Sickle cell disease

Researchers are testing a one-time gene therapy called exa-cel for people with severe sickle cell disease. The treatment uses CRISPR-Cas9 to edit a patient's own blood stem cells, which are then infused back after chemotherapy. The trial measures whether participants remain free …

This trial tests whether MitoQ, an antioxidant supplement, can improve platelet function in adults with sickle cell anemia. Platelets in sickle cell disease are often overactive, which may contribute to blood vessel damage and complications. Researchers will give 18 participants,…

This study looks at whether voxelotor, a drug that helps red blood cells stay healthy, can improve physical function in people with sickle cell disease. Six adults aged 18-65 will take the drug for 6 months and have their walking ability tested. The goal is to see if the drug hel…

This study tests whether video directly observed therapy (VDOT) — where patients or caregivers use a smartphone app to record themselves taking hydroxyurea — can improve adherence in young people with sickle cell disease. Participants will use an electronic pill bottle to track t…

This study follows adolescents and young adults (ages 12–21) with sickle cell disease who take hydroxyurea, a medication that helps prevent pain crises and other complications. The researchers are testing whether a mobile app called HU-Go can help these patients build a consisten…

This pilot trial tests whether a smartphone app called MED-Go can help adolescents and young adults with sickle cell disease take their daily hydroxyurea more consistently. Participants aged 12 to 21 will use the app for 12 weeks, and researchers will measure how often they log t…

This study tested a new oral medicine called NDec in 96 adults with sickle cell disease. The goal was to see if it safely raises hemoglobin levels and reduces painful crises. Participants took capsules twice weekly for about a year, with some also continuing their usual hydroxyur…

This study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…

This study tests whether a peer coaching program delivered by phone and text can help adolescents and young adults (ages 16–22) with chronic conditions like sickle cell disease, diabetes, or cystic fibrosis improve their self-management skills. Participants are paired with a trai…

This phase 2 trial tested a half-matched (haploidentical) bone marrow transplant in 95 children and adults with severe sickle cell disease. The goal was to see if this approach could help patients live without major complications for two years. Participants received a reduced-int…

This study tested a nutraceutical supplement called NUV001 in 168 adults with sickle cell disease. Participants took either an immediate-release or gastro-resistant version of NUV001, or a placebo, daily for 90 days. The main goal was to check safety and tolerability, with early …

This study looked at whether teaching mothers about nutritious local foods can prevent malnutrition in children with sickle cell disease (SCD) in northern Nigeria. 146 children aged 6 to 18 months with SCD took part. Community health workers gave mothers special nutrition advice,…

This early-stage trial tested a new drug called PF-07209326 in 52 people, first in healthy volunteers and then in patients with sickle cell disease. The main goal was to check safety and how the body processes the drug. Researchers also looked for any early signs that the drug mi…

This study tested whether losartan, a blood pressure medication, can reduce or stop scarring in the heart muscle of people with sickle cell disease. Fourteen participants aged 6 and older took losartan for 12 months. The goal was to see if the drug could improve heart health and …

This completed Phase 2 trial tested an oral drug called epeleuton in 35 adults with sickle cell disease. The goal was to see if it could reduce painful vaso-occlusive crises and improve blood cell health. Participants took 2000 mg capsules twice daily for 16 weeks, and researcher…

This pilot study tested a stem cell transplant from closely matched unrelated donors for 8 patients with severe sickle cell disease or thalassemia major. The donated stem cells were specially processed to remove certain immune cells to reduce the risk of rejection and graft-versu…

This study tested an experimental drug called etavopivat in 12 children and young adults (ages 12-21) with sickle cell disease. The goal was to see if the drug could improve blood flow and oxygen delivery to the brain and muscles. Participants took the drug and had scans to measu…

This early-phase study tested a gene therapy for sickle cell disease in 6 patients aged 12-20. Doctors took the patients' own blood stem cells, added a working gene to help produce healthy red blood cells, and infused them back after chemotherapy. The goal was to see if the treat…

