Hemoglobinopathy

This study tests a new, gentler stem cell transplant for children with sickle cell disease using a combination of drugs and low-dose radiation. The goal is to cure the disease with fewer side effects than standard transplants. About 100 children will receive a transplant from a h…

This early-stage trial tests a gene-editing treatment for people with severe sickle cell disease or beta-thalassemia. The approach uses the patient's own blood stem cells, which are edited to boost fetal hemoglobin—a healthy type that can replace the defective hemoglobin. Up to 1…

This study tests a new 21-valent pneumococcal vaccine (PCV21) in 100 children and teens with sickle cell disease, aged 2 to 17. The goal is to see if it's safe and helps the body make antibodies better than the current 20-valent vaccine. Participants get one shot and are monitore…

This early-stage trial is testing a new gene therapy called nula-cel for people with severe sickle cell disease. The therapy modifies a patient's own blood stem cells to produce normal hemoglobin instead of sickle hemoglobin. About 15 participants aged 12 to 40 will receive the t…

This clinical trial tests a partial stem cell transplant for people with severe sickle cell disease or beta-thalassemia who need regular blood transfusions. The transplant uses a lower-intensity conditioning regimen with alemtuzumab, low-dose radiation, and abatacept to help dono…

This early-stage trial is testing whether a skin gel made from curcumin (a compound in turmeric) can be safely absorbed by adults with stable sickle cell disease. Researchers will apply the gel to the forearms twice a week for six weeks and monitor for side effects. The goal is t…

This study follows about 665 people with myelodysplastic syndromes, beta-thalassemia, or myelofibrosis who have already taken luspatercept in earlier trials. Researchers want to see how safe the drug is over many years by tracking side effects, disease progression, and survival. …

This study tests a new type of stem cell transplant for children with severe blood disorders like sickle cell disease or aplastic anemia. The transplant uses stem cells from a parent or other half-matched family donor, which are specially processed to remove certain immune cells.…

This 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 symptoms. The trial is in early phases (1 and 2) and involves about 161 participants. I…

This phase 2 trial tests luspatercept (Reblozyl) in adults and adolescents with alpha-thalassemia, a genetic blood disorder that causes anemia. The goal is to see if the drug can reduce the need for blood transfusions and raise hemoglobin levels. About 189 participants will recei…

This study tests whether a single dose of tocilizumab, an anti-inflammatory drug, can help children and adults with sickle cell disease recover faster from acute chest syndrome—a serious lung condition. Participants receive either the drug or a placebo, and researchers measure ho…

This Phase 3 trial tests whether the oral drug rilzabrutinib can lower the number of painful vaso-occlusive crises in people with sickle cell disease. About 192 participants aged 10 to 65 will receive either the drug or a placebo for 52 weeks. The main goal is to see if the drug …

This study tests a drug called crizanlizumab in people aged 12 and older with sickle cell disease who have frequent pain 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 their usual treatment…

This global phase 3 trial is testing whether a daily pill called etavopivat can reduce the number of painful sickle cell crises in adolescents and adults with sickle cell disease. About 408 participants will be randomly assigned to receive either etavopivat or a placebo for aroun…

This study tests a new drug called BMS-986470 in healthy volunteers and people with sickle cell disease. The goal is to check its safety, how the body processes it, and whether it might help control the disease. The trial is in early phases and involves about 184 participants.

This early-phase study is testing a new drug called 131I-apamistamab, which combines an antibody with radioactive iodine, to prepare people with severe sickle cell disease for a bone marrow transplant. The goal is to find the smallest effective dose that allows the donor stem cel…

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

This study offers continued access to crizanlizumab, a drug that helps reduce painful crises in sickle cell disease, for 130 patients who benefited from it in earlier Novartis trials. Participants must have completed a prior study and lack other access to the drug. The main goal …

This study tests whether adding the drug ruxolitinib to a standard stem cell transplant can prevent graft failure in children and young adults with severe sickle cell disease. Up to 24 participants will receive the drug during conditioning and be followed for two years after tran…

This early-stage study tests a new drug, DISC-3405, in 24 adults with sickle cell disease to see if it is safe and how the body processes it. Participants will receive increasing doses of the drug, and researchers will monitor side effects and changes in blood counts. The goal is…

This phase 2 trial tests a stem cell transplant from a donor for people with high-risk sickle cell disease, thalassemia, and other red blood cell disorders. Participants receive drugs and low-dose radiation before the transplant to help the new cells take hold. The study will tra…

