 children with severe sickle cell dise…

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

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 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, reducing complications like severe anemia. Up to 12 chil…

This study tests a new gene therapy called nula-cel for people with severe sickle cell disease. The treatment modifies a patient's own blood stem cells to produce healthy hemoglobin instead of sickle hemoglobin. About 15 participants aged 12 to 40 will receive the therapy to see …

This early-stage trial tests a one-time infusion of a person's own blood stem cells that have been corrected using CRISPR gene editing. The goal is to reduce painful blockages in blood vessels caused by sickle cell disease. The study involves 9 people aged 12 to 35 with severe fo…

This study looks at whether checking the heart early for iron buildup in people with sickle cell disease can help prevent heart problems and reduce hospital visits. About 100 adults with sickle cell disease and high iron levels will get regular heart scans and treatment. The goal…

This study tests a single dose of a CRISPR-modified stem cell therapy (CTX001) in 26 people with transfusion-dependent β-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, edited to produce fetal hemoglobin, aiming to reduce or elimi…

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

This expanded access program offers a special stem cell filtering technique (CD34+ selection) to patients with severe blood disorders like sickle cell disease and immune deficiencies. The goal is to lower the risk of graft-versus-host disease, a serious complication where donor c…

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 tests a stem cell transplant using a half-matched family donor for people with severe sickle cell disease. Participants receive low-dose radiation and chemotherapy before the transplant, followed by drugs to prevent rejection. The main goal is to see if the donor cells…

This study tests whether a mobile app that provides easy-to-understand sickle cell disease guidelines can help adults reduce emergency room visits, hospital stays, and infusion center visits. About 287 adults with sickle cell disease will use the app along with a booklet, and the…

This study tests a new way to prepare people with sickle cell disease or beta-thalassemia for a stem cell transplant from a half-matched donor. Before the transplant, participants receive drugs to lower their immune system, which may help prevent serious side effects like graft f…

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

This study looks at whether giving regular blood transfusions to pregnant women with sickle cell disease can reduce serious health problems for both mother and baby. About 50 women will be randomly assigned to receive either repeated transfusions or standard care during pregnancy…

This study is testing whether giving hydroxycarbamide (a medicine that helps prevent sickle cell crises) twice a day works better than the usual once-daily dose in children aged 9 months to 11 years with sickle cell disease. About 50 children will take the medicine twice daily fo…

This study tests a stem cell transplant from a donor to treat people with severe blood disorders like sickle cell disease and thalassemia. About 62 participants will receive the transplant and be monitored for side effects like graft-versus-host disease. The goal is to control th…

This study follows 50 people with sickle cell disease who received a gene therapy called BEAM-101. Researchers will check for side effects and monitor health for up to 15 years after treatment. The goal is to understand long-term safety and how well the therapy controls the disea…

This early-stage study tests a new drug, ITU512, in healthy volunteers and people with sickle cell disease. The goal is to see if it safely boosts fetal hemoglobin, a natural protein that can reduce painful sickle cell complications. The trial first checks safety and dosing in he…

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 early-stage study tests whether a single infusion of a patient's own gene-edited stem cells is safe for people with severe sickle cell disease. The goal is to boost fetal hemoglobin, which may reduce painful crises and other complications. Up to 25 participants aged 18 to 24…

This study tests whether adding the drug ruxolitinib to a standard stem cell transplant regimen can lower the chance of transplant failure in children and young adults with severe sickle cell disease. About 24 participants aged 12-45 will receive a transplant from a half-matched …

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 daily pill called etavopivat in 27 children (ages 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 and reduce stroke risk. Participants take the pill for one year, with an option to …

This early-stage study tests a new drug, DISC-3405, in 24 adults with sickle cell disease. The main goal is to check safety and how the body handles the drug at different doses. Researchers will also measure changes in blood counts to see if the drug might help control the diseas…

This study offers ongoing access to crizanlizumab for people with sickle cell disease who have already completed a Novartis-sponsored trial and are benefiting from the drug. About 130 participants will continue receiving treatment to manage their condition. The goal is to provide…

This study tests whether a low dose of tocilizumab, a drug used for arthritis and COVID-19, can help people with sickle cell disease who develop acute chest syndrome—a serious lung complication. Researchers will measure how the drug affects oxygen levels and other outcomes in 200…

This study tests whether hydroxyurea can prevent first strokes in 220 high-risk children with sickle cell anemia in Nigeria. Participants take low or moderate doses of the drug and are monitored for strokes or mini-strokes. The goal is to see if this approach works in real-world …