This pilot study tested a combined bone marrow and kidney transplant from a partially matched family donor in 10 people with both a blood disorder and chronic kidney disease. The goal was to treat both conditions at once and possibly reduce the need for lifelong anti-rejection dr…

This early-stage study tested a new drug, Pociredir (FTX-6058), in 45 adults with sickle cell disease to see if it is safe and how the body processes it. The main goal was to check for side effects and measure drug levels in the blood. Researchers also looked at changes in fetal …

This study tested whether L-Arginine, a natural amino acid, could improve heart function in children with sickle cell disease who have increased blood pressure in their lungs. Fifty children aged 5-18 took L-Arginine capsules for 3 months. Researchers measured changes in heart bl…

This study tested a drug called crizanlizumab in 36 men with sickle cell disease who suffer from priapism—painful erections lasting an hour or more. The goal was to see if the drug could reduce how often these events happen. Participants received the drug by IV and reported their…

This study tested whether adding erythropoietin (EPO) to the standard drug hydroxyurea can safely raise hemoglobin levels in adults with sickle cell disease. Seventeen participants with chronic anemia took both drugs for 12 weeks. The main goal was to see if at least a 1.0 g/dL i…

This Phase 2 study tested an experimental drug called etavopivat in 53 people with thalassemia or sickle cell disease. The goal was to see if the pill could safely reduce the number of red blood cell transfusions needed and raise hemoglobin levels. Participants took 400 mg of eta…

This early-phase clinical trial tested a gene therapy for adults with severe sickle cell disease. The treatment involved taking the patient's own blood stem cells, modifying them with a harmless virus to carry a healthy gene, and infusing them back after mild chemotherapy. Only 4…

This study tested a drug called inclacumab in about 240 people with sickle cell disease who experience painful vaso-occlusive crises. Participants received either inclacumab or a placebo by IV infusion. The main goal was to see if inclacumab could reduce the number of these painf…

This study tested whether giving oxygen at home during the night could prevent severe pain episodes and other complications in pregnant women with sickle cell disease. 178 pregnant women took part, using an oxygen device for 4 hours each night. The goal was to see if this simple …

This study tested a drug called crizanlizumab in 117 children aged 2 to 18 with sickle cell disease who have had painful vaso-occlusive crises. The goal was to find the right dose and check safety, with some children also taking hydroxyurea. The drug works by blocking a protein i…

This small pilot study tested whether famotidine, a widely used heartburn drug, can reduce a blood marker linked to painful crises in children with sickle cell disease. Thirty children took the medicine for 29 days. The goal was to see if the drug lowers P-selectin levels, which …

This study tested whether giving sickle cell patients two drugs (azathioprine and hydroxyurea) for three months before a stem cell transplant could help the transplant work better. Twenty adults with a matched sibling donor took part. The goal was to see if this approach could re…

This study tested the safety of a drug called crizanlizumab in 140 Indian patients aged 16 and older with sickle cell disease who have had painful crises. The drug was given by IV infusion over several months, with or without standard hydroxyurea therapy. The main goal was to che…

This study tested whether taking vitamin D every day could reduce lung complications in children and teens with sickle cell disease. Lung problems like infections, asthma attacks, and acute chest syndrome are major causes of illness and death in this condition. Researchers gave d…

This early-phase trial tested a new gene therapy called BIVV003 in 7 adults with severe sickle cell disease. The treatment uses the patient's own stem cells, which are gene-edited in a lab and then infused back after chemotherapy. The main goals were to check safety and see if th…

This phase 2 trial tested a bone marrow transplant from a half-matched (haploidentical) donor in 25 people with severe sickle cell disease. The goal was to see if a less intense conditioning regimen and a drug called cyclophosphamide could prevent graft-versus-host disease while …

Children with sickle cell disease who are hospitalized for painful vaso-occlusive crises can develop acute chest syndrome, a serious lung complication. Doctors usually diagnose it with a chest X-ray, but researchers want to know whether lung ultrasound works as well or better. In…

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 tests whether a special PET scan tracer can visualize and measure vaso-occlusive crises (painful blockages of blood vessels) in people with sickle cell disease. Researchers will compare tracer uptake with pain levels and clinical markers to see if this imaging method c…