This study is testing a stem cell transplant from half-matched (haploidentical) donors for people with sickle cell disease or beta-thalassemia. Before the transplant, patients receive drugs to lower their immune system, which may reduce the risk of serious side effects like graft…

This study is testing a drug called luspatercept in adults with a severe blood disorder called beta-thalassemia who need regular blood transfusions. The goal is to see if the drug can reduce how many transfusions they need and improve their hemoglobin levels. Researchers will fol…

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

This early-stage trial is testing the safety of fostamatinib, a drug already used for arthritis, in 25 adults with stable sickle cell disease. Participants take the pill twice daily for up to 6 weeks, with close monitoring for side effects. The goal is to see if the drug is safe …

This study tests whether empagliflozin, a drug already approved for kidney disease in diabetes, can slow kidney damage in people with sickle cell anemia. About 20 adults with sickle cell disease and early signs of kidney trouble will take the drug for 6 months. Researchers will m…

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

This early-phase study tests a new approach to stem cell transplants for sickle cell disease. It uses a half-matched (haploidentical) donor, which could help more patients find a donor. Before the transplant, patients receive two cycles of immune-suppressing therapy to prepare th…

This early-phase study tests the safety of a one-time gene-edited stem cell treatment for people with severe sickle cell disease. The therapy uses CRISPR technology to boost fetal hemoglobin, which may reduce painful crises and other complications. Up to 25 participants aged 18 t…

This study tests a stem cell transplant for children with sickle cell disease who have a matched family donor whose blood type is incompatible. The goal is to see if a gentle conditioning regimen using alemtuzumab, low-dose radiation, and sirolimus can safely allow the transplant…

This study is testing whether giving blood transfusions that match the patient's own RH genes can prevent the body from making harmful antibodies. About 35 people with sickle cell disease who need regular transfusions will receive specially matched donor blood. The goal is to see…

This study tests a once-daily pill called etavopivat in children (6 months to 18 years) with sickle cell disease. The main goals are to check safety and how the drug works in the body, and to see if it can raise hemoglobin levels and reduce painful crises. About 95 children will …

This study tests whether testicular tissue frozen before puberty can be transplanted back into adult men to restore sperm production. Five men who had fertility-threatening treatments as children will receive their own preserved tissue. The goal is to see if sperm can be found in…

This study tests whether adding memory immune cells (CD45RO) to a stem cell transplant can help children fight off dangerous viral and fungal infections after the procedure. The transplant uses donor stem cells that have been stripped of certain cells to prevent graft-versus-host…

This study tests a new gene therapy for adults with transfusion-dependent beta-thalassemia, a severe blood disorder. Doctors take the patient's own blood stem cells, add a healthy gene to make normal hemoglobin, and return the cells. The goal is to reduce or stop the need for reg…

This study tests a one-time treatment using CRISPR to fix the sickle cell gene in a patient's own blood stem cells. Up to 9 people aged 12 to 35 with severe sickle cell disease will receive the edited cells. The goal is to reduce painful blockages in blood vessels and improve qua…

This program provides crizanlizumab, a drug that may help reduce pain crises, to people with sickle cell disease who have no other treatment options. It is for patients who cannot join a clinical trial. The goal is to offer potential benefit when standard therapies are not availa…

This study is checking whether unlicensed cord blood units are safe to use for stem cell transplants in children and adults with blood disorders. Researchers will closely watch for any infusion-related problems. The goal is to see if these units can be a safe option for patients …

This study tests whether alendronate, a drug used for osteoporosis, can help adults with sickle cell disease who have bone damage (osteonecrosis). Thirty participants will take a weekly pill for 24 weeks. The main goals are to see if the treatment is feasible and safe, and to mea…

This phase 2 trial is testing whether a single low dose of tocilizumab, a drug that blocks inflammation, can help people with sickle cell disease who develop acute chest syndrome—a serious lung complication. Researchers will enroll 200 patients aged 12 and older and track their o…

This study tests a special stem cell transplant for children and adults with blood cancers like leukemia. The transplant uses stem cells that have had certain immune cells removed to lower the risk of graft-versus-host disease, a serious complication. About 70 participants will r…

