A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease
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|ClinicalTrials.gov Identifier: NCT02314689|
Recruitment Status : Completed
First Posted : December 11, 2014
Results First Posted : March 7, 2016
Last Update Posted : September 3, 2019
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|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Intravenous (IV) citrulline||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Actual Study Start Date :||January 2015|
|Actual Primary Completion Date :||December 2015|
|Actual Study Completion Date :||December 2015|
Experimental: IV citrulline
IV citrulline 20 mg/kg bolus with dose escalation of 10 mg/kg to target citrulline concentration of 100 µmol/L with a maximum dose of 60 mg/kg.
Drug: Intravenous (IV) citrulline
- Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria [ Time Frame: 12 months ]
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|Ages Eligible for Study:||18 Years to 25 Years (Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Sickle cell disease genotypes (HbSS, HbS/β° thalassemia, HbS/β+thalassemia, HbSC)
- Presence of any acