View clinical trials related to Sickle Cell Disease.
Filter by:Hydroxyurea Optimization through Precision Study (HOPS) is a prospective, multi-center, randomized trial that will directly compare a novel, individualized dosing strategy of hydroxyurea to standard weight-based dosing for children with SCA. The primary objective of the study is to evaluate whether a pharmacokinetics-based starting hydroxyurea dose thieves superior fetal hemoglobin response to to standard weight-based initial dosing. Patients will be recruited from the pediatric sickle cell clinic at Cincinnati Children's Hospital Medical Center and from additional pediatric sickle cell centers within the United States.
Background The pediatric-adult care transition is a risk-disrupting time for patients with chronic disease. This care transition takes place during adolescence; a period of psychological upheavals and adaptations of family roles. During this period, medication adherence is non-optimal and absenteeism at medical appointments is high. Sickle cell disease (SCD) is the first genetic disease detected in France. It is chronic disease characterized by frequent painful vaso-occlusive crises (VOC) requiring emergency hospitalization when they are severe. Other serious complications are acute chest syndromes (ACS) and stroke. In order to improve the health status of teenagers with sickle cell disease, it is necessary to anticipate this care transition and to involve the pediatric and adult sectors. The biopsychosocial health approach and the Social-Ecological Model of Adolescent and Young Adult Readiness to Transition (SMART) describe a care transition integrating bioclinical and psychosocial factors such as integration of the patient's family, education on disease and therapeutics, psychological management of pain and medico-social orientation. The pediatric-adult transition program proposed is based on this biopsychosocial approach. It aims to improve the health status of adolescents with SCD, their quality of life and the use of health care service. Objective of the study To assess the impact of a pediatric-adult transition program on the incidence of sickle-cell-related complications leading to hospitalization on 24-months after transfer to the adult sector. The evaluation focuses on severe complications leading to hospitalization, such as VOC, ACS, and stroke. Study design Multicenter Open-label individual Randomized Controlled Trial Population : Patients aged at least 16 years old with sickle cell disease, and their parents (or legal representatives Number of subject : 196 patients (98 patients by arm) The study will last 24 months Expected results For patients and families Better health and quality of life for patients is expected, including better use of medical care after the transition program. It is also expected a better experience of the pediatric-adult care transition and indirectly a better experience of intrafamilial relations. For health professionals This project is expected to provide solutions to improve the pediatric-adult care transition of patients with chronic disease. Indeed, the methodological quality of the study will make it possible to evaluate the efficiency of the proposed program, to possibly adapt it and test it to other chronic diseases presenting the same care transition problematic. In terms of public health SCD mainly affects populations of sub-Saharan origin, with low visibility and high social vulnerability. By focusing on this population, this project will reduce the social inequalities in health, experienced by patients with SCD and their families. By improving the health, quality of life and care of patients with SCD, this project is expected to decrease the cost of the pediatric-adult care transition period.
In this single-arm, one-stage Phase II study, the investigators hypothesize that gut decontamination with rifaximin will reduce the frequency of hospital admission due to painful crisis in patients with SCD. The study will accrue 20 SCD patients who had at least two hospital admissions in the previous 12 months. These patients will receive rifaximin 550 mg twice a day for a total of 12 months. This following clinical parameters will be measured: 1. Changes in the annual rate of hospital admissions due to painful crisis; 2. Changes in the annual rate of days hospitalized; 3. Annual rates of uncomplicated crises; 4. Annual rate of acute chest syndrome; 5. Changes in the quality of life; and 6). Toxicities. The following laboratory parameters will be measured: 1. Changes in the number of circulating activated neutrophils; 2. Changes in the intestinal microbiome diversity; 3. Changes in the urinary 3-indoxyl sulfate levels; 4. Changes in the serum biomarkers of intestinal permeability (lipopolysaccharides; zonulin, citrulline, and fatty acid binding proteins).
The Investigators hypothesize that older red cell units trigger phagocytosis and activation of circulating macrophages with a downstream immunomodulatory cascade and release of excess Non Transferrin Bound Iron(NTBI) that leads to increased rates of infection in adults with Sickle Cell Disease(SCD). To test this hypothesis, the study staff will perform a randomized prospective clinical trial. In aim 1, the study staff will determine the biochemical differences between ≥30 day-old versus ≤10 day-old units. In aim 2, the study staff will determine the physiologic effects of the transfused blood in a patient with SCD. Lastly, in aim 3, the study staff will explore the clinical implications of receiving older red cells over a 3 month period.