This study is testing a milder type of stem cell transplant for people with non-cancerous blood, immune, or metabolic disorders. The goal is to safely get donor cells to grow in the patient's body while reducing harsh side effects. About 220 participants will receive a reduced-in…

This study tests whether the drug lusutrombopag can help restore platelet levels faster in patients who have had a stem cell transplant for blood disorders. About 45 adults will receive the drug after transplant, and doctors will check platelet counts at 21 days. The goal is to i…

This study looks at whether a team-based care program can help pregnant women with sickle cell disease (SCD) in a non-academic hospital in Ghana. The goal is to reduce the number of mothers who die during pregnancy or childbirth. About 198 women will take part, and their care wil…

This study tests whether a mobile health app and educational booklets can help adults with sickle cell disease reduce emergency room visits and hospital stays. Researchers will compare the app-plus-booklet approach to booklets alone in 287 participants. The goal is to see if givi…

This study tests a one-time treatment called CTX001, which uses CRISPR gene editing to modify a patient's own stem cells to produce more fetal hemoglobin. The goal is to reduce or eliminate the need for blood transfusions in people with transfusion-dependent beta-thalassemia or s…

This study tests an oral drug called osivelotor in about 389 adults and adolescents with sickle cell disease. The goal is to see if it can raise hemoglobin levels and reduce the number of painful vaso-occlusive crises. Participants will take the pill daily, and researchers will m…

This study tests a cord blood transplant in children and young adults (up to age 21) with life-threatening blood cancers or non-cancer blood disorders who do not have a matched family donor. The goal is to see if this approach lowers the risk of death from treatment one year afte…

This phase 2 trial tests a combination of three chemotherapy drugs (treosulfan, fludarabine, thiotepa) plus an immune-suppressing antibody before a donor stem cell transplant in up to 40 people under 50 with non-cancerous blood disorders. The goal is to see if this milder conditi…

This trial tests a new gene therapy called DREAM01 for people with severe sickle cell disease who cannot find a matching bone marrow donor. The therapy uses the patient's own blood stem cells, modified to produce healthy hemoglobin and block the sickle cell gene. Up to 15 partici…

This Phase 2 study tests an oral drug called AND017 in 64 people with β-thalassemia, a blood disorder that often requires regular transfusions. Half the participants get the drug, half get a placebo, and everyone continues standard care. The goal is to see if AND017 is safe and c…

This expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic disea…

This expanded access program offers a special stem cell purification technique (CD34+ selection) for patients undergoing stem cell transplants. The method removes many T-cells from the donated stem cells, which lowers the risk of a serious complication called graft-versus-host di…

This study tests whether giving regular blood transfusions during pregnancy can reduce sickle cell complications and pregnancy-related problems in women with sickle cell disease. About 50 pregnant women will be randomly assigned to receive transfusions or standard care. The goal …

This study looks at growth problems in children with thalassemia, a blood disorder. Researchers will test a program that includes health education, nutrition plans, exercise, and support to help kids grow better. The goal is to improve height, weight, and overall well-being in 36…

This early-phase trial is testing a new treatment called CS-101 for severe sickle cell disease. It uses a person's own blood stem cells, which are gene-edited in a lab to correct the disease, then given back as a one-time infusion. The study will enroll 5 people aged 12 to 35 and…

This study offers a second stem cell transplant to people with sickle cell disease or beta-thalassemia whose first transplant is losing its effect. Participants receive donor stem cells along with drugs to help their body accept them. The goal is to restore donor cell levels and …

This long-term follow-up study tracks 50 patients with sickle cell disease who received the experimental gene therapy BEAM-101. The goal is to monitor safety, side effects, and overall health for up to 15 years after treatment. Participants will have regular check-ups to see how …

This phase II trial is testing a stem cell transplant from a half-matched donor for people with severe sickle cell disease. Participants receive chemotherapy, radiation, and immune-suppressing drugs before the transplant to help the new cells take hold. The main goal is to see if…

This study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…

This Phase 2 trial tests whether adding low-dose thalidomide to luspatercept can reduce the number of blood transfusions needed by adults with transfusion-dependent β-thalassemia. 78 participants will be randomly assigned to receive either the combination or luspatercept alone fo…

This early-stage trial is testing 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 control the disease. Participants take capsules three times a week for 12 weeks, and r…

This study tests a drug called luspatercept in children aged 6 to 18 with beta-thalassemia, a blood disorder that often requires regular transfusions. The goal is to see if the drug is safe and can reduce the need for transfusions or raise hemoglobin levels. The trial is still re…