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 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 tests an investigational drug called crizanlizumab in people aged 12 and older with sickle cell disease who have frequent painful crises. The goal is to see if the drug can reduce the number of these crises compared to a placebo. Participants may also continue taking h…

This study tests whether losartan, a common 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 daily for one year. Researchers will use heart scans and exercis…

This study is testing if a weekly pill called alendronate is safe and can help adults with sickle cell disease who have a painful bone condition called osteonecrosis. Researchers will give the pill to about 30 participants for 6 months to see if it reduces pain and improves bone …

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 if adding a drug called daratumumab before a bone marrow transplant from a matched sibling can safely help children and young adults with severe sickle cell disease who are usually excluded from this treatment. These patients are excluded because their immun…

This study aims to see if a blood test from the mother can accurately diagnose genetic disorders in the fetus, such as sickle cell disease or cystic fibrosis, as early as 9 weeks into pregnancy. The test uses fetal DNA found in the mother's blood, avoiding the miscarriage risk of…

This study is testing a special skin sensor that measures water levels in the skin of people with sickle cell disease. Dehydration can trigger painful crises, so finding a quick, easy way to check hydration could help guide treatment. About 30 participants will be enrolled to see…

This study aims to improve how doctors diagnose sickle cell disease and other rare anemias by combining advanced blood tests with genetic analysis. Researchers will enroll 200 people with confirmed or suspected rare anemia to see if a new test can predict disease severity. The go…

This study looks at whether a bedside ultrasound (POCUS) can reliably diagnose acute chest syndrome in children and young adults with sickle cell disease. Researchers will compare ultrasound results to standard chest X-rays. The goal is to see if this faster, radiation-free tool …

This study looks at whether a liquid medicine called Xromi (hydroxycarbamide) can safely prevent painful blood vessel blockages in children aged 9 months to 2 years with sickle cell disease. About 180 children will take the medicine and be compared to a group of similar children …

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 early-phase study tests the safety of a drug called fostamatinib in 25 adults with stable sickle cell disease. Participants take the pill twice daily for up to 6 weeks, with regular checkups to monitor side effects. The goal is to see if the drug is safe enough for larger st…

This study tests whether dronabinol, a drug similar to cannabis, 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. The goal is to fi…

This study looks at whether songwriting can help reduce anxiety in adolescents aged 11-18 with sickle cell disease. Participants will write and play relaxing songs, and researchers will measure changes in anxiety, mood, and pain. The goal is to see if this creative activity is a …

This study tests a group training program called I-STRONG for teens with sickle cell disease who have chronic pain. The program teaches skills to strengthen both mind and body, with the goal of improving everyday functioning and reducing pain symptoms. About 155 teens and their p…

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 the Mediterranean diet versus their usual diet. The study involves 30 adults with sickle cell dis…

This study is testing whether adding a low dose of ketamine to standard pain medicine (like morphine) can better relieve severe pain from sickle cell disease in children and young adults ages 5 to 20. Participants will be randomly assigned to receive either standard care plus ket…

This study looks at whether music therapy is a practical way to help teens and adults (ages 14+) with sickle cell disease feel better. Ninety people will be split into three groups: in-person music therapy, a mix of in-person and virtual music therapy, or health education. The ma…

This study tests whether using virtual reality (VR) alongside standard pain medicine can better reduce pain in children and young adults with sickle cell disease during a painful crisis. About 76 participants, ages 6 to 25, will either get standard care or standard care plus VR. …

This study tests whether using a virtual reality headset can help reduce pain for adults with sickle cell disease who are in the emergency room for a painful crisis. About 30 participants will receive either standard care alone or standard care plus a VR experience. The goal is t…

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 called SickleFit that combines exercise and nutrition advice for adults aged 35 and older with sickle cell disease. The goal is to see if the program is safe, doable, and helpful for improving health and quality of life. About 40 participants will be ra…

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 120 participants aged 14 to 80 who have chronic pain or recent pain crises. It aims to…

This study tests whether a stress management and mindfulness program can improve quality of life for adults with sickle cell disease. Participants will learn techniques like relaxation exercises, mindfulness meditation, and assertive communication. The study aims to reduce pain i…

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 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 pain lasts and …

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 tests whether children with sickle cell disease (SCD) can safely take a daily stimulant drug called methylphenidate for 4 weeks to improve attention, thinking, and schoolwork. About 72 children ages 8 to 17 with SCD and thinking or attention problems will take the drug…