The objective of this study is to investigate if up to two injections of plerixafor represent a safe and effective strategy to mobilize adequate numbers of CD34+ hematopoietic stem progenitor cells (HSPC) for autologous hematopoietic cell transplantation (HCT) in sickle cell disease (SCD) patients
Recommendations for exercise prescription currently do not exist for individuals with sickle cell anemia (SCA) despite the known impact that SCA-related complications has on physical functioning and fitness. A major barrier to increasing physical activity in SCA is the concern that the well-described inflammatory effects of exercise could precipitate or exacerbate complications such as vaso-occlusive pain or airway bronchoconstriction (i.e. exercise-induced asthma). Although the investigator's preliminary data suggest that increasing physical activity may be beneficial rather than harmful in children with SCA, the pro-inflammatory effects associated with repeated bouts of moderate to vigorous exercise remain poorly understood in this population. The long term goal is to address the safety and health impact of regular exercise in children with SCA. This proposal would help establish the safety of moderate to vigorous intensity exercise in children with SCA and importantly, will inform the design of future clinical trials focused on exercise training as a transformative strategy to improve fitness and overall well-being in this population.
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).
Sickle Cell Disease (SCD) is a rare disease occurring in an estimated 100,000 individuals, often poor and underserved, in the US. Silent and overt strokes contribute significantly to morbidity in adults with SCD, resulting in functional impairment, challenges with school and job performance, and premature death. Five NIH-funded randomized controlled trials have identified therapies to prevent silent and overt strokes in children with SCD, including monthly blood transfusion therapy (for preventing initial and recurrent strokes) and hydroxyurea (for preventing initial strokes). Despite the observation that at least 99% of children with SCD in high-income countries reach adulthood, and approximately 60% of adults will experience one or more strokes (~50% with silent strokes and ~10% with overt strokes), no stroke trials have established therapeutic approaches for adults with SCD. For adults with SCD, inadequate evidence-based guidelines exist for secondary stroke prevention strategies. Applying stroke prevention strategies in children may not be effective for stroke prevention in adults with SCD, particularly given the high rate of co-morbidities. Identifying subgroups of adults with SCD and higher incidence coupled with the contribution of established stroke risk factors in the general population (smoking, diabetes, obesity, renal disease) will provide the requisite data required for the first-ever phase III clinical trials focused on secondary stroke prevention in adults.
Acute vaso-occlusive crisis (VOC) is the most common complication in patients with sickle cell disease (SCD) and pain related to VOC is often inadequately treated. This is a phase II randomized controlled clinical trial evaluating the efficacy of virtual reality technology when added to standard pain management for patients with sickle cell disease who are experiencing acute pain crisis in the ambulatory care setting. Patients will be randomized to receive either standard management only or standard management in addition to virtual reality therapy. The remainder of care for the painful event will continue per institutional standards according to clinical indication, including reassessment and documentation of pain and additional doses of pain medicines by intravenous (IV) or oral route. Pain scores and opioid requirement will be measured and compared across treatment arms, along with the outcomes of discharge from clinic versus admission to the inpatient unit. PRIMARY OBJECTIVE: To assess the efficacy of virtual reality (VR) technology in reducing pain at 30 minutes after intervention during an acute vaso-occlusive crisis in patients with sickle cell disease. Primary endpoint will be change in pain scores in Standard versus VR arms, between the first pain assessment at the time of presentation and the subsequent pain assessments up to 30 minutes after intervention. Secondary Objectives: - To compare total opioid consumption from the time of presentation to the time of discharge from acute care setting in Standard versus VR arms. - To assess the efficacy of virtual reality (VR) technology in reducing pain at 60 minutes after the first IV medication administered or 60 minutes after completion VR during an acute vaso-occlusive crisis in patients with sickle cell disease.
BIOCADRE is a CADRE substudy and aims to characterize more precisely the sickle cell patients with extreme phenotype.