This study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…

This study is for adults with blood disorders who are getting a stem cell transplant from a matched donor. Researchers want to see if giving a special potato starch along with a drug that lowers iron (deferasirox) can help prevent serious complications like graft-versus-host dise…

This program offers extra stem cells from a half-matched family donor to patients who are getting a cord blood transplant for serious blood cancers or immune disorders. The goal is to help the bone marrow recover more quickly while the cord blood cells take over permanently. It i…

This study tests whether early heart screening and treatment for iron overload in people with sickle cell disease can prevent heart problems and reduce hospital stays. About 100 adults with sickle cell disease and high iron levels will receive regular heart imaging and follow-up …

This trial tests an oral drug called SP-420 in about 90 adults with transfusion-dependent β-thalassemia or low-risk myelodysplastic syndromes (MDS). The goal is to see how well SP-420 removes excess iron from the liver and other organs, and whether it is safe. Participants take t…

This study tests whether giving a drug called daratumumab before a stem cell transplant can prevent a serious complication called pure red blood cell aplasia in children with sickle cell disease. The transplant uses a gentle, low-intensity approach and a matched sibling donor. Ab…

This study is testing whether giving hydroxyurea twice a day works better than the usual once-daily dose for children with sickle cell disease. About 50 children aged 9 months to 11 years will take the drug for 12 months. Researchers will measure how the drug behaves in the body …

This study tests a personalized way to dose hydroxyurea for children with sickle cell anemia. Instead of using weight alone, doctors will measure how each child's body processes the drug to find the best dose. The goal is to see if this approach reduces damage to the brain, kidne…

This study tests a new medicine called etavopivat in 27 children aged 12-16 with sickle cell disease who are at higher risk for stroke. Participants take one daily pill for a year, and doctors use ultrasound to measure blood flow in the brain. The goal is to see if etavopivat can…

This study follows 5 people with β-thalassemia who received CS-101, a gene-edited stem cell therapy, in an earlier trial. Researchers will monitor them for years to see if the treatment remains safe and allows them to stay free from blood transfusions. The goal is to understand w…

This early study tests a new type of stem cell transplant called 'super transplant' for children aged 7-12 with severe thalassemia. Unlike standard transplants, this one does not require chemotherapy beforehand and aims to avoid graft-versus-host disease. Only 5 participants will…

This study tests a mobile health information system to help clinics in Uganda screen newborns for sickle cell disease and coordinate their care. Researchers will compare clinics using the new system to those using standard screening. The goal is to see if the system helps more ba…

This study is working on a new blood test for pregnant women that can check for serious genetic conditions like cystic fibrosis, spinal muscular atrophy, and sickle cell disease. Researchers will collect blood samples from 4,000 pregnant women who are at higher risk of passing on…

This study is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

This study looks at whether a bedside ultrasound (POCUS) can help doctors quickly diagnose acute chest syndrome in children and young adults with sickle cell disease. Researchers will enroll 30 hospitalized patients aged 0-25 to see if the ultrasound is practical and reliable com…

This study is testing a special skin sensor to see if it can accurately measure hydration levels in people with sickle cell disease. Dehydration can trigger painful crises, so a quick, non-invasive way to check hydration could help guide treatment. The study will involve 30 parti…

This study is testing a new type of MRI scan called eASL that measures blood flow in the brain without needing a contrast injection. It will include 50 children with conditions like Moya-moya or sickle cell disease that affect brain arteries. The goal is to see if eASL gives clea…

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 part, with some receiving Xromi and a comparison group of similar child…

This study tests whether hydroxyurea, a drug already used for sickle cell disease, can prevent strokes in 220 high-risk children in Nigeria. Children with sickle cell anemia and abnormal blood flow in the brain will receive low then moderate doses of hydroxyurea. The goal is to s…

This study tests whether the birth control shot Depo-Provera can reduce painful sickle cell episodes in women. Sixty-five women with sickle cell disease who have frequent pain will be monitored for 3 months without the shot, then for 3 months after receiving it. Researchers will …

This study tests whether using virtual reality (VR) goggles during a sickle cell pain crisis can help children and young adults feel less pain. Participants will either get standard pain treatment alone or standard treatment plus a calming VR underwater experience. Researchers wi…

