View clinical trials related to Anemia, Sickle Cell.
Filter by:This will be an open-label, dose escalating study with a starting dose of 2mg. Up to 6 additional cohorts will be enrolled at subsequently higher doses of 4mg, 8mg, 10mg, 12mg, 16mg, and 32mg. In each dose escalation cohort, each dose will be taken orally, once daily, for 8 weeks.
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and episodically transfused patients. The objective of the study is to determine how specific genetic and non-genetic factors in donors and recipients may impact RBC survival after transfusion - in short, what factors on both the donor and recipient side may improve the efficacy of the transfusion.
Pregnancy in sickle cell disease (SCD) is fraught with many complications including preeclampsia (PE) and intrauterine growth restriction (IUGR). Previously, the investigators found an abnormality in prostacyclin-thromboxane ratio in sickle cell pregnant women, a situation that is also found in non-sickle pregnancies with PE and unexplained IUGR. Low dose aspirin (LDA) has been found to reduce the incidence of PE and IUGR in high-risk women due to its reduction of vasoconstrictor thromboxane whilst sparing prostacyclin, in effect "correcting" the ratio. It has been found to be safe for use in pregnancy and is recommended in obstetric guidelines for this use but has not been tested in sickle cell pregnancy. The investigators hypothesize that LDA would reduce the incidence of IUGR and PE in pregnant haemoglobin (Hb)SS women. The investigators also plan to build a machine-learning model to predict severe maternal outcomes in them. The investigators propose a multi-site, randomized, controlled, double blind trial comparing a daily dose of 100mg aspirin with placebo, from 12 - 28 weeks gestation until 36 weeks. The study sites are three teaching hospitals in Lagos and Ile-Ife, and twelve general hospitals and one federal medical centre within Lagos state, with the coordinating centre at the College of Medicine, University of Lagos (CMUL), Idi-Araba, Lagos. A total of 476 eligible pregnant HbSS and HbSC women will be recruited consecutively and randomly assigned to either group using a web-based app, sealed envelope. Each study group will comprise 238 pregnant women with SCD. All participants will be followed from recruitment till delivery. They will have their body weight, blood pressure and haematocrit checked at each antenatal visit. Their full blood count, vital signs and oxygen saturation will be checked and recorded at each visit. Primary outcome measure will be birth weight below 10th centile for gestational age on INTERGROWTH 21 birthweight charts, and incidence of miscarriage or perinatal death. Analysis will be by intention to treat, and the main treatment effects will be quantified by relative risk with 95% confidence intervals, at a 5% significance level. The investigators plan to develop a prediction model to predict the risk of complications in these women using machine learning. The prediction outcome will be severe maternal outcomes comprising maternal near miss or death.
In this study the investigators will prospectively investigate whether the addition of a 3-months long preconditioning with azathioprine to the alemtuzumab/TBI non-myeloablative conditioning results in improved disease-free survival and donor chimerism after allo-SCT in SCD patients. Furthermore, the investigators will evaluate whether azathioprine/hydroxyurea preconditioning leads to more patients being able to taper and discontinue sirolimus at 12 months post-transplantation.
This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition Module of executive abilities in pediatric participants (8 to < 18 years) with SCD.
This is an open label, single arm multicenter trial to evaluate the effect of voxelotor treatment on cerebral blood flow (CBF) and neurocognitive function in adolescent and young adult participants (12-30 years of age) with sickle cell disease (SCD).
The purpose of this project is to develop novel approaches to promote health and longevity while enhancing quality of life among persons with Sickle cell disease (SCD). Therefore, investigators are aiming to adapt an evidence-based exercise intervention for adults with SCD informed by culturally- relevant and biologic factors.
This is a multicenter prospective, longitudinal cohort study which will evaluate the predictive capacity of machine learning (ML) models for progression of CKD in eligible patients for a minimum of 12 months and potentially for up to 4 years.
Background: People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant. Researchers want to learn how and why this happens. Objective: To study the benefits of treatments that are intended to cure SCD. Eligibility: People aged 18 and older with SCD who are either receiving curative therapy in the next 3 months or don t have any plans to receive a curative therapy in the next 2 years. Design: At their first visit, participants will be screened with their medical history and a physical exam. Participants will then have a baseline visit. This will take about a week to complete and will include: Blood and heart tests MRI of the brain, heart, and lungs. Participants will lie on a bed that will move into the MRI scanner. Special padding may be placed around their head to keep it still. Interactive games. Participants will complete computer games that test memory, attention, problem solving, language, spatial orientation, processing speed, and emotion. Questionnaire rating quality of life Iothalamate test. An IV catheter will be placed into a vein. A contrast agent will be injected through the IV. Blood will then be collected at different time points. Lung function tests and a 6-minute walk test Vibration controlled transient elastography. A probe placed on the abdomen will measure liver scarring. DOS test. A light attached to the finger or toe will measure blood oxygen. Participants will have an end-of-study visit about 2 years after their baseline visit. This will include repeats of the baseline visit tests.
Sickle cell disease is the subject of targeted neonatal screening (carried out when one of the two parents is from an endemic country - sub-Saharan Africa, South-East Asia, Central America, the Caribbean) during the Guthrie test. Haemolysis, which results from the abnormality of the haemoglobin, and the vascular activation it causes, are responsible for multiple organ damage. Major sickle cell syndromes (MSC), by several mechanisms, are responsible for a wide range of renal damage, culminating in end-stage renal failure at an average age of 45 years and with an average survival of 3 years beyond ESRD. The various renal disorders are : glomerular hyperfiltration and then glomerulosclerosis; tubular dysfunction, especially proximal and distal hyposthenuria (a factor in enuresis); papillary necrosis, renal infarction, episodes of acute renal failure during vaso-occlusive crises; dysregulation of the renin-angiotensin system with early arterial hypertension and, more rarely, extra-membranous glomerulonephritis. In the early stages of these conditions, simple paraclinical tests can identify them before the appearance of specific clinical signs. In patients suffering from MDS, the HAS (High Authority of Health) recommends an annual check-up carried out in a Competence Centre. According to the HAS recommendations for annual surveillance, in addition to the search for other organic complications, for renal pathology, only microalbuminuria and renal ultrasound are recommended. However, as the literature shows, microalbuminuria and ultrasound only detect some of these renal disorders and at a very late stage. A large number of publications in adults and, to a lesser degree, in children, demonstrate the correlation between the frequency of acute complications of sickle cell disease (episodes of haemolysis, etc.) and the occurrence of kidney damage.