SICKLE CELL DISEASE

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 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 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 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 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 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 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 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 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 …

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 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 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 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.…

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 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 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 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 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 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 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 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 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 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 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…