This study looked at whether the Montreal Cognitive Assessment (MoCA), a short test, can accurately screen for thinking and memory problems in adults with sickle cell disease. Researchers compared MoCA results to a full set of standard cognitive tests in 65 adults. The goal is to…

This study tested a drug called crovalimab in 95 people with sickle cell disease to see if it could prevent vaso-occlusive episodes (painful blockages in blood vessels). Participants received either crovalimab or a placebo alongside their usual treatments. The goal was to measure…

This study tested whether daily zinc supplements can lower the number of serious infections in young children with sickle cell anemia. Researchers in Uganda gave 100 children either zinc or a placebo for a period and tracked infections. The goal is to find an affordable, easy-to-…

This trial tests whether a smartphone app called You Cope, We Support (YCWS) can help adults with sickle cell disease better manage stress and pain, and possibly reduce opioid use. Participants will be randomly assigned to either use the app along with self-monitoring, or just se…

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 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 trial tests a computer-based tool called PAINRelieveIt for adults with sickle cell disease, a condition that causes intense pain episodes. The tool has three parts: one helps patients report their pain, another educates them about pain management, and a third helps doctors c…

This study tested whether hyperbaric oxygen therapy — breathing pure oxygen in a pressurized chamber — could reduce pain and hospital stays for adults having a sickle cell pain crisis. Eight adults with uncomplicated crises received 1 to 3 sessions. Researchers measured pain chan…

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 tested a personalized exercise program called Gerofit for older adults with sickle cell disease. The goal was to see if the program is safe, doable, and helps improve physical health and quality of life. Thirty participants took part in tele-exercise sessions focusing …

This phase 3 trial tests whether intravenous arginine, an amino acid, can shorten painful episodes in children with sickle cell disease. Participants aged 3 to 21 receive either arginine or a placebo alongside standard pain treatment. The study measures how quickly the crisis res…

This study tested a daily dietary supplement called NUV001 in 12 adults with sickle cell disease (SS type) over 120 days. The main goals were to see if it is safe and tolerable, and to measure its effects on blood health and quality of life. Researchers tracked side effects, hosp…

This study tested a mobile prayer program called PUSH (Pray Until Something Happens) to see if it could help adults with sickle cell disease feel less stress and pain. 40 people who had pain and used opioids took part. They listened to guided prayer recordings on a smartphone. Th…

This study tested whether a medicine called Intravenous Immunoglobulin (IVIG) is safe and helps shorten painful episodes in people with sickle cell disease. 300 participants aged 6 to 65 took part. The goal was to see if IVIG could reduce how long the pain lasts and how much stro…

This study tested whether guided relaxation and acupuncture can reduce chronic pain and opioid use in adults with sickle cell disease. About 360 participants across three health centers were randomly assigned to one of these therapies or usual care. The goal was to see if these m…

This small study tested whether nitrous oxide (laughing gas) can quickly relieve severe pain in children with sickle cell disease during a pain crisis. Five patients aged 8 to 18 who still had high pain after standard treatment received nitrous oxide in the emergency room. Resear…

This study tested a drug called canakinumab (ACZ885) in 49 children and young adults aged 8-20 with sickle cell anemia. The goal was to see if it could reduce daily pain compared to a placebo. Participants rated their pain daily using a 0-10 scale, and researchers measured change…

This study looked at whether aquatic (water) exercises could improve muscle strength and quality of life in 50 children aged 6-10 with sickle cell anemia. Children did water-based activities to see if it helped their grip strength, leg and trunk muscles, and overall well-being. T…

This study tested a program called I-STRONG, which combines mind and body training to help teenagers with sickle cell disease cope with pain. Researchers worked with 45 teens to see if the program was practical and well-liked. The goal was to reduce pain severity and improve dail…

This study tested whether a 12-minute guided relaxation video on a tablet could help adults with sickle cell disease manage pain and stress during a hospital stay. 46 participants were randomly assigned to watch the relaxation video or discuss their sickle cell experiences. The g…

