View clinical trials related to Sickle Cell Disease.
Filter by:Background: - Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs. They want to know if it helps a body to better accept donor stem cells. Objectives: - To see if low dose radiation (300 rads), oral cyclophosphamide, pentostatin, and sirolimus help a body to better accept donor stem cells. Eligibility: - People 4 and older with beta-thalassemia or sickle cell disease that can be cured with transplant, and their donors. Design: - Participants and donors will be screened with medical history, physical exam, blood test, tissue and blood typing, and bone marrow sampling. They will visit a social worker. - Donors: - may receive an intravenous (IV) tube in their groin vein. - will receive a drug injection daily for 5 or 6 days to move the blood stem cells from the bone marrow into general blood circulation. - will undergo apheresis: an IV is put into a vein in each arm. Blood is taken from one arm, a machine removes the white blood cells that contain blood stem cells, and the rest is returned through the other arm. - Participants: - may undergo red cell exchange procedure. - will remain in the hospital for about 30 days. - will receive a large IV line that can stay in their body from transplant through recovery. - will receive a dose of radiation, and transplant related drugs by mouth or IV. - will receive blood stem cells over 8 hours by IV. - will take neuropsychological tests and may complete questionnaires throughout the transplant process. - must stay near NIH for 4 months. They will visit the outpatient clinic weekly. - will have 5 follow-up visits for 3 years after transplant, then annually.
The purpose of this study is to determine the feasibility of performing a larger multicenter phase III trial to assess the effects of unfractionated heparin (UFH) in acute chest syndrome (ACS). Prespecified feasibility criteria consists of the ability to enroll potential study participants, which includes the timely notification of hospitalized patients with ACS, the capacity to consent eligible individuals, and the ability to appropriately randomize eligible patients within 24 hours of diagnosis. Additional feasibility objectives involve ensuring appropriate eligibility criteria, proper administration of the study drug, and the ability to completely and accurately collect clinical data of interest. The final aim of our pilot study is to provide preliminary data, with respect to treatment effect and variance, to allow sample size calculation in a larger trial given the lack of data available to help guide this process. The investigators hypothesize that the use of UFH in ACS will result in a decrease in the duration of hospitalization and improve other clinical outcomes, such as the duration of hypoxemia and duration of moderate to severe pain.
Despite the important work of previous sickle cell disease (SCD) cohort studies, there remain many understudied areas that require investigation. An important knowledge deficit is the slow but progressive process of chronic end-organ dysfunction. The majority of organ dysfunction becomes apparent in the young adult years, but comprehensive assessment of adults and understanding of predictors of adulthood organ dysfunction are insufficient. Similarly, the role of disease-modifying therapies, such as hydroxyurea, in preventing organ dysfunction later in life is not clear. Extended follow-up of patients through the transition into adulthood is imperative to understand the long-term implications of pediatric sickle cell care. This observational study will collect data in a systematic fashion at participants' regular clinic visits (in-person or remote) to answer the objectives described below. In addition to primary study objectives, SCCRIP participants will be eligible to participate in a sub-study, which will investigate genetically determined responses to Hydroxyurea (HU) via a pharmacokinetic study (PK). This one time study will involve blood collection at timed intervals proceeding a dose of HU. Defining the basis for this inter-individual variability will allow the identification of poor HU responders prior to initiation of therapy and the seeking of alternative treatments which seek to optimize disease treatment by accounting for individual variability in genes, environment, and lifestyle.
Sickle cell disease (SCD) affects haemoglobin - the molecule in blood cells which carries oxygen. It causes red blood cells to become abnormal crescent (or sickle)- shaped. Sickled red blood cells cannot travel through small blood vessels as easily as normal red blood cells which can lead to blockages. This means that oxygen may be prevented from getting to where it is needed. Individuals with sickle cell disease also suffer form abnormality in the lining of their blood vessels, which contributes to the damage. Damage and blockage can occur in the blood vessels in the brain and means that children with sickle cell disease have a significant risk of suffering from strokes. Research has shown that transcranial Doppler ultrasonography can be used in this setting to identify children at most risk of getting strokes. Ultrasound is therefore used in children with sickle cell disease to measure the blood flow in the vessels in the brain. This research has formed the basis of the National Health Service (NHS) Standard of Care for Sickle Cell Disease in the United Kingdom (UK) which uses transcranial Doppler ultrasonography at once a year to screen children with sickle cell disease aged 2 to 16. Ultrasound is used because it is portable, does not uses ionising radiation such as x-rays, is non-invasive and gives good results. However, the results are dependent on the operator. This means that the screening service is provided by centres of excellence with experienced scanning staff visiting clinics in smaller hospitals with portable machines. There is a lack of research comparing the use of portable machines to laboratory-based machines. This is important because screening can identify children at high risk of stroke and may be used by clinical staff to make a decision about the care of the child.
