Clinical Trials

The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients

This study is design to assess the effects of an increase in nutritional intake on the bone mineral density of children with sickle cell disease, for 12 months.

The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD) are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized, prospective study of paired cohort will be conducted, with 2 groups of exposed (SCT) and non-exposed (TP) patients.

As part of routine care for SCD, some people are found to have low oxygen levels (≤ 88%) while sleeping, at rest, or with exercise. Testing is done with a small portable device positioned on the finger that measures oxygen levels during sleep, at rest, or following exercise. The investigators start oxygen treatment for people with low levels of oxygen. As a part of this study, the investigators will find out if any changes in cell "stickiness" occur with low oxygen levels (at rest, at night, or with exertion) and if cells become "less sticky" with oxygen treatment.

Stress is known to trigger acute pain crisis of sickle cell disease (SCD). SCD is an inherited blood disorder that afflicts about 100,000 people in the United States, and is among the most common lethal genetic diseases in the United States. Though worldwide in distribution, in the US it is most commonly found in African Americans. Its best known complication is severe, recurrent relentless pain, often known as pain crisis.

Sickle Cell Anemia is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body. In sickle cell disease, as red blood cells circulate and oxygen is released in the circulatory system, the deoxygenated abnormal hemoglobin S can begin to polymerize. When this occurs, the red blood cells can become sticky and elongated.

Iron overload, which can be defined operationally as too much iron in the body, develops as a consequence of too many blood transfusions given, or due to genetic defects hereditary hemochromatosis). Iron accumulates in several organs in the body, such as the heart, liver, endocrine glands (pancreas, thyroid, etc.), and spleen. Excessive iron can damage organs and may even cause death. Iron overload needs to be appropriately monitored and treated to avoid unnecessary morbidity and mortality.

The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of GBT440 compared with placebo in healthy subjects and subjects with sickle cell disease (SCD).

This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.

Patients suffering from Thalassemia or another hemoglobinopathies required regular blood transfusions. The complications and adverse effects of blood transfusions can be classified as immediate and late. Among the immediate effects the most common are allergic reactions and fever, besides congestive heart failure in patients with cardiomyopathy. The late effects are mostly related to blood transmitted infections like HIV or Hepatitis C infections.