This study tested whether a 20-minute virtual reality session with hypnosis suggestions could reduce pain and anxiety in children with sickle cell disease during a painful crisis. Twelve children aged 6 and older used a VR headset while listening to calming narratives designed fo…

This study measured how common pulmonary hypertension (high blood pressure in the lungs) is in adults with sickle cell anemia. Nearly 1,000 participants had heart ultrasounds and blood tests, then were contacted by phone for up to 3 years to track their health. The goal was to un…

This completed study looked at why people with sickle cell disease develop chronic leg ulcers. Researchers collected skin swabs, blood samples, and survey data from 405 adults to explore the role of skin bacteria and environmental factors. The goal was to better understand the ca…

This study explored how family members share health information about genetic diseases like sickle cell, diabetes, and cancer. Over 1,000 adults completed surveys or interviews about their family health history and support. The goal was to understand social and relational factors…

This study looked at what happens in the blood of people with sickle cell disease during painful attacks. Researchers compared blood samples taken during a pain crisis to samples taken when participants were feeling well. The goal was to learn more about the role of inflammation …

This study aims to understand why some parents of children with sickle cell disorder (SCD) may not engage with health services during the first year after diagnosis, and to co-design strategies with parents and health professionals to improve support accessibility. The research i…

This completed Phase 1 study looked at 27 people with and without pulmonary hypertension to learn more about how blood vessel problems contribute to the disease. Researchers used heart catheterization, imaging, and blood tests to find biomarkers and understand disease mechanisms.…

Sickle cell disease can affect brain development even in very young children. This study uses eye-tracking—a simple, non-invasive method—to measure attention and processing speed in infants aged 6 to 24 months. The goal is to identify those at highest risk for cognitive challenge…

This study looked at 70 adults with sickle cell disease to see which lab test—cystatin C or creatinine—better measures kidney function. Participants gave blood and urine samples and had a special dye test to check how well their kidneys work. The goal is to help doctors catch kid…

This completed observational study looked at how often young people with sickle cell disease (under 26) in France develop problematic use of painkillers and a gas mixture called EMONO. Researchers interviewed over 1,000 patients by phone to check for signs of addiction or depende…

This study looked at over 30,000 adults admitted to hospitals in Maryland to understand why some patients wait in the emergency room even after being admitted (called boarding). Researchers wanted to find out what patient traits lead to longer waits, how boarding increases health…

This study followed 67 children with sickle cell disease who had a high risk of stroke. Researchers compared those who received a bone marrow transplant to those who got standard care, checking brain scans and thinking skills 9-10 years later. The goal was to see which approach b…

This study looked at whether two different telemedicine models could help children with sickle cell disease living in medically underserved areas get better care. Researchers tested the models with 24 families and measured things like appointment attendance, lab work, and satisfa…

This study tested an electronic diary for adults with sickle cell disease to track daily symptoms like pain, tiredness, and ability to do usual activities. About 98 participants used the diary for 6 months, and researchers compared those on disease-modifying treatment to those no…

This study examined blood samples from 119 adults with sickle cell disease, sickle cell trait, or no condition to understand why some develop dangerous blood clots. Participants gave blood and had health check-ins over two years. The goal was to find biomarkers that could lead to…

This study surveyed 186 adults with sickle cell disease in the U.S. to learn how the COVID-19 pandemic impacted their stress, anxiety, pain, and healthcare use. Participants completed online questionnaires about their medical history, mental health, and experiences during the pan…

This study looked at how long donor red blood cells survive in children with sickle cell disease who get regular blood transfusions. Researchers labeled a small part of the transfused blood with a harmless marker and tracked it over time. The goal was to better understand why som…

This completed study monitored pregnancy and infant outcomes in 13 women with sickle cell disease who were treated with the drug crizanlizumab (Adakveo) during pregnancy or shortly before. The goal was to track rates of fetal loss and birth defects. Because it was a small observa…

This study looked at whether a special kidney scan (scintigraphy) can find early kidney damage in children and young adults with sickle cell disease better than standard tests. 18 patients were checked during their annual visit. The goal is to catch kidney problems sooner so trea…