This study tests an investigational drug called CSL889 in 70 adults and adolescents with sickle cell disease who are having a painful vaso-occlusive crisis. The goal is to see if the drug can safely shorten the crisis and reduce the need for strong pain medicines. Participants wi…

This study tests a program called SCThrive, which includes virtual group sessions and a mobile app, to help 260 teens and young adults (ages 13-21) with sickle cell disease take charge of their health. Participants will complete surveys before, after, and 3 months after the 8-wee…

This study tests a 12-week exercise and nutrition program called SickleFit in 40 adults aged 35 and older with sickle cell disease. The goal is to see if the program is safe, doable, and liked by participants. It does not aim to cure the disease but to improve fitness and quality…

This study tests a program called PRISM that aims to help teens and young adults with sickle cell disease build resilience and cope better. Researchers will check if the program is easy to use and liked by participants. They will also see if it helps reduce depression, anxiety, a…

This study is testing whether a type of talk therapy called acceptance and commitment therapy (ACT) can help Black teenagers and young adults with sickle cell disease feel less stressed, anxious, and depressed. About 66 youth and their parents will join group therapy sessions and…

This study tests whether a special cooling cap can prevent hair loss in children receiving chemotherapy for non-cancerous conditions or solid tumors. The cap is worn before, during, and after chemo to keep the scalp cool. Researchers want to see if it is safe and tolerable for 40…

This pilot study at St. Jude Children's Research Hospital is testing whether a daily dose of extended-release methylphenidate (Ritalin) can safely improve attention, thinking, and school performance in children aged 8 to 17 with sickle cell disease. The trial will enroll 72 parti…

This early-stage study tests a new drug called APG-5918 in healthy volunteers and people with anemia (low red blood cells). The main goal is to check safety and how the body handles the drug, while also seeing if it can raise hemoglobin levels. About 105 participants will take pa…

This study tests whether a natural substance called citrulline, given through an IV, can help children and teens with sickle cell disease recover faster from severe pain episodes. About 99 hospitalized participants aged 4 to 21 will receive either citrulline or a placebo for 16 h…

This study looks at whether following a Mediterranean diet can help reduce chronic pain in people with sickle cell disease. About 30 adults with sickle cell disease who have pain most days will try the diet and report their pain levels. Researchers will also check changes in gut …

This study looks at whether giving less IV fluid to people with sickle cell disease during a painful crisis works just as well as giving more fluid, while possibly causing fewer side effects like fluid overload. About 394 adults with sickle cell disease will be randomly assigned …

This study is testing whether a low dose of ketamine, given alongside standard pain medicine, can better control severe pain from sickle cell disease in children and young adults aged 5 to 20. Participants will receive either ketamine or a placebo infusion, and researchers will m…

This study tests whether a stress management and mindfulness program can improve quality of life for adults with sickle cell disease. The program includes techniques from cognitive-behavioral therapy, relaxation exercises, and mindfulness meditation. Researchers will enroll 50 pa…

This study tests a telehealth program called C-READY that helps teens with sickle cell disease improve thinking skills like planning and problem-solving. The goal is to prepare them to manage their own health care as they grow up. About 120 teens aged 10-18 will take part, and th…

This study explores whether writing and playing songs with a music therapist can help teens (ages 11-18) with sickle cell disease feel less anxious and more resilient. Thirty participants will try the songwriting program and report changes in anxiety, mood, and pain-related stres…

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

This study is testing whether music therapy can help improve quality of life for people with sickle cell disease. About 90 teens and adults will be split into three groups: in-person music therapy, a mix of in-person and virtual music therapy, or health education sessions. The ma…

This study tests a small device that gently stimulates nerves in the ear to see if it can help relieve pain in children with sickle cell disease during a pain crisis. About 20 children hospitalized for pain will try the device. The main goal is to see if kids can tolerate it and …

This study tests a new liquid drug called HBI-002, which contains carbon monoxide, in 9 people with sickle cell disease aged 14 to 55. Participants take the drug daily for 14 days to see if it is safe and helps with symptoms. The goal is to find a better way to manage this painfu…

This study tests whether a digital cognitive behavioral therapy (CBT) program, with or without a peer health coach, can reduce pain and opioid use in 470 teens and young adults (ages 16–30) with sickle cell disease and chronic pain. Participants use an app to track pain and mood,…

