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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center Health System
Pittsburgh, Pennsylvania, United States
PK and PD responses to L-citrulline
Endpoints will be determined as change from baseline
Assessment of safety and tolerability of L-citrulline,
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