This study measured how often iron builds up in the heart muscle of people who receive many blood transfusions. Researchers used MRI scans to check heart iron levels in 110 patients with thalassemia, sickle cell disease, or myelodysplasia. The goal was to understand how common th…

This study followed 94 young people with sickle cell disease as they moved from pediatric to adult medical care. Researchers tracked hospital visits, medication use, and quality of life to find out what makes this transition successful. The goal is to improve programs that help p…

This study asked over 1,000 emergency doctors in Europe what they would prescribe for a patient with sudden belly pain. The patient's race was changed to see if it affected the doctor's choice to give morphine. The goal was to understand if racial bias plays a role in pain manage…

This study collected umbilical cord blood from over 300 newborns with sickle cell disease, sickle cell trait, or no disease to find the best ways to process and store it. The blood is rich in stem cells that might one day be used in treatments like gene therapy or transplants. Th…

This study looked at 1,753 patients needing a stem cell transplant for blood cancers or other serious blood disorders. Researchers compared two strategies: first searching for a matched unrelated donor, then trying an alternative donor if needed, versus going straight to an alter…

This completed phase 1 trial studied how etavopivat, a potential new medicine for sickle cell disease and thalassemia, affects the heart's electrical activity. Thirty-three healthy adults received single doses of etavopivat, a placebo, or an approved heart medication (moxifloxaci…

This completed Phase 1 study tested a single dose of the drug etavopivat in 24 healthy Chinese adults. Researchers measured how much of the drug got into the blood and how long it stayed, as well as any side effects. The goal was to gather basic safety and drug-processing informa…

This study screened 8,400 people living in Chattogram, Bangladesh to find out how common abnormal hemoglobin types are. Researchers used a blood test called HbA1c capillary electrophoresis to detect conditions like HbE trait. The goal was to understand the prevalence of these inh…

This study looked at why people with sickle cell disease often get hard-to-heal leg ulcers. Researchers measured blood flow, nerve function, and blood markers in 70 patients with and without ulcers. The goal was to better understand what causes these wounds, not to test a new tre…

This study looked at how sickle cell disease affects leg muscles. Researchers compared muscle strength and fatigue in 77 people with and without sickle cell disease during a short exercise. The goal was to learn if muscle problems contribute to poor physical fitness in sickle cel…

This study measured blood vessel function in 49 adults with sickle cell disease to see how it relates to disease severity. Researchers used laser and pulse wave tests to check small and large blood vessels. The goal was to better understand the disease, not to test a new treatmen…

This completed study surveyed 257 adults with sickle cell disease to understand how they use painkillers at home, including opioids and cannabis. Researchers wanted to find out how common opioid addiction is in this group. Patients answered a one-time questionnaire at three speci…

This study looked at how many girls and young women aged 11 to 20 with chronic diseases (like diabetes or immune conditions) got the HPV vaccine, compared to those without chronic illness. Researchers reviewed records of 223 participants from a hospital in France. The goal was to…

This study collected umbilical cord blood from 44 newborns whose mothers carry the sickle cell trait. The goal was to study the cells in the lab to better understand the disease and develop ways to modify and preserve them. No treatment was given; this was purely for research pur…

This study worked to improve how doctors estimate kidney function in people with sickle cell disease. Researchers measured actual kidney function in 120 patients and used that data to develop a new formula tailored to sickle cell. The goal is to replace current equations that wer…

This completed study checked how often inherited blood disorders (hemoglobinopathies) occur in people from Mardan, Pakistan, and nearby areas. Researchers analyzed blood samples from 839 people using special lab tests. The goal was to gather local data to help improve public heal…

This study followed 58 babies with sickle cell disease from 3 months to 2 years old to find early clues that might predict a life-threatening spleen complication called acute splenic sequestration. Researchers took blood samples and used special scans to measure spleen size and f…

This early-phase study tested whether a single injection of plerixafor could safely and effectively mobilize stem cells in 3 adults with severe sickle cell disease who lacked a matched sibling donor. The goal was to see if enough stem cells could be collected for a possible futur…