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 or sleep problems. Participants use guided mindfulness exercises on their phone for about 8 weeks. Th…

This study tests new brain imaging tools to measure oxygen and blood flow in children with sickle cell disease. Researchers will compare these scans with standard MRI to see if they can better track how well treatments like gene therapy or transfusions are working. The goal is to…

This study follows 750 people with sickle cell disease who have received curative therapies (like stem cell transplants) to see how their heart, lung, and kidney health changes over time compared to those on standard treatment. The goal is to understand if these therapies improve…

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 early-stage study is testing a new drug called pociredir in 24 adults with sickle cell disease. Researchers want to see how the drug moves through the body when taken with or without food. The goal is to gather safety and dosing information, not to treat the disease.

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 talks to parents and teens with sickle cell disease to learn what they think about genetic research and gene therapy. Researchers will use surveys and interviews to understand concerns and expectations. The goal is to create a web tool that helps families and doctors d…

This study is creating new, kid-friendly exercise tests for children with cystic fibrosis, sickle cell disease, or long-term effects of COVID-19. Current tests are designed for healthy athletes and don't work well for these children. Researchers will track 240 kids over 3-4 years…

This study tests a telehealth program called C-READY designed to help teens with sickle cell disease improve thinking skills and learn to manage their own health. About 120 participants aged 10-18 will be randomly assigned to the program or usual care. The goal is to see if the p…

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 at-home sleep monitor and complete thinking tests during a regular clinic visit. The goal is to better understand the link be…

This study tests a new communication tool called 'Let's Get REAL' that helps children and teens (ages 8-17) and their families talk about stem cell transplant and cellular therapy decisions. About 60 youth and their parents will try the tool, and researchers will measure if it is…

This study collects biological samples like blood, saliva, and urine from people with blood disorders (such as sickle cell disease and thalassemia) and healthy volunteers. The goal is to help researchers better understand these conditions and related diseases like malaria. Partic…

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 faster kidney function causes morphine to leave the body too quickly, making pain relief less effective. By measuring kidney filtrat…

This study looks at why the heart gets damaged in people with sickle cell disease. Researchers will use special 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 reduc…

This study looks at how zinc supplements affect bone health in people with sickle cell disease. About 34 participants aged 15 to 40 will take either 25 or 40 mg of zinc daily for 12 weeks. The goal is to find which dose works best and is safe, so it can be used in a larger future…

This study follows 10,000 people with sickle cell disease over many years to understand how treatments and genetics affect long-term health, especially organ function in adulthood. Participants provide data during regular clinic visits and may join a sub-study on how their genes …

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 8 doctors will take part. The program includes virtual reality or standard education mat…

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 tho…

This study talks to patients and parents of children with rare diseases like sickle cell disease to learn what they believe and want to know about gene therapy. Researchers will use these insights to build an online tool with educational content and decision aids. No treatment is…

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

This study looks at how early childhood experiences—like breastfeeding, stress, and social factors—affect children with sickle cell disease. Researchers will work with 20 mothers of infants with sickle cell disease in Birmingham, Alabama, to see if a community-based breastfeeding…

This study looks at how blood flow and metabolism changes in the brain affect development in children with sickle cell anemia. Researchers will use MRI scans and a special mask to measure brain blood vessel function. The study includes 120 children aged 4-21, both with sickle cel…

This study collects blood, bone marrow, and other samples from people with non-cancerous blood diseases and their family members. Researchers will analyze DNA and other markers to find genetic causes and factors that affect disease severity and treatment response. The goal is to …

This study looks at whether girls with sickle cell disease have lower levels of a hormone called AMH, which is a marker of egg supply in the ovaries. Researchers will compare AMH levels in girls with sickle cell disease to those in healthy girls of the same age and stage of puber…

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 track symptoms like pain and mood. The goal is to understand ho…

This study is creating a huge 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 and improve treatments. Anyone seen at a particip…

This early-stage study tests whether the drug motixafortide is safe for people with sickle cell disease and whether it can increase the number of stem cells in the blood for collection. The stem cells could be used for future transplants or gene therapy. About 15 adults with seve…

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 follows 3,000 people with non-cancer blood disorders (like hemophilia, sickle cell disease, and clotting issues) to see how well treatments work and what side effects occur over time. Researchers will collect safety data, including allergic reactions, infections, and o…

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…

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 people who have had these reactions with those who have not. The goal is to better understand…