Hemoglobinopathy

This study tests a single-dose gene therapy for people with transfusion-dependent beta-thalassemia, a blood disorder that requires regular red blood cell transfusions. The therapy uses a patient's own stem cells, modified with a virus to produce healthy hemoglobin, and then infus…

This study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells…

This study tests whether a combination of two drugs, thalidomide and hydroxyurea, can safely boost fetal hemoglobin levels in people with beta thalassemia major. The goal is to improve overall hemoglobin levels and reduce the need for regular blood transfusions. The trial include…

This study tests a drug called mitapivat in children aged 1 to 17 with alpha- or beta-thalassemia who need regular blood transfusions. The goal is to see if mitapivat can reduce the number of transfusions needed compared to a placebo. The study involves 54 participants and lasts …

This Phase 3 trial tests whether the drug mitapivat can reduce the need for blood transfusions in people with sickle cell disease. About 159 participants aged 12 and older will receive either mitapivat or a placebo daily for a year. The main goal is to see if more people can avoi…

This Phase 3 trial tests whether the drug mitapivat can improve anemia in children aged 1 to 17 with alpha- or beta-thalassemia who do not need regular blood transfusions. About 45 participants will receive either mitapivat or a placebo for 24 weeks. The main goal is to see if mi…

This study tests whether removing a specific type of immune cell (naive T cells) from a donor's stem cell graft can prevent graft-versus-host disease (GVHD), a serious complication where donor cells attack the recipient's body. The trial enrolls people with non-malignant blood di…

This study tests a stem cell transplant from a half-matched family donor for children and young adults with sickle cell disease. The goal is to control the disease while reducing serious side effects like graft-versus-host disease. Up to 45 participants will receive a special dru…

This early-stage study tests whether high-dose Vitamin C is safe for people with two rare inherited blood disorders—Pyruvate Kinase Deficiency (PKD) and a form of G6PD deficiency. Both conditions cause red blood cells to break down too quickly, leading to anemia. The study will e…

This early-stage study tests a one-time gene therapy (CS-206) in 5 teenagers with severe sickle cell disease. The treatment uses the patient's own blood stem cells, modified with a gene-editing tool, to help produce healthy red blood cells. The goal is to reduce painful blockages…

This study tests a new drug called SNH-119014 in 30 adults with a type of thalassemia that does not require regular blood transfusions. The goal is to see if the drug is safe and helps improve anemia. Participants will be monitored for side effects and how their body responds to …

This study compares transfusions using red blood cells from umbilical cord blood versus standard adult donor blood in extremely premature infants. About 176 babies born before 28 weeks or weighing under 1000 grams will be enrolled. The goal is to see if cord blood reduces the ris…

This study tests a single dose of CS-101, a gene therapy, in 20 people aged 12-35 with β-thalassemia major, a severe blood disorder requiring regular transfusions. The goal is to see if it is safe and can help patients produce enough healthy red blood cells to stop needing transf…

This study will test 120 babies in Côte d'Ivoire for sickle cell disease using a simple point-of-care test. Babies who test positive will start comprehensive care early, including medicines and vaccines. Researchers want to see if this approach reduces serious health problems and…

This early-stage study tests a new treatment using special immune cells (CAR-T cells) designed to reduce harmful antibodies that can prevent or damage bone marrow transplants. About 18 people with blood diseases who have these antibodies will receive the cells in increasing doses…

This early study tests a gene therapy called KL003 in just 3 people with severe sickle cell disease. The therapy uses the patient's own stem cells, modified to produce healthy hemoglobin, to reduce painful crises and the need for transfusions. The main goals are to see if the tre…

This study tests whether a high dose of plerixafor can safely collect enough stem cells from people with sickle cell disease for a future transplant. Twelve patients aged 18-25 will receive the drug and have their stem cells collected through a process called apheresis. The goal …

This study tests whether starting tube feeding right after a stem cell transplant can reduce severe gut graft-versus-host disease (GVHD) in people with blood cancers or disorders. About 112 participants will be randomly assigned to either standard care or early tube feeding. The …

This study tests whether red blood cells processed to be low in oxygen (hypoxic) last longer in the body than standard cells for people with sickle cell anemia who need regular transfusions. About 48 participants aged 7 and older will receive both types of blood in a crossover de…

This study tests a drug called SIL-8301 (senicapoc) in 105 adults with sickle cell disease who have low hemoglobin but few pain crises. Participants take the drug or a placebo daily for 24 weeks, along with their usual hydroxyurea. The main goal is to see if the drug raises hemog…

This early-phase trial will test a single dose of a new drug called YOLT-204 in 18 children and teens with transfusion-dependent β-thalassemia or sickle cell disease. The goal is to see if it is safe and can raise fetal hemoglobin levels enough to reduce the need for blood transf…

This Phase 3 trial tests whether two antioxidant supplements, N-acetyl cysteine and alpha-lipoic acid, can improve blood vessel health and reduce oxidative stress in adults with beta thalassemia. Sixty-six participants will take one of the supplements or a placebo alongside their…

This study will test whether a 4-week daily music therapy app can reduce chronic pain and improve quality of life in 25 youth with sickle cell disease. Participants will either use the app or receive standard educational materials. Researchers will also look at changes in immune …

