View clinical trials related to Sickle Cell Disease.
Filter by:Sickle cell disease (SCD) is an inherited disorder of the red blood cell. It is now the commonest genetic disorder in the UK and of childhood stroke, with up to 40% of children having a stroke (clinical or picked up on a scan) by school age. Patients are prone to develop acute crises necessitating hospital admission and resulting in long-term complications. Such events result in considerable morbidity, disability and mortality with its consequent burden on patients, families, the health service and society as a whole. Doctors have very little ability to predict who will get ill and when and so it is very difficult to known when and how to administer treatments. Furthermore there is very little in the way of treatments available and the mainstay of prevention is a chronic blood transfusion programme which is expensive, requires time off work and school and can be fraught with complications. This, in a population who is frequently educationally and socially disadvantaged at the outset. Recent evidence in sickle cell disease and other diseases that have similar underlying processes, points towards the importance of microparticles (circulating broken pieces of cells) and the coagulation system as being important. By comparing levels of these particles and molecules in patients with those found in healthy volunteers and with other measures known to be important, this study hopes to identify their role so as to improve the management of these patients and potentially to lead the way for new therapies. Participants will be required to donate a small amount of blood (1 teaspoon in the very young, two in older children and adults). The investigators aim to take this sample where possible when people are having a blood test in any case.
The purpose of the study is to determine the effectiveness of LOVAZA (fish oil capsules) to decrease inflammation in children and adolescents with Sickle Cell Disease (SCD). It has been found that besides the damage caused by sickle red blood cells themselves, the inflammatory response that occurs in SCD patients could potentially play a significant role in the occurrence of painful episodes or pain crises. The investigators will also study whether the subject/caregiver feels that there is an improvement in the child's quality of life by taking the medication. Besides the effect of LOVAZA on inflammation,the investigators are also testing whether the drug will have a beneficial effect on blood clotting ability (which is known to be increased in SCD) and on the anemia (low red blood cells) that is part of the disease entity.
To evaluate PK and PD responses to L-citrulline given orally for four weeks to patients with sickle cell disease who are otherwise healthy.