View clinical trials related to Sickle Cell Disease.
Filter by:The investigators' primary objective is to study prevalences of myocardial iron overload, defined as a cardiac T2*< 20 ms, in 3 populations of multiply transfused patients, affected with thalassemia, sickle cell disease, and myelodysplasia.
The proposed research study is a cross-sectional study enrolling young children with sickle cell disease between 5 and 12 years of age. They will be screened as outpatients for consent to perform pulmonary function testing (PFT) and echocardiography. In addition, the degree of bronchodilator response will be assessed at each session. To estimate presence of pulmonary hypertension, echocardiography will be performed at the time of PFT measures. Study Design: 1. Enroll children aged 5 to 12 years of age with sickle cell disease (HbSS, HbSC, HbS beta plus thalassemia, HbS beta zero thalassemia, and HbS OArab) who are established patients within the Duke Pediatric Sickle Cell Clinic. 2. Perform a chart review of all enrolled subjects to obtain specific details regarding birth history, nutritional status (weight, height), family history, sickle cell genotype, parental smoking history, recent laboratory parameters, parental smoking history, any concurrent conditions (atopy, asthma, airway anomaly), history of sickle cell complications and prescribed medications. 3. Perform spirometry and plethysmography with the administration of albuterol. 4. Before or after completion the PFT session, the patient will have echocardiography in the PFT lab area 5. Using medical record information, determine number of hospitalizations for any pulmonary symptoms indicative of acute chest syndrome (ACS) (dyspnea, fever, wheezing, hypoxia, cough, chest pain). In addition, we will track any respiratory or cardiac symptoms or therapies for each subject 6 years after enrollment up to age 18 years using the registry. 6. As standard of care, refer any child identified as having lung disease or pulmonary hypertension to a pediatric pulmonologist and/or cardiologist for monitoring, treatment and ongoing care.
The purpose of this study was to determine whether the investigational drug SelG1 when given to sickle cell disease patients either taking or not taking hydroxyurea was effective in preventing or reducing the occurrence of pain crises. SelG1 prevents various cells in the bloodstream from sticking together. By stopping these cell-cell interactions, SelG1 may prevent small blood vessels from becoming blocked and therefore reduce the occurrence and severity of pain crises. Other effects of SelG1 was evaluated, as well as the safety of the drug and how long it stayed in the blood stream. Funding Source - FDA Office of Orphan Products Development (OOPD)
Asthma and sickle cell disease each are serious medical problems. People with asthma have difficulty breathing, wheeze (a whistling noise when breathing), cough, produce sputum or phlegm, and have inflammation (swelling, irritation, redness) and narrowing of the bronchial tubes. When a person has both asthma and sickle cell disease together, more serious medical problems can occur such as having acute chest syndrome and pain episodes more often. It is sometimes hard to diagnose asthma in a person with sickle cell disease because sickle cell disease can also cause lung problems. The purpose of this study is to see if the investigators can better understand asthma when it occurs in a person who has sickle cell disease. The investigators will do this by taking a blood, urine, and saliva sample. The blood and urine samples will be analyzed for chemicals and DNA (genes). Certain genes can cause patients to have sickle cell disease or asthma. The investigators will use the saliva sample for future studies to compare the results from the blood testing with saliva. The investigator's long-term goal is to make sure people who have asthma and sickle cell disease are getting the best asthma treatments. The investigator's hypothesis is that the analysis of the blood, urine and saliva using a method called, metabolomics, may identify a unique asthma signature in children with sickle cell disease which may lead to targeted treatments.
This protocol will be investigating the use of stem cell transplantation, in related donors, to cure sickle cell disease. Sickle cell disease is a recessive disorder caused by a point mutation that results in the substitution of valine for glutamic acid at the sixth position in the B-chain of hemoglobin. This leads to sickling of the red blood cells under many conditions, such as hypoxia, dehydration, and hyperthermia. The sickling leads to vaso-occlusion, which causes irreversible damage in almost all systems in the body, including the central nervous system (CNS), lungs, heart, bones, eyes, liver, and kidneys.
The primary aim of this study is to evaluate the effect of the drug N-Acetylcysteine on the frequency of pain in daily life in patients with Sickle Cell Disease (SCD). Pain is an invalidating hallmark of this disease and has a considerable impact on the Quality of Life of patients and the medical health care system. Oxidative stress is hypothesized to play a central role in its pathophysiology. In pilot studies the administration of N-Acetylcysteine (NAC) resulted in a reduction of oxidative stress. Moreover, administration of NAC seemed to decrease hospitalization for painful crises in a small pilot study in patients with SCD. This study will be performed as a multicenter, randomized, controlled trial where patients will be treated with either NAC or placebo for a period of 6 months. The investigators expect that NAC can reduce the frequency of pain in patients with SCD, thereby improving their quality of life and participation in society.
The purpose of this study is to compare the safety of SANGUINATE™ versus Hydroxyurea in patients suffering from Sickle Cell Disease.
The objective of this study is to evaluate the pharmacokinetics of deferiprone and its 3-O-glucuronide metabolite following administration of a single 1500 mg dose of Ferriprox in patients with sickle cell disease.
The study will seek to enroll 100 sickle cell or thalassemia patients who are age 12 or older who have access to a smartphone or tablet with Internet access daily. The study will evaluate patient-reported comfort level with using a mobile device to record their pain levels, as well as adherence to recording these levels daily. The study will track patients' assessment of their pain, actions taken, and outcomes related to pain management and provider involvement. This study will attempt to collect information about differences in the use of two traditional pain assessment modes (verbal scale and paper) versus the use of a pain assessment tool on a mobile device in the form of a smartphone, tablet, or iPad with an Android or iOS operating system.
Given large absolute numbers of individuals with sickle cell disease in Nigeria, hydroxyurea therapy for all individuals with sickle cell disease may not be initially feasible; however, a targeted strategy of hydroxyurea use for primary prevention of strokes is an alternative to the standard therapy (observation) for high-risk individuals. The investigators propose a feasibility study, Sickle Cell Disease - Stroke Prevention in Nigeria (SPIN) Trial, to determine whether hydroxyurea can be used for primary prevention of strokes in Nigerian children with sickle cell anemia.