View clinical trials related to Sickle Cell Disease.
Filter by:The proposed study is a Phase 2 multi-center, randomized, double-blind, placebo-controlled, parallel groups study aimed to evaluate the safety, tolerability and the efficacy of riociguat compared with placebo in patients with sickle cell disease (SCD).
Acute chest syndrome (ACS), a lung complication in sickle cell disease (SCD), is the second most common cause of hospitalization and leading cause of death in SCD. ACS is associated with airway inflammation, and a major cause is pulmonary infection from atypical organisms. To date, there are no drugs available to reduce inflammation and risk of recurrent ACS. Macrolides are a group of antibiotics that exert immunomodulatory and anti-inflammatory actions both in vitro and in vivo. In addition, macrolides reduce bacterial burden in the airway of atypical organisms, all of which play an important role in the pathophysiology of ACS. Numerous studies have evaluated macrolide prophylaxis in conditions associated with lung inflammation, such as cystic fibrosis, asthma, bronchiectasis etc., and high quality evidence have found macrolides to be beneficial as a disease modifying agent that leads to improvement in airway inflammation, reduced pulmonary exacerbations and improved lung function. The investigators hypothesize that azithromycin prophylaxis is well tolerated and has the potential to reduce inflammation and improve lung outcome in children with SCD with a history of ACS. A prospective, single arm, open label feasibility study of azithromycin prophylaxis will be performed in children with SCD with a history ACS with the specific aim to examine the feasibility, safety and tolerability of azithromycin prophylaxis administration in participants with SCD , and to examine whether azithromycin prophylaxis has the potential to improve lung outcome. In addition, this study will determine whether azithromycin prophylaxis reduces inflammation in participants with SCD with a history of ACS.
This pilot study will assess the effects of a brief laboratory-based guided imagery procedure on responses to pain in patients with sickle cell disease (SCD) and healthy controls.
The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow stem cell implantation for the treatment of leg ulcer in adult patients with sickle cell disease.
Symptomatic sickle cell disease is worldwide the most frequent cause for hereditary hemolytic anemia with recurrent pain crisis. Hemolysis, vaso- occlusive and pain crises are hallmarks of this disease and are causative for an important socio-economic burden worldwide, especially in Africa. Aside from allogenic stem cell transplantation, which is rarely available and very expensive, at present there is no curative treatment for patients with sickle cell disease (SCD). The current standard of care includes treatment with hydroxycarbamide and symptomatic care such as transfusions, antibiotic/analgesic treatment. This study has the aim to study the safety and tolerability of Memantin in patients with sickle cell disease.
Sickle cell disease is a genetic disease responsible for an abnormal hemoglobin.The anomaly has several consequences: a low hemoglobin rate (chronic anemia), plugs formed by red blood cells in blood vessels (extremely painful vaso-occlusive crises) and greater susceptibility to infections. Patients with this disease should be monitored medically continuously from birth. At adulthood, they will pass from a pediatric medical care system to an adult medical care system.This transition can be experienced with more or less ease, depending on the organization within the pediatric and adult hospitals. This questionnaire aims to assess the quality of the transition between pediatric and adult services.The investigators want to better estimate hospital work and improve the quality of care for this type of patients, throughout their entire medical history.
The objective of this study is to assess the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects receiving SC411 compared to those subjects receiving placebo.
The overall goal of the IQ-MAPLE project is to improve the quality of care provided to patients with several heart, lung and blood conditions by facilitating more accurate and complete problem list documentation. In the first aim, the investigators will design and validate a series of problem inference algorithms, using rule-based techniques on structured data in the electronic health record (EHR) and natural language processing on unstructured data. Both of these techniques will yield candidate problems that the patient is likely to have, and the results will be integrated. In Aim 2, the investigators will design clinical decision support interventions in the EHRs of the four study sites to alert physicians when a candidate problem is detected that is missing from the patient's problem list - the clinician will then be able to accept the alert and add the problem, override the alert, or ignore it entirely. In Aim 3, the investigators will conduct a randomized trial and evaluate the effect of the problem list alert on three endpoints: alert acceptance, problem list addition rate and clinical quality.
Sickle cell anemia is a homozygous genetic disease with high prevalence in Brazil. There are changes in conformation and physicochemical properties of red cells that generate varied clinical manifestations among which is chronic hemolytic anemia, cardiovascular diseases, fever, splenic sequestration and usually painful crises. Women with sickle cell anemia have high maternal-fetal and neonatal morbidity and mortality. During pregnancy, there is intensification of maternal anemia, episodes of painful crises; and also, more obstetric risks, such as pre-eclampsia, thromboembolism and hemorrhage. Thus, there is the need for adequate reproductive family planning for this population conducted mainly through hormonal contraception. The World Health Organization recommends that all contraceptive methods may be prescribed for people with sickle cell anemia women, being the progestogen-only contraceptive methods the most indicated due to no changes in venous or arterial thrombosis. Nevertheless, there is need for further scientific evidence as the best contraceptive choice among women with sickle cell anemia in relation to safety, adhesion and reduction of pain crises. The objective of this study is to evaluate the clinical effect through safety of etonogestrel-releasing contraceptive implant in women with sickle cell anemia during twelve months.
The use of analgesics can lead to cases of drug abuse and dependence. It can also cause pseudo-addiction in patients suffering from pain. What is the actual situation in patients suffering from severe sickle-cell disease, exposed to acute pain during vaso-occlusive crises? Evaluation of the use of analgesics, on the basis of Diagnostic and Statistical Manual of Mental Disorders criteria for substance abuse and dependence, makes it possible to differentiate the symptoms occurring only in a context of pain, in the aim of managing the pain, and thus describing pseudo-addiction, from symptoms also occurring when there is no pain, and more in favour of true addiction. Currently there is no data available in France on this problem, and no studies have been carried out in children or adolescents with sickle-cell disease. The purpose of the study is to evaluate the prevalence of problematic use of equimolar mixture of oxygen and nitrous oxide and other analgesic drugs in a population of subjects with severe sickle-cell disease in France. PHEDRE (Pharmacodépendance Et DREpanocytose-drug dependence and sickle-cell disease) is an observational, descriptive and transversal study. Patients under the age of 26 with sickle-cell disease are included in the study by the doctors looking after them in sickle-cell disease centres. The patients are then contacted by a trained researcher for a telephone interview, including an evaluation of the Diagnostic and Statistical Manual of Mental Disorders criteria for abuse and dependence to equimolar mixture of oxygen and nitrous oxide and for each of the analgesic drugs taken by the patient. The data are also completed using the subject's medical record. This study will make it possible to provide an initial quantitative and qualitative evaluation of problematic use of equimolar mixture of oxygen and nitrous oxide and analgesic drugs in the sickle-cell disease population. The results will be used firstly to provide additional data essential for monitoring the risk of overdose, abuse, dependence and misuse of these products, and to begin awareness-raising and to provide information for health care professionals, in order to significantly improve the management of sickle-cell disease-related pain.