View clinical trials related to Sickle Cell Disease.
Filter by:Research Question: In adult SCD patients with CVC, is it feasible and safe to conduct an adequately powered RCT to evaluate the use of rivaroxaban as thromboprophylaxis in this population? Study Design: The study is a vanguard pilot double blind multi-centre randomized controlled trial. Participants with SCD and CVC will be randomized to either rivaroxaban 10mg PO daily or placebo for the duration of CVC in situ or for up to one year, whichever is less. After screening (day -7 to day -1), patients will be followed at day 1, months 3 (+/- 15 days), 6 (+/- 15 days), 9 (+/- 15 days), and 12 (+/- 15 days). Study Objectives: The primary objective is to estimate the proportion of eligible patients who will enroll into a trial of thromboprophylaxis. Secondary objectives include (a) document indications for central venous catheter (CVC), (b) summarize duration of CVC insertion prior to enrollment, (c) estimate adherence to the study drug, (d) estimate proportions of participants being compliant with study procedures, and lost to follow up. Exploratory objectives will assess thrombotic, bleeding, and quality of life outcomes.
This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of hemoglobin in the blood and to reduce or prevent the occurrence of sickle cell pain crises. In addition, the long-term effect of mitapivat on efficacy and safety will be explored in an open-label extension portion.
This study is a pilot, phase II, open-label study of the angiotensin II receptor blocker, losartan, in patients with Sickle Cell Disease (SCD) 6 years or older for 12 months. The investigators will enroll 24 patients with SCD over the course of 1 year with a goal to complete all study procedures in 2 years. The short-term goal is to obtain clinical pilot data regarding the safety and efficacy of losartan in stabilizing or decreasing extracellular volume fraction (ECV) after 12 months of therapy.
This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell transfusions required and increase hemoglobin.
This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.
The purpose of this study is to find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with SCD. Using information from earlier studies and work being done, researchers have developed a strategy to image VOC, using positron emission tomography (PET). The ability to see and measure VOC in SCD patients can help patient care, by showing when and how a VOC is occurring or going to occur. Studying this method will also help in future drug research, as it will allow researchers to deliver promising new medications that target hyper-adhesion and sickling in people with SCD.
The purpose of this study is to evaluate the effect of Virtual Reality induced Hypnosis on patients' pain scores, anxiety and the use of analgesics during a vaso-occlusive crisis
The purpose of this study is to evaluate crovalimab for the treatment of a sickle cell pain crisis (also known as a VOE) that requires hospitalisation in adult and adolescent participants with SCD. The primary objective of this study is safety and will additionally evaluate pharmacokinetics (how crovalimab is processed by your body), pharmacodynamics (how your body reacts to crovalimab) and the preliminary efficacy of crovalimab compared with placebo.
It is usually found that the blood pressure of adults with sickle cell disease is lower than in non-sickle cell patients. On the other hand, three recent prospective studies in children with sickle cell disease show prevalence of hypertension diagnosed by ambulatory blood pressure measurement (ABPM) ranging from 32 to 45% but on small numbers of patients (n = 54 at most). This hypertension appears to affect kidney function and has been previously associated with the risk of hemorrhagic stroke. It is therefore important to know the prevalence of hypertension in children with sickle cell disease and to determine its mechanisms. The factors which could explain this high prevalence are the increase in arterial stiffness and the increase in systemic vascular resistance linked to the alteration of the sympathovagal balance contributing to the regulation of vascular tone. Indeed, a disturbance of this balance with an increase in vasoconstrictor sympathetic tone has already been found. Hypothesis: In a subgroup of sickle cell children there is systemic hypertension (prevalence: main objective) linked to the alteration of the sympathovagal balance already established during sickle cell disease (increase in sympathetic tone and decrease in parasympathetic tone) affecting systemic vascular resistance (secondary pathophysiological objectives).
The investigators will conduct a hybrid type 1 effectiveness implementation trial to assess the effectiveness of acupuncture and guided relaxation on 360 people with Sickle Cell Disease (SCD), while observing and gathering information on implementation in three health systems: University of Illinois Hospital & Health Sciences System, University of Florida Health, and Duke University Health Systems. Each serves a large population with SCD, uses EPIC as their electronic health record, and has a Clinical and Translational Science Award (CTSA), which will help speed the translation of discovery into improved patient care. During the UH3 Implementation Phase, the 3-arm, 3-site randomized controlled trial will follow a quantitative modified SMART design, a pragmatic trial that evaluates adaptive interventions where the guided relaxation and acupuncture interventions respond to patients' characteristics and evolving pain status. The investigators rely on the Consolidated Framework for Implementation Research (CFIR) to plan, execute, and evaluate associated implementation processes. The use of complementary and integrative health (CIH) therapies by those with SCD to reduce pain and opioid use, to help enable them to better cope with their pain, is well known, but there are few studies that evaluate the effectiveness of these therapies, and none that also evaluates the implementation across multiple health care systems and patient populations as this study will. Aim 1: Determine the effectiveness of guided relaxation and acupuncture as compared to usual care in decreasing pain and opioid use for SCD patients. Hypothesis: At 6-weeks, SCD patients randomized to either CIH intervention will have a greater decrease in pain, opioid use, sleep, anxiety, depressive symptoms, and pain catastrophizing compared to SCD patients randomized to usual care. Aim 2: Identify the best adaptive intervention for improved outcomes by documenting outcomes among adaptive intervention sequences: (1) initiate guided relaxation and switch to acupuncture for non-responders at midpoint; (2) initiate guided relaxation and continue with guided relaxation for non-responders at midpoint; (3) initiate acupuncture and switch to guided relaxation for non-responders at midpoint or (4) initiate acupuncture and continue with acupuncture for non-responders at midpoint. Aim 3: Explore differences in response to the adaptive interventions by age and sex. Aim 4: Identify implementation facilitators, challenges, and solutions for structures and processes that contribute to the seamless integration of CIH therapies into the 3 health systems by conducting individual interviews with participants in the intervention group who responded to the intervention and those who did not. The investigators will also conduct focus groups with hospital personnel at 4 timepoints.