This is a study of patients with sickle cell disease. It aims to find out if people with sickle cell disease can be cured by changing their immune system before they have blood stem cell transplants. Doctors will give patients a new drug (fludarabine) to see if this drug changes patients immune system and reduces the patient's cells (host) from rejecting donor cells (graft) after the patient gets a Hematopoietic (blood) stem cell transplant.
The proposed research is designed to test the global hypothesis that inhaled corticosteroids (ICS), a therapy developed to treat asthma, will prevent vasoocclusive painful episodes in adults with Sickle Cell Disease (SCD) who wheeze, but do not meet criteria for a diagnosis of asthma. The specific aims of this proposal are 1) Conduct a feasibility study - a randomized controlled trial of ICS for adults with SCD who do not meet criteria for a diagnosis of asthma but report recurrent cough or wheezing, 2) Measure the effects of ICS on biological correlates of pulmonary inflammation (as determined by exhaled nitric oxide) and vascular injury (as determined by sVCAM) in SCD, and 3) Compare properties of traditional and Bayesian adaptive clinical trial design for therapeutic trials in SCD in preparation for designing a definitive trial of ICS. These aims have the potential to 1) change the standard of care for individuals with SCD and recurrent cough or wheeze, 2) provide insight into the pathogenesis of non-asthmatic wheezing in SCD and its response to treatment, 3) explore the suitability of innovative clinical trial designs to overcome the challenges that have hindered therapeutic innovation for SCD.
Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This can result in painful episodes, serious infections, chronic anemia (a decrease in the number of red blood cells), and damage to body organs. Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with sickle cell anemia. These include a reduction in the frequency of pain crises and acute chest syndrome (inflammation of the lungs) and an increase in hemoglobin (the oxygen-carrying protein) in the blood. Patients on hydroxyurea who receive a maximum tolerated dose (MTD) that is specific for them have greater clinical benefit than those who receive a standard lower dose. There is, however, no way currently to predict the MTD for individual patients. As such, MTD for each patient is currently determined by gradual increases in the dose over several months. This process is time-consuming, requires monthly clinic visits, and delays the benefits of hydroxyurea therapy. Our research group has come up with an equation that could be used to predict each patient's MTD using baseline clinical and laboratory measures before starting hydroxyurea treatment. The purpose of this research study is to compare the use of our equation for predicting MTD to the current standard practice of gradually increasing the hydroxyurea dose until MTD is reached. We want to see if the use of our predictive equation will allow us to achieve MTD faster and with no more side effects than with the standard practice.
This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body. We will be comparing deferiprone with deferoxamine, another drug that removes iron from the body.
The investigators propose that culturally aligned community-based interventions in our multi-ethnic sickle cell disease (SCD) population, augmented by task-focused communication technology, can improve self-managed adherence to hydroxyurea (HU) by decreasing barriers to use, supporting parent-youth partnerships for chronic disease self-management and reinforcing the behavior of daily medication use. Culturally aligned community health workers (CHW) are a well-established means to support chronic disease self-management by underserved families, in partnership with medical homes. CHWs can identify and address multiple barriers and reinforce developmentally appropriate self-management to help youth reach and maintain their best fetal hemoglobin (HbF) levels. However, this strategy alone may be insufficient to achieve daily HU adherence. The investigators therefore propose a feasibility trial to test the feasibility and acceptability of a structured intervention of CHW support to address existing barriers to improve HU use, augmented by daily cue-based parent and youth text message reminders, to efficiently extend CHW family support and reinforce family partnerships for self-management.
Related donor stem cell transplantation using the alemtuzumab/ TBI platform has been shown to be a safe strategy to cure severe sickle cell disease. However, due to a lack of suitable donors, many patients cannot benefit from this strategy. Alternative donor sources are desperately needed to fill this gap. Nearly all patients will have a haploidentical family member who would be able to donate. The use of post transplantation cyclophosphamide has greatly improved the outcome of haploidentical stem cell transplantation. The investigators propose to combine this with alemtuzumab/TBI conditioning.