A Study of Oral L-citrulline in Sickle Cell Disease
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|ClinicalTrials.gov Identifier: NCT02659644|
Recruitment Status : Withdrawn (Principal Investigator moved to another institution)
First Posted : January 20, 2016
Last Update Posted : June 21, 2018
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.
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Oral L-citrulline||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I Dose-Ranging Study of Oral L-citrulline in Steady-State Sickle Cell|
|Study Start Date :||December 2015|
|Estimated Primary Completion Date :||December 31, 2017|
|Actual Study Completion Date :||December 31, 2017|
|Experimental: Oral citrulline||
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.
- Peak plasma citrulline concentration (Cmax) [ Time Frame: 7 days ]
- Rate of citrulline appearance (Rapp) [ Time Frame: 7 days ]
- Constant of citrulline removal (krem) [ Time Frame: 7 days ]
- Volume of distribution [ Time Frame: 7 days ]
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 7 days ]
- Level of nitric oxide [ Time Frame: 7 days ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02659644
|United States, Mississippi|
|University of Mississippi Medical Center|
|Jackson, Mississippi, United States, 39216|