This study tests whether using virtual reality (VR) alongside standard care can reduce pain for adults with sickle cell disease who are in the emergency department with a painful crisis. About 30 participants will be split into three groups to compare pain scores and comfort. The…

This study tests whether a web-based mobile app can help 272 teens and young adults with sickle cell disease learn to manage their symptoms and care for themselves. Participants use the app to track symptoms and get tips, and researchers measure how well they take charge of their…

This study tests if oral ketamine can help control severe pain from sickle-cell crises in teens and young adults (ages 12–24). Participants receive the drug every 8 hours for 48 hours alongside standard pain care. The goal is to find better ways to manage pain and reduce the need…

This study tests a group telehealth program called I-STRONG for teens with sickle cell disease who have chronic pain. The program combines mind-body skills with movement training to help teens cope better and reduce fear of pain. Researchers will enroll 155 teens aged 12-18 to se…

This study looks at whether acupuncture can reduce 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 chronic pain or recent pain crises.

This study tests whether dronabinol, a drug made from 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 and quality of life. The goal is to find a new…

This study collects blood stem cells and immune cells from healthy volunteers and patients with primary immune deficiencies or blood disorders. The cells are used in the lab to develop new gene and cell therapies. Up to 850 adults aged 18-70 will participate. The goal is to advan…

This study provides ongoing check-ups for people who received a donor stem cell transplant at the NIH at least three years ago. Researchers will monitor for late side effects, disease return, and overall health. Participants will have yearly visits that may include blood tests, b…

This study follows up to 3,500 people with sickle cell disease to learn more about how the condition progresses and what complications arise. Participants receive their usual medical care outside the hospital and visit the clinic for check-ups and research tests. No experimental …

This study looks at how sleep problems might affect thinking, memory, and brain health in 65 people aged 12–25 with sickle cell disease. Participants will use a home sleep monitor and complete thinking tests during a clinic visit. The goal is to understand links between nighttime…

This study looks at how blood transfusions affect tiny molecules called peroxides in very premature babies. These molecules can damage cells if they build up. Researchers will measure peroxide levels before and after transfusions, and also use a special light device to check oxyg…

This study collects blood, bone marrow, urine, and tissue samples from up to 6,000 people with solid tumors, blood cancers, or non-cancerous blood disorders, as well as from their healthy family members who are stem cell donors. The samples are stored and used in research to bett…

This study looks for better ways to detect lung inflammation and infections early in people who have received a stem cell transplant from a donor. About 40 participants aged 5 to 70 will get regular lung scans, breathing tests, blood draws, and a procedure where a tube collects f…

This study collects biological samples like blood, saliva, and urine from 300 adults aged 18-70, both with and without blood disorders such as sickle cell disease and thalassemia. The goal is to use these samples for research to better understand these conditions and related dise…

This study looks at how the drug hydroxyurea affects women with sickle cell disease and their babies during pregnancy and breastfeeding. Researchers will review medical records from 200 women to measure drug exposure and compare health outcomes. The goal is to better understand t…

This study collects blood samples and health information from 375 people with non-cancerous blood disorders like aplastic anemia, sickle cell disease, or thalassemia who are receiving a stem cell transplant or gene therapy. The goal is to create a biobank that helps researchers u…

This study aims to determine how common sickle cell trait and sickle cell disease are across Uganda by analyzing up to one million blood samples. Researchers want to create a detailed map of where the condition is most prevalent, which could help improve healthcare planning. The …

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 treatments. About 68 patients, their caregivers, and 8 doctors will take part. The program includes virtual reality or standard education …

This study collects blood, bone marrow, and other samples along with health information from up to 1,716 people with non-cancerous blood diseases and their family members. Researchers will analyze the participants' genes to find new genetic causes of these conditions and understa…

This study collects blood samples from 250 adults with sickle cell disease, sickle cell trait, or healthy red blood cells. Researchers will use these samples to screen thousands of compounds, including FDA-approved drugs, to find ones that stop red blood cells from sickling. The …

This study looks at how differences in the PKLR gene might change sickle cell disease symptoms. Researchers will collect blood samples from 800 adults of African descent, with and without sickle cell disease, to measure certain proteins and energy molecules in red blood cells. Th…

This large study will follow 480 children with sickle cell disease to compare two treatment approaches: a stem cell transplant from a matched family donor versus standard disease-modifying therapies. The goal is to see which option leads to better quality of life and thinking ski…

