PK and PD Responses to Oral L-Citrulline in Patients With Sickle Cell Disease
Recruitment status was Not yet recruiting
To evaluate PK and PD responses to L-citrulline given orally for four weeks to patients with sickle cell disease who are otherwise healthy.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics/Dynamics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase 1 Pharmacokinetic and Pharmacodynamic Responses to Oral L-Citrulline in Patients With Sickle Cell Disease|
- PK and PD responses to L-citrulline
- Endpoints will be determined as change from baseline
- Assessment of safety and tolerability of L-citrulline,
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.
|United States, Pennsylvania|
|Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center Health System||Not yet recruiting|
|Pittsburgh, Pennsylvania, United States, 15213|
|Contact: Lakshmanan Krishnamurti, MD 412-692-7192|
|Contact: Sidney Morris, PhD (412) 648-9338|
|Principal Investigator:||Lakshmanam Krishnamurti, MD||Children's Hospital of Pittsburgh, Univ. of Pittsburgh Medical Center|