Erythrocyte disorder

This early-stage trial tested a new vaccine against Plasmodium vivax malaria, given with an adjuvant to boost immune response. It involved 25 healthy adults in the UK to check safety and immune reactions. The study was terminated early, so full results are not available.

This study tested a drug called siplizumab alongside a stem cell transplant for people with severe sickle cell disease. The goal was to see if it could prevent the body from rejecting the new cells and reduce a serious side effect called graft-versus-host disease. Only one person…

This study looked at the long-term safety of the drug osivelotor in people with sickle cell disease who had already taken it in an earlier trial. The goal was to track side effects and blood changes over time. However, the study was terminated early, so the full safety picture is…

This study tested a daily pill called voxelotor in children aged 2 to 14 with sickle cell disease who had slightly abnormal blood flow in the brain. The goal was to see if the drug could improve that blood flow and reduce stroke risk. The trial was stopped early, but the results …

This small pilot study tested whether imatinib, a drug used for certain cancers, could reduce painful blockages in blood vessels (vaso-occlusive crises) in people with sickle cell anemia. Seven participants aged 18–25 took the drug for 6 months while researchers measured changes …

This was a first-in-human study testing a single dose of an experimental drug called GSK4172239D in 11 people with sickle cell disease. The main goals were to check safety and how the drug moves through the body. The study was terminated early, so results are limited.

This study tested two extra care programs—supportive palliative care and clinical management of other health issues—for older or frail patients who had a donor stem cell transplant. The goal was to see if these programs could improve quality of life and survival compared to stand…

This study tested a new, easy-to-dissolve malaria tablet in infants and newborns weighing less than 5 kg who had uncomplicated malaria. The goal was to see how the medicine worked in the body, how safe it was, and how well it treated the infection. The trial was stopped early aft…

This early-phase trial tested a gene-edited stem cell product called OTQ923 in 4 people with severe sickle cell disease. The goal was to boost fetal hemoglobin to reduce painful crises and other complications. The study was terminated early, so we have limited data on safety and …

This study followed 265 people with sickle cell disease who were taking Oxbryta (voxelotor) as part of their normal care. Researchers wanted to see how the drug affected hemoglobin levels, sickle cell complications, and overall health over up to 5 years. The study was observation…

This study looks at the long-term safety of inclacumab in people with sickle cell disease who have already taken the drug in a previous study. It tracks side effects and how often pain crises occur. The goal is to see if long-term use is safe and helps control the disease.

This study looked at whether voxelotor, a daily pill for sickle cell anemia, can improve blood flow and oxygen supply to the brain in children. The trial planned to enroll 22 participants aged 4 to 30 years with a severe form of sickle cell disease. However, the study was termina…

This study looked at the long-term safety of voxelotor (a daily pill) in 179 people with sickle cell disease who had already taken it in a previous trial. Participants continued taking the drug and were monitored for side effects and complications like pain crises. The trial was …

This study looked at the long-term safety of the drug voxelotor in 162 people with sickle cell disease who had already taken it in earlier trials. Everyone received voxelotor once daily. The main goal was to track side effects. The trial was terminated early, so the full safety p…

This study tested whether a non-invasive breathing machine (ventilation) could prevent acute chest syndrome, a serious lung complication, in children with sickle cell disease who were hospitalized for severe pain. The standard prevention is spirometry, which requires active deep …

This study tested a nasal spray containing a strong painkiller (sufentanil) for adults with sickle cell disease experiencing a painful crisis. The goal was to see if it could provide faster relief than the standard gas mixture (EMONO) before giving morphine through an IV. The tri…

This study looks at how the drug luspatercept is used in real-world clinics for patients with lower-risk myelodysplastic syndromes (MDS) or beta-thalassemia. Researchers will track patient characteristics, treatments, and outcomes like reduced need for blood transfusions. The goa…

This early-stage study is testing a new drug called pociredir in 24 adults with sickle cell disease. Researchers want to see how the drug moves through the body and whether taking it with food changes its effects. The goal is to gather safety and dosing information, not to treat …