This study will follow 10,000 people with sickle cell disease for many years, tracking their health during regular clinic visits. Researchers want to learn how the disease damages organs over time and how treatments like hydroxyurea might help prevent that damage. A smaller part …

This study looks at whether girls with sickle cell disease have lower levels of a hormone called AMH, which is linked to egg supply, compared to healthy girls of the same age and puberty stage. Researchers will also check how treatments and pain crises affect these levels. About …

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. The goal is to see if the tool is easy to use and helpful for families. About 60 …

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 that can be collected from their blood. These stem cells could be used in future gene therapies or transplants. The study i…

This study is observing 400 people aged 12 and older who have noncirrhotic portal hypertension (NCPH) or are at risk for it. NCPH is a liver condition that increases blood pressure in the liver's blood vessels without causing cirrhosis, and often has no early symptoms. Researcher…

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 reactions with those who have not. The goal is to identify specific…

This study looks at adults with sickle cell disease to see how their heart, brain, kidneys, liver, and lungs change after treatments meant to cure the disease. Researchers will compare people getting curative therapy (like a bone marrow transplant or gene therapy) with those who …

This study tests ways to increase the use of hydroxyurea, a medicine that helps manage sickle cell disease, in Nigeria. Researchers will train healthcare workers and provide tools to help them prescribe and monitor the drug. The goal is to see if these strategies lead to more pat…

This study interviews patients and families who have experience with gene therapy for rare pediatric diseases, including sickle cell disease. Researchers want to understand their beliefs, attitudes, and what information they need. The goal is to create an online platform with edu…

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—some with sickle cell disease and some without—to se…

This study aims to find out why some people with sickle cell disease develop severe, disabling chronic pain while others do not. Researchers will enroll 70 participants aged 15 to 40 and use surveys, virtual visits, and in-person pain sensitivity tests to track pain and related o…

This study collects bone marrow that is normally thrown away during surgery from people with and without blood disorders like sickle cell disease. Researchers will use these samples in the lab to learn how to better manipulate stem cells for gene therapy and to study cell health.…

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 better educational tools and build trust bet…

This study tests whether a virtual 8-week school readiness program helps preschool children (ages 3.5–6.5) with sickle cell disease improve social skills, early reading, and math. About 36 children and their caregivers will take part. The goal is to see if the program is practica…

This study is a large registry that collects health information from up to 200,000 people with blood disorders such as hemophilia, thrombosis, and sickle cell disease. Participants are seen at Hemophilia Treatment Centers across the U.S. The goal is to gather real-world data to h…

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, and collect blood and urine samples. The goal is to understand links between nutrition and symptoms …

This study is building a special collection of blood and urine samples from 800 adults with sickle cell disease or sickle cell trait, along with some healthy volunteers. Researchers will use these samples and medical records to learn how kidney disease starts and gets worse in pe…

This study is working to create better exercise tests for children with cystic fibrosis, sickle cell disease, or long COVID. Current tests are designed for healthy athletes and may not be safe or useful for these kids. Researchers will track 240 children over 3-4 years, measuring…

This study tests two daily doses of zinc (25 mg and 40 mg) in 34 adults with sickle cell disease to see which dose works best for bone health. Participants take zinc for 12 weeks, and researchers measure changes in bone formation and breakdown markers. The goal is to pick the rig…

This study looks at how gut bacteria and the immune system interact in children and adults receiving stem cell transplants or CAR-T therapy. Researchers will collect stool and blood samples over time to find patterns linked to complications like infections or graft-versus-host di…

This study looks at how quickly the kidneys clear morphine in sickle cell patients having a painful crisis. Researchers will measure kidney function and morphine levels in 100 intensive care patients to see if higher doses are needed for effective pain relief. The goal is to pers…

This study aims to measure how long red blood cells survive in people with sickle cell disease, thalassemia, and other inherited blood disorders. Researchers will take a blood sample, label the red cells with biotin (a vitamin), and infuse them back into the participant. Over up …

This study looks at 350 adults with transfusion-dependent beta-thalassemia to find differences between those who have atrial fibrillation (a type of irregular heartbeat) and those who do not. Researchers will compare clinical tests, lab results, and imaging to better understand t…

This study looks at how early childhood experiences—like breastfeeding, stress, and social factors—affect children with sickle cell disease. Researchers will test a community-based breastfeeding support program for mothers and track health outcomes like asthma and chest pain epis…

