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Sickle Cell Disease clinical trials

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NCT ID: NCT02447874 Recruiting - Sickle Cell Disease Clinical Trials

Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease

R34 pK/PD
Start date: May 2015
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications or decrease length of hospital stay or emergency department visit.

NCT ID: NCT02447627 Completed - Healthy Clinical Trials

Study of Methodologies to Measure Blood Flow and Oxygenation in Adults With Sickle Cell Disease

Start date: June 2015
Phase:
Study type: Observational

The purpose of the study is to determine whether imaging techniques, such as magnetic resonance imaging (MRI), near infrared spectroscopy (NIRS), laser speckle contrast imaging (LSCI), and optical imaging (OI), can detect differences in blood flow and oxygen levels in different organ systems of participants with sickle cell disease (SCD). Differences in blood flow and oxygen levels detected by these techniques will be evaluated to determine their utility as biomarkers of clinical disease pathophysiology.

NCT ID: NCT02443545 Terminated - Sickle Cell Disease Clinical Trials

Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias

Start date: May 21, 2015
Phase: Phase 4
Study type: Interventional

This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions.

NCT ID: NCT02435901 Completed - Sickle Cell Disease Clinical Trials

HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity

Start date: December 2008
Phase: Phase 1/Phase 2
Study type: Interventional

This study will evaluate the use of reduced intensity conditioning regimen in patients with high risk hemoglobinopathy Sickle Cell and B-Thalassemia Major in combination with standard immunosuppressive medications, followed by a routine stem cell transplant in order to assess whether or not it is as effective as myeloablative high dose chemotherapy and transplant.

NCT ID: NCT02433158 Terminated - Sickle Cell Disease Clinical Trials

Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease

Start date: December 16, 2015
Phase: Phase 3
Study type: Interventional

This is an open label extension study in subjects with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002).

NCT ID: NCT02411396 Completed - Sickle Cell Disease Clinical Trials

Comparison of Patient Centered Outcomes for People With Sickle Cell Disease in the Acute Care Setting

ESCAPED
Start date: April 2015
Phase:
Study type: Observational

The Emergency Department has been the standard location where patients with Sickle Cell Disease (SCD) go to seek care for the treatment of acute painful events. Vaso- Occlusive Crisis (VOC) is the most common complication of SCD, The purpose of this study is to compare patient centered outcomes for patients being treated for an uncomplicated VOC in Infusion Centers (IC) and Emergency Departments (ED) in four locations around the United States.

NCT ID: NCT02410811 Completed - Sickle Cell Disease Clinical Trials

Novel Cardiac Magnetic Resonance Imaging to Define a Unique Restrictive Cardiomyopathy in Sickle Cell Disease

Start date: January 31, 2014
Phase:
Study type: Observational

The purpose of this study is to use cardiac magnetic resonance imaging (CMR) and echocardiographic tissue Doppler imaging to demonstrate a unique restrictive cardiomyopathy of sickle cell disease. The investigators will characterize its frequency and how it might change (e.g., presence/absence and severity) over a 2-year period.

NCT ID: NCT02394899 Completed - Sickle Cell Disease Clinical Trials

Improving Parental Psychosocial Functioning and Early Developmental Outcomes in Children With Sickle Cell Disease

Start date: October 2014
Phase: N/A
Study type: Interventional

There are all significant risk factors for poor early cognitive development and, as such, neurocognitive deficits have been demonstrated in pre-school children with sickle cell disease (SCD). This project assesses the efficacy of using an evidence-based early stimulation program, combined with components to help parents cope with stress, delivered during six routine monthly clinic visits to parents of children with sickle cell disease. It is hoped that this innovation will improve parental psychological outcomes, as well as child developmental outcomes.

NCT ID: NCT02394431 Completed - Sickle Cell Disease Clinical Trials

Added Value of Speckle Tracking in the Evaluation of Patients With Sickle Cell Disease

Start date: November 2013
Phase: N/A
Study type: Observational

Sickle Cell Disease is a serious disease that is life-threatening for patients being homozygous for the SS form or heterozygous for the SC or βthal forms. The CHU Brugmann hospital currently regularly treats about 70 homozygous adult patients and this number is in constant augmentation. Sickle cell disease patients may develop a cardiomyopathy due to chronic anemia, the haemosiderosis risk or, less frequently, to coronary vaso-occlusive damages. The hypervolemia in patients with sickle cell disease causes an overestimation of the ejected left ventricular fraction measured by echocardiography, this parameter being very dependent of the blood volume.It has already been shown that the left ventricular ejection fraction was normal in most patients with sickle cell disease, but that its evaluation by parameters independent from the blood volume showed the existence of a dysfunction. Myocardial strain, as measured by speckle tracking, is a echographic evaluation method of the cardiac function, independent of the blood volume. This technique hasn't been used much in sickle cell disease patients. A study using 3D speckle tracking on a limited number of sickle cell disease patients failed to show a strain anomaly. Moreover, the study highlighted a higher global longitudinal strain in this patient population. The investigators find these data hard to explain and in contradiction with previous studies using other cardiac function evaluation techniques, independent from the blood volume. The primary goal of this study is thus - to study the longitudinal strain by 2D echography - to determine if anomalies of the longitudinal strain exist in sickle cell disease patients with a normal ejected left ventricular fraction, compared to a control group of healthy patients. The secondary goal of this study is to correlate, inside the sickle cell disease group, the possible strain anomalies with biological gravity parameters of the disease.

NCT ID: NCT02386657 Completed - Sickle Cell Disease Clinical Trials

Single-center Prospective Evaluation of Sickle Cell Patient Care in the CHU Brugmann Emergency Department

Start date: November 2012
Phase: N/A
Study type: Observational

Sickle Cell Disease is a serious disease that is life-threatening for patients being homozygous for the SS form or heterozygous for the SC or bthal forms. The CHU Brugmann hospital currently regularly treats about 70 homozygous adult patients and this number is in constant augmentation. The age average of the patients is below 30. The hospital developed a close collaboration with the Queen Fabiola Kids University Hospital to optimize the transition of young sickle cell patients from the pediatric to the adult network. The emergency care of sickle cell patients remains a source of worry. Even with a correct treatment (Hydroxy-urea or exsanguineous transfusions), patients suffer from frequent sickle cell disease crisis when stress or infection cause hemolysis. The pain level is intolerable and causes emergency hospital admission (2 to 3 crisis per patient per year on average). The crisis are more frequent with poor compliance to the treatments. There are several obstacles to the rapid and optimal management of these patients: - fear of causing addiction to heavy pain releaf products (high dosis of morphine) - lack of biological parameters for the determination of the crisis severity. The prognostic value of the lactate dehydrogenase (LDH) level in a vaso-occlusive crisis was recently stressed while activation of the coagulation, translated by the elevation of various parameters including the rate of DD dimers, seemed associated with clinical complications. The deleterious role of increased oxidative stress has also been recently demonstrated in patients with sickle cell disease, opening new therapeutic avenues. This study aims to prospectively evaluate the management of sickle cell patients being admitted in the emergency department for a vaso-occlusive crisis. The level of satisfaction of the patients will be measured. The investigators will also evaluate the predictive value of several routine biological parameters regarding the severity of the crisis, including the values of nitrous albumin (PNA) as marker of oxydative stress. This last dosage will be made in collaboration with the team of Dr Wayenberg and Pr Bottari in Grenoble.