View clinical trials related to Sickle Cell Disease.
Filter by:Background: - Many people with sickle cell disease have repeated episodes of severe pain that lasts for days, requiring hospital care. These episodes, called pain crises, may be caused by changes in blood flow. Researchers want to study blood flow in people with sickle cell disease who are having a pain crisis and compare it with their blood flow after the pain crisis has resolved. They also want to compare these measurements against blood flow in healthy people who do not have sickle cell disease. Objectives: - To study whether changes in blood flow cause pain crises in people with sickle cell disease. Eligibility: - Individuals at least 18 years of age who have sickle cell disease and are being treated for a pain crisis. - Individuals at least 18 years of age who have sickle cell disease and are not experiencing a pain crisis. - Healthy volunteers matched by age and gender with the participants who have sickle cell disease. Design: - Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. - Participants having a sickle cell pain crisis will have two visits, one during the crisis and one about 4 weeks after the crisis has resolved. - Participants not having a sickle cell pain crisis will have one or two study visits. Blood samples will be collected during at least one of these visits. - Healthy volunteers will have one or two study visits. Blood samples will be collected during at least one of these visits. - During each visit for all participants, cameras and blood flow monitoring equipment will be used to measure blood flow in the forearm. sickle cell disease.
Sickle cell disease (SCD) is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape. Sickle-shaped cells can cause problems by getting stuck in blood vessels, blocking blood flow, and can cause inflammation and injury to important body parts. There are no specific treatments that improve this condition and promote blood flow hindered by sickle cell blockages. Another big challenge in managing sickle cell disease is that there are no good measures to determine changes and improvements in blood flow. Contrast-enhanced ultrasound is a technique currently used to detect blood flow in the heart, muscles, and other organs. It is extremely sensitive and can detect blood flow in the smallest of blood vessels. It would be very useful in helping healthcare providers know whether treatment strategies are improving blood flow during sickle cell blockages. The hypothesis is that contrast-enhanced ultrasound will be a feasible tool for determining changes in blood flow of subjects with sickle cell disease.
In Sickle cell disease children, sleep respiratory abnormalities are risk factors for vaso-occlusive complications, as well as cerebral vasculopathy. A 18 months follow-up children with sickle cell disease evaluating sleep respiratory problems frequency and etiology, as well as their influence on sickle cell disease complications.
This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features. The primary goal of this multi-center Phase II study is to determine the safety and feasibility of a conditioning regimen consisting of busulfan (Bu)/ fludarabine (Flu)/ anti-thymocyte globulin (ATG) in adult patients with severe SCD. A two-component design will be used for this study. The first component will be restricted to patients who have an HLA-identical sibling donor. Five patients will be transplanted during the first component of the study. If no more than 2 of the first 5 patients experience unacceptable toxicity, including death, within the first six months after transplantation, then the safety of the regimen will be considered promising in adult SCD patients. The second component will include patients who have a related or an unrelated human leukocyte antigen (HLA) matched donor. Up to 15 additional patients will be transplanted in this component of the study which will evaluate the safety and feasibility of unrelated donor hematopoietic cell transplantation (HCT) in adults with SCD. Data related to study endpoints for 1 year after transplantation will be collected; however, participating centers will be encouraged to conduct long-term follow-up evaluations of patients according to standard institutional guidelines. The purpose of this pilot safety trial is to see if this approach is feasible and meets accrual goals lending support to the development of a subsequent full scale investigation of HCT and comparing outcomes in a transplantation cohort to a control cohort of adults eligible for, but unwilling or unable to receive HCT treated by supportive therapy with a primary endpoint of five years survival for this full scale comparative trial.
Background: - Some people with sickle cell disease have different health problems than others. This may be related to how easily and frequently the red blood cells break apart in the blood. Researchers want to test breath and blood samples from people with sickle cell disease to look for very small amounts of carbon monoxide, which is produced when red blood cells break apart. They will compare these results with breath samples from healthy volunteers. Studying different levels of carbon monoxide may help predict what health problems a person with sickle cell disease may get. It may also provide more information on possible treatments. Objectives: - To study breath carbon monoxide levels and their possible relation to the severity of sickle cell disease. Eligibility: - Individuals at least 18 years of age with sickle cell disease. - Healthy volunteers who are matched for age, sex, and race with the sickle cell disease group. Design: - Participants will be screened with a medical history. - Participants with sickle cell disease will provide a blood sample and have a heart function test. They will also breathe into a bag to provide an exhaled breath sample. - Healthy volunteers will provide an exhaled breath sample. - No treatment or care will be provided as part of this study.
Epidemiologic studies suggest that older stored blood is associated with worse outcomes in certain hospitalized patients. Storage of red cells is associated with a storage lesion and the survival of transfused red cells decreases with increasing storage time, thus older blood is associated with an increased acute delivery of hemoglobin-iron to the reticuloendothelial system. The investigators have preliminary data in healthy human volunteers suggesting that delivery of a significant iron load to the reticuloendothelial system from aged red cells leads to the elaboration of a potentially toxic form of iron known as non-transferrin--bound iron. The investigators will extend these results by testing whether a similar effect is seen in chronically transfused patients with hemoglobinopathies. This patient population will also allow the investigators to test whether iron- chelation therapy is beneficial in this setting. Finally, the investigators will also test whether washing or cryopreserving the red blood cells has any effect on this outcome. These findings may explain the immunomodulatory effects of older stored blood in patients and will help us develop safer transfusion products for patients.
The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).
The objective of this study is to evaluate the effectiveness of 1-year administration of laying-on-of-hands on the morbidity and mortality of patients with sickle cell disease in Africa.
Interventional Allocation: Randomized Endpoint Classification: Safety/Efficacy Study of combined chelation therapy Masking: Open Label Primary Purpose: Treatment of transfusional iron overload Primary Outcome Measures: • The primary outcome measure is to assess efficacy in lowering serum ferritin level(the change in serum ferritin compared to baseline) with combining DFP and deferasirox compared to combined DFP and DFO in conditions with severe chronic iron overload; showing an up-trend of SF over previous 12 months on single chelator. Secondary Outcome Measures: • The secondary outcome measure is to determine the number of patients who will develop adverse events in order to assess safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP.
The investigators propose to determine the engraftment and transplant related morbidity and mortality after a non-myeloablative allogeneic hematopoietic stem cell transplant protocol using immune- suppressive agents and low-dose total body irradiation (TBI) without standard chemotherapy in patients with aggressive sickle cell disease who are not candidates for or experienced complications from hydroxyurea therapy. Fully HLA matched siblings will be used as donors for hematopoietic stem cells to reduce the risk of morbidity and mortality in this cohort of patients.