The SAFE study is a long-term research project following 250 adolescents and young adults with sickle cell anemia. It aims to understand how the medicine hydroxyurea affects growth, puberty, and the ability to have children. Researchers will also compare pregnancy outcomes betwee…

This study follows 750 people with sickle cell disease to see how curative therapies affect the heart, lungs, and kidneys over time. Researchers will compare those who received a stem cell transplant with those on standard treatment. The goal is to understand whether curative the…

This study looks at whether children with sickle cell disease (SCD) develop enough antibodies after getting the measles vaccine. Researchers will enroll 130 children aged 6 months to 6 years who are due for their measles shots. Blood samples will be taken a few weeks after each d…

This study follows 102 women who had fertility preservation before cancer treatment to see what happens long-term. Researchers want to know if they get pregnant naturally, use stored eggs or tissue, or decide not to have children. The goal is to improve support and care for femal…

This study is collecting long-term safety and effectiveness data on treatments for various blood disorders, including hemophilia, sickle cell disease, and clotting disorders. It aims to enroll 3000 people of any age with these conditions. Researchers will monitor side effects, co…

This study is creating a collection of blood samples from 2,000 people with various blood disorders. The samples will be stored and used for future research approved by an ethics board. No treatments or drugs are being tested, and participants continue their normal care.

This study looks at gut bacteria in 300 people with colorectal cancer to see if certain bacteria are linked to cancer spread. Researchers will compare stool and tissue samples from patients whose cancer has spread versus those whose cancer has not. The goal is to find bacterial p…

This study tests new brain imaging tools to measure oxygen flow in children with sickle cell disease. Researchers will compare healthy kids, untreated patients, and those who had gene therapy. The goal is to find better ways to decide when treatment is needed and how well it work…

This study follows 29 children with sickle cell disease who receive MEOPA gas for pain during a crisis. Researchers will check their vitamin B12 levels before and after treatment, and again at 7 days and 1 month. The goal is to see how often MEOPA causes a B12 deficiency and whet…

This study looks at families who carry a change in the DDX41 gene, which may raise the risk of blood cancers like leukemia. Researchers will collect health questionnaires and saliva samples from up to 910 people to track who develops blood problems and when. The goal is to better…

This study looks at using a needle placed into the bone marrow (intraosseous infusion) to give fluids and medicines quickly to critically ill patients with blood diseases. Researchers will enroll 52 adults aged 18-65 to see how often the first attempt works and how safe it is. Th…

This study looks at why people with transfusion-dependent beta thalassemia have trouble exercising. Researchers will compare 36 patients to healthy volunteers using bike tests, blood samples, and questionnaires. The goal is to find the physical and mental factors that limit their…

This study is for people with sickle cell disease or other rare anemias. Researchers want to use advanced genetic and blood tests to better understand each person's condition. The goal is to make diagnosis more precise so that treatments can be tailored to each patient. About 200…

This study is a registry that tracks patients with blood diseases who receive stem cell transplants at a hospital on the Qinghai-Tibet Plateau. Researchers will collect information on recovery, complications, and costs over time. The goal is to understand how high altitude affect…

This study looks at whether a quick ultrasound of the thigh muscle can detect muscle loss (sarcopenia) in people who have finished cancer treatment. Researchers will compare the ultrasound results with a standard whole-body scan (DEXA) in 55 adults aged 18-74 with certain cancers…

This study aims to better understand how the spleen filters and responds to abnormal blood cells in various diseases. Researchers will collect spleen tissue and blood from 100 adults undergoing planned splenectomy. By perfusing the spleen in the lab, they hope to uncover its role…

This study aims to learn how blood flow and oxygen use in the brain change as children grow, especially in those with sickle cell disease who are at risk for stroke. Researchers will use MRI scans and have participants breathe in small amounts of carbon dioxide to see how well th…

This study looks at fetal cells that remain in a mother's blood after pregnancy, called fetal microchimeric cells, in women with chronic skin ulcers like venous or diabetic ulcers. Researchers will analyze these cells' genetic activity to see if they might help with wound healing…

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 have participants breathe carbon dioxide, which helps measure how well brain blood vessels expand. The goal is to u…

This study aims to learn more about rare disorders that affect how the body processes chemicals called pyrimidines and purines. These disorders can cause problems in the brain, blood, kidneys, and immune system, ranging from mild to life-threatening. Researchers will compare test…