Thalassemia

This study explores whether romiplostim, a drug that stimulates platelet production, can safely and effectively help platelet recovery in children with thalassemia who receive a half-matched stem cell transplant. Researchers will track how quickly platelet levels rise, how many p…

This observational study tests whether hetrombopag, a platelet-boosting drug, can safely speed platelet recovery in children with thalassemia who receive a half-matched (haploidentical) stem cell transplant. Thirty children aged 2 to 17 will be followed for 28 days after transpla…

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 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 study tested a single treatment using CRISPR gene editing to modify a person's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the therapy and were monitored for safety and how long th…

This small study tested whether L-glutamine, a common supplement, can reduce high blood pressure in the lungs of adults with non-transfusion-dependent thalassemia. Eight participants took either glutamine plus standard care or standard care alone for 60 days. The main goal was to…

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 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 observational study checked whether children with hepatitis C who also have thalassemia or survived childhood cancer are more likely to develop insulin resistance (a step toward diabetes). Researchers compared 90 infected children to healthy kids of the same age and gender. …

This study looked at how a high-fat meal changes the amount of the drug etavopivat in the blood of 16 healthy adults. Participants took a single dose of etavopivat after eating a high-fat meal and on an empty stomach on separate days. The goal is to understand if food affects how…

This completed Phase 1 study in 37 healthy adults tested whether a new drug called etavopivat affects how the body processes five common medicines: digoxin, rosuvastatin, midazolam, pitavastatin, and metformin. Participants took etavopivat daily for 10 days along with single dose…

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…