A Study of Intravenous L-Citrulline in Patients Aged 6-21 Years Old With Sickle Cell Disease Presenting to Emergency Departments in Acute Vaso-Occlusive Crisis

PHASE3Not yet recruitingINTERVENTIONAL
Enrollment

120

Participants

Timeline

Start Date

July 25, 2025

Primary Completion Date

June 30, 2027

Study Completion Date

June 30, 2029

Conditions
Sickle Cell Disease
Interventions
DRUG

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.

OTHER

Placebo

Placebo intravenous (IV) treatment designed to match the appearance and administration schedule of L-citrulline

Trial Locations (6)

20010

Children's National Hospital, Washington D.C.

21287

Johns Hopkins Hospital, Baltimore

27858

East Carolina (University of North Carolina), Greenville

30322

Emory University, Atlanta

33146

University of Miami, Coral Gables

48201

Children's Hospital of Michigan, Detroit

Sponsors
All Listed Sponsors
lead

Asklepion Pharmaceuticals, LLC

INDUSTRY