This study explores whether using a virtual reality headset can help reduce pain for adults with sickle cell disease during a painful crisis. Participants will try different types of headset experiences—like watching a 2D video or exploring a 3D world—during their infusion center…

This study tests whether motivational interviewing, a type of counseling, can help adults with beta-thalassemia major follow their iron chelation therapy more consistently. Forty-five participants in Oman will receive three one-on-one sessions with a nurse over six to eight weeks…

This study tests a virtual reality app called Amani designed to help adults with sickle cell disease manage pain, emotional distress, and social isolation. Researchers will enroll 40 adults to see if the app is easy to use and helpful for improving coping skills and peer support.…

This study tests whether minocycline, an antibiotic that reduces brain inflammation, can safely prevent or reverse memory and thinking problems in adults with sickle cell disease. About 30 participants will take either a low dose, high dose, or placebo for one year. Researchers w…

This study will test whether eating flaxseed, a source of omega-3 fatty acids, can help reduce pain and improve daily life for children with sickle cell disease. Twenty children aged 5 to 18 will take part. The main goal is to see if it's possible for them to eat flaxseed regular…

This study tests if iloprost, a drug that widens blood vessels and reduces inflammation, can help adults with sickle cell disease during painful vaso-occlusive crises. About 144 participants hospitalized for a crisis will receive either iloprost or a placebo, plus standard care. …

This study tests whether giving ketamine within an hour of pain onset can prevent hospital admission for children and young adults with sickle cell disease. 90 participants aged 6-24 will receive either ketamine or a placebo by mouth. If admitted, they can still receive standard …

This study tests whether adding lidocaine, a numbing medicine, to standard pain treatment can lower the amount of strong opioids needed during severe sickle cell pain crises. About 104 adults with sickle cell disease who are in the intensive care unit for a pain crisis will take …

This study tests whether awake prone positioning (lying on the stomach while awake) can help sickle cell patients with severe acute chest syndrome in the ICU. The approach aims to improve oxygen levels and breathing by evening out air flow in the lungs. Fifteen adults will be enr…

This study tests whether a computer simulation can help pharmacists and pharmacy students better understand the pain struggles of sickle cell disease patients. About 200 healthcare professionals will use a point-of-view simulation and then complete surveys to see if their misconc…

This study tests whether shining a special light on the head (transcranial photobiomodulation) can improve oxygen use and thinking skills in children with sickle cell disease. The brain needs steady oxygen, but sickle cell can reduce blood flow. Researchers will randomly assign 6…

This study will look at medical records of 350 children with sickle cell disease in France who had serious bacterial infections between 2020 and 2025. The goal is to find out which bacteria are causing these infections today, especially with new vaccines available. The results co…

This study will test if a special oral health education program can improve the oral hygiene and quality of life of 45 children aged 8-10 with β-thalassemia major. The program teaches proper brushing and dental care. Researchers will measure changes in oral cleanliness and how ch…

This study looks at how genetic differences affect the way Nigerian sickle cell patients respond to hydroxyurea, a common treatment. Researchers will monitor 100 patients to build a computer model that predicts the best dose for each person. The goal is to reduce side effects and…

This study tests a 90-minute virtual training for healthcare providers that teaches them to see each patient's unique perspective and communicate more clearly. The goal is to improve how doctors talk to children with sickle cell disease. Thirty providers from three hospitals will…

This study will measure levels of a small molecule called microRNA-155 in 100 people who need regular blood transfusions, mostly due to beta thalassemia. The goal is to see if higher levels of this molecule are linked to the formation of harmful antibodies against donated blood. …

This study looks at children with beta-thalassemia major, a genetic blood disorder requiring lifelong transfusions. Researchers will measure growth and check for risk factors like iron overload that may stunt development. No new treatment is given—just careful observation of 85 c…

This study looks at how different types of anemia—like iron deficiency or thalassemia—can change HbA1c levels, a common test for diabetes, in people who do not have diabetes. Researchers will compare HbA1c results between 100 non-diabetic adults with various anemias and a healthy…

This study looks at how thalassemia patients in Greece feel day-to-day while taking a standard iron-removing medicine called deferasirox. Researchers will survey 150 adults to measure their physical and mental well-being. The goal is to understand real-world quality of life, not …

This study will measure certain hormones in 80 boys aged 10 to 18 who have transfusion-dependent beta thalassemia. The goal is to see how iron buildup from repeated blood transfusions affects the glands that control puberty and fertility. By checking levels of inhibin B, INSL3, a…

This study will review medical records of 25 children with sickle cell disease to see how often they experience painful vaso-occlusive crises and what complications arise. Researchers hope to identify risk factors that could lead to better care. No new treatments are being tested…

This study looks at body composition—fat, muscle, and bone mass—in children aged 5 to 18 with beta thalassemia major who receive regular blood transfusions. Researchers will use a simple test to measure these body components and see how they relate to transfusion frequency, iron …

This study examines how regular blood transfusions (exchange transfusions) affect thinking speed in adults with severe sickle cell disease. Researchers will test 85 participants remotely using a simple symbol-matching task and other cognitive assessments at different points in th…