NCT02659644

A Study of Oral L-citrulline in Sickle Cell Disease

A Phase I Dose-Ranging Study of Oral L-citrulline in Steady-State Sickle Cell

• Status: Withdrawn
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: University of Mississippi Medical Center · Academic / Other
• Sex: All
• Age: 10 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

Sickle cell disease is a genetic red blood cell disorder that can result in blocking of the small blood vessels from sickle shaped red blood cells. This causes pain, the main feature of sickle cell disease. Also, low amounts of nitric oxide can occur in sickle cell disease, a substance important for widening the blood vessel wall and therefore preventing blockage of the small blood vessels. Citrulline is a drug that is known to increase nitric oxide. This is a phase I study of citrulline given by mouth to evaluate the safety, tolerability and appropriate dosing of this medication for individuals with sickle cell disease.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Oral L-citrulline	There will be a total of 16 patients divided into 4 equal cohorts. The first 4 cohorts will be started on a 7 day oral regimen of twice daily L-citrulline. They will return to clinic on day #7 to evaluate nitric oxide metabolite levels and pharmacokinetic (PK) profile of citrulline. After PK analysis, the next cohort of patients will be given a higher or lower dose based on their weight and also based safety and tolerability. Of note, a PK analysis will be performed for each 4 patient cohort prior to starting citrulline for the subsequent cohort. Assessment for adverse events will be done at specified times throughout the study duration via both phone and clinic encounters.

Timeline

• Start date: 2015-12-01
• Primary completion: 2017-12-31
• Completion: 2017-12-31
• First posted: 2016-01-20
• Last updated: 2018-06-21

Locations

1 site across 1 country: United States