View clinical trials related to Anemia, Sickle Cell.
Filter by:The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK), and pharmacodynamics (i.e., how the body absorbs, distributes, breaks down, and excretes) of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in participants with SCD, following single and multiple ascending doses.
This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age) with sickle cell disease (SCD). Participants will be randomized to receive inclacumab or placebo.
This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.
The aim of this study is to collect and analyze retrospective data on Oxbryta in a real-world setting. This is a multicenter, retrospective data collection and analysis study to characterize health outcomes in approximately 300 patients with SCD who have been treated with Oxbryta as part of their usual care. Any patient with SCD who received Oxbryta treatment for at least 2 weeks as part of their usual care according to the Oxbryta US Prescribing Information (USPI) is eligible to participate. Study data from 1 year before and up to 1 year after the first dose of Oxbryta will be entered in case report forms (CRFs) via an electronic data capture (EDC) system by the study staff.
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD). Participants will be randomized to receive either inclacumab or placebo.
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 determine feasibility and potential benefits of providing a passport card with a summary of neurocognitive feedback results to families of patients with sickle cell disease. Given recent literature suggesting the need to be conscious of health literacy in populations with low socioeconomic status, this project is intended to provide a more health-literate appropriate format of neurocognitive evaluation feedback in the context of a routine screening program offered as a standard of care in the CHW pediatric sickle cell disease clinic. The specific aims is (1) to evaluate differences in caregiver understanding of neurocognitive report findings when provided with a health-literate passport card compared to control group and (2) to evaluate differences in follow-through on neurocognitive report recommendations when provided with a health-literate passport card compared to control group.
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).