120
Participants
Start Date
July 25, 2025
Primary Completion Date
June 30, 2027
Study Completion Date
June 30, 2029
L-citrulline
This intervention uses intravenous L-citrulline as an acute treatment for vaso-occlusive crisis (VOC) in sickle cell disease, administered as a 50 mg/kg IV bolus followed by a 9 mg/kg/hr continuous infusion. Unlike other studies that evaluate oral citrulline or chronic VOC prevention, this study focuses on real-time symptom resolution in the emergency department setting by targeting impaired nitric oxide production and vasoconstriction. The selected dosing regimen was optimized in prior studies to achieve a therapeutic plasma concentration associated with improved pain scores and reduced opioid use, distinguishing it mechanistically and clinically from other interventions.
Placebo
Placebo intravenous (IV) treatment designed to match the appearance and administration schedule of L-citrulline
Children's National Hospital, Washington D.C.
Johns Hopkins Hospital, Baltimore
East Carolina (University of North Carolina), Greenville
Emory University, Atlanta
University of Miami, Coral Gables
Children's Hospital of Michigan, Detroit
Lead Sponsor
Asklepion Pharmaceuticals, LLC
INDUSTRY