Sickle Cell Disease Clinical Trial
Official title:
Phase 1 Pharmacokinetic and Pharmacodynamic Responses to Oral L-Citrulline in Patients With Sickle Cell Disease
To evaluate PK and PD responses to L-citrulline given orally for four weeks to patients with sickle cell disease who are otherwise healthy.
Phase 1, single center, open label, multiple-dose study consisting of a screening period
followed by four weeks of treatment. At least 12 male and female patients, 10 years of age
or older, with sickle cell disease but otherwise healthy, will be enrolled in the study.
PK measurements include plasma concentrations of L-citrulline, L-arginine, L-ornithine, and
L-proline following the first dose of orally administered L-citrulline and after four weeks
of twice daily administration of the drug.
PD measurements include intercellular and vascular adhesion molecules (ICAM, VCAM, and
E-selectin), surrogate markers of sickle cell disease activity, and PAT, a measurement of
vascular function.
;
Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics/Dynamics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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