View clinical trials related to Sickle Cell Disease.
Filter by:Background: - Assessing pain levels is important to improve treatments for different illnesses. Most pain rating scales are used to determine pain levels in adults. Pain is also a common symptom among children who have cancer. Those who have genetic conditions that may lead to cancer may also have pain symptoms. However, the pain scales used for adults have not been fully tested in children and young adults. As a result, they may not be as accurate. Researchers want to test pain rating scales in children and young adults who have cancer and genetic conditions that can lead to cancer. Objectives: - To study the effectiveness of pain rating scales given to children and adults with Sickle Cell Disease (SCD),cancer, and related genetic conditions. Eligibility: - Adults 18 and 34 years of age and older who have SCD, cancer, or other genetic conditions that can lead to cancer. Design: - Participants with SCD, cancer or related genetic conditions will fill out four questionnaires. These questionnaires will ask about pain levels and how much pain interferes with daily life. - Pain treatments will not be provided as part of this study.
The objective of this study is to design, implement and test quality improvement measures to improve the care of adults with sickle cell disease in the emergency department.
Background: - Small blood vessels (microvessels) in many different organs are affected by diseases such as diabetes and atherosclerosis. These microvessels may also be abnormal in people who have sickle cell disease. Stiffness of the red blood cells leads to problems in the microvessels of the heart and kidneys. However, these problems may not be detected until these organs are severely affected. Researchers want to study problems with microvessels in people with and without sickle cell disease. Objectives: - To study how microvessels in the heart and other organs are affected by sickle cell disease. Eligibility: - Individuals at least 18 years of age who have sickle cell disease. - Healthy volunteers at least 18 years of age. Design: - Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. - All participants will have about 3 to 4 hours of testing for the study. Participants with sickle cell disease who are having a pain crisis at the time they enter the study may be asked to have the testing again when the crisis is over. The repeat testing will occur at least 4 weeks after the pain crisis ends. - All participants will have the following tests: - Blood draws to check kidney and liver function, and other blood tests - Measure of blood flow in the brachial (upper arm) artery - Heart ultrasound - Ultrasound scans of arm muscles to study blood flow - Ultrasound scans after taking vasodilators to increase blood flow - Healthy volunteers will also have a magnetic resonance imaging scan. It will show blood flow in the heart. This scan will involve another dose of a vasodilator.
The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of Aes-103 (active ingredient 5-hydroxymethyl-2-furfural [5-HMF]) compared with placebo in subjects with stable sickle cell disease (SCD). Safety will be measured by monitoring adverse events (AEs), electrocardiograms (ECGs), vital signs, and laboratory values. Pharmacokinetics of Aes-103 will be measured over time in plasma, red blood cell hemolysate and binding of Aes-103 to hemoglobin. Pharmacodynamic effects will be assessed by measuring partial pressure of oxygen at which 50% of hemoglobin is saturated with oxygen (p50) while breathing normal air, blood oxygen levels (SpO2), ex-vivo antisickling effects in a hypoxic environment, and by imaging related changes in tissue blood flow and oxygen levels.
Exposure to secondhand smoke is a leading preventable cause of child morbidity and mortality, and the adverse health consequences of secondhand smoke are magnified among youngsters with cancer and sickle cell disease. Current methods for measuring secondhand smoke exposure (SHSe) rely on retrospective reports over extended time periods that are subject to recall errors and systematic inaccuracies in reporting and often do not include the youngster as the primary informant. These methods may underestimate the extent of cumulative SHSe and are not well suited to capturing exposure over time and across settings where young people frequent. More appealing methods that engage youngsters to better monitor tobacco smoke in their environment are warranted. The study will examine the feasibility of cell phone texting to obtain measures of secondhand smoke exposure (SHSe) in children treated for cancer or sickle cell disease (SCD).
