Sickle Cell Disease Clinical Trial
Official title:
A Phase I Dose-Ranging Study of Oral L-citrulline in Steady-State Sickle Cell
Sickle cell disease is a genetic red blood cell disorder that can result in blocking of the
small blood vessels from sickle shaped red blood cells. This causes pain, the main feature of
sickle cell disease. Also, low amounts of nitric oxide can occur in sickle cell disease, a
substance important for widening the blood vessel wall and therefore preventing blockage of
the small blood vessels.
Citrulline is a drug that is known to increase nitric oxide. This is a phase I study of
citrulline given by mouth to evaluate the safety, tolerability and appropriate dosing of this
medication for individuals with sickle cell disease.
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