Sickle cell disease (SCD) is a debilitating genetic disorder affecting 70,000-100,000 Americans. It is frequently associated with very serious medical complications. For children with SCD, successfully transitioning to adult care is a vital step in ensuring continuity of care, managing their disease, and improving their health outcomes. Transition programs have been created to facilitate the transition process. However, few studies have assessed transition readiness and whether transition program components meet the transition needs of patients and families. The purpose of this study is to explore transitioning from pediatric care to adult care and to assess components of the SJCRH SCD Transition Program from three perspectives: adolescents with SCD, their caregivers, and young adults with SCD who have transitioned to adult care. Data collection methods will include focus groups, questionnaires, and checklists. Qualitative data analysis procedures will be used to examine the data.
Background: - Many people with sickle cell disease have repeated episodes of severe pain that lasts for days, requiring hospital care. These episodes, called pain crises, may be caused by changes in blood flow. Researchers want to study blood flow in people with sickle cell disease who are having a pain crisis and compare it with their blood flow after the pain crisis has resolved. They also want to compare these measurements against blood flow in healthy people who do not have sickle cell disease. Objectives: - To study whether changes in blood flow cause pain crises in people with sickle cell disease. Eligibility: - Individuals at least 18 years of age who have sickle cell disease and are being treated for a pain crisis. - Individuals at least 18 years of age who have sickle cell disease and are not experiencing a pain crisis. - Healthy volunteers matched by age and gender with the participants who have sickle cell disease. Design: - Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. - Participants having a sickle cell pain crisis will have two visits, one during the crisis and one about 4 weeks after the crisis has resolved. - Participants not having a sickle cell pain crisis will have one or two study visits. Blood samples will be collected during at least one of these visits. - Healthy volunteers will have one or two study visits. Blood samples will be collected during at least one of these visits. - During each visit for all participants, cameras and blood flow monitoring equipment will be used to measure blood flow in the forearm. sickle cell disease.
In Sickle cell disease children, sleep respiratory abnormalities are risk factors for vaso-occlusive complications, as well as cerebral vasculopathy. A 18 months follow-up children with sickle cell disease evaluating sleep respiratory problems frequency and etiology, as well as their influence on sickle cell disease complications.
This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features. The primary goal of this multi-center Phase II study is to determine the safety and feasibility of a conditioning regimen consisting of busulfan (Bu)/ fludarabine (Flu)/ anti-thymocyte globulin (ATG) in adult patients with severe SCD. A two-component design will be used for this study. The first component will be restricted to patients who have an HLA-identical sibling donor. Five patients will be transplanted during the first component of the study. If no more than 2 of the first 5 patients experience unacceptable toxicity, including death, within the first six months after transplantation, then the safety of the regimen will be considered promising in adult SCD patients. The second component will include patients who have a related or an unrelated human leukocyte antigen (HLA) matched donor. Up to 15 additional patients will be transplanted in this component of the study which will evaluate the safety and feasibility of unrelated donor hematopoietic cell transplantation (HCT) in adults with SCD. Data related to study endpoints for 1 year after transplantation will be collected; however, participating centers will be encouraged to conduct long-term follow-up evaluations of patients according to standard institutional guidelines. The purpose of this pilot safety trial is to see if this approach is feasible and meets accrual goals lending support to the development of a subsequent full scale investigation of HCT and comparing outcomes in a transplantation cohort to a control cohort of adults eligible for, but unwilling or unable to receive HCT treated by supportive therapy with a primary endpoint of five years survival for this full scale comparative trial.
Background: - Some people with sickle cell disease have different health problems than others. This may be related to how easily and frequently the red blood cells break apart in the blood. Researchers want to test breath and blood samples from people with sickle cell disease to look for very small amounts of carbon monoxide, which is produced when red blood cells break apart. They will compare these results with breath samples from healthy volunteers. Studying different levels of carbon monoxide may help predict what health problems a person with sickle cell disease may get. It may also provide more information on possible treatments. Objectives: - To study breath carbon monoxide levels and their possible relation to the severity of sickle cell disease. Eligibility: - Individuals at least 18 years of age with sickle cell disease. - Healthy volunteers who are matched for age, sex, and race with the sickle cell disease group. Design: - Participants will be screened with a medical history. - Participants with sickle cell disease will provide a blood sample and have a heart function test. They will also breathe into a bag to provide an exhaled breath sample. - Healthy volunteers will provide an exhaled breath sample. - No treatment or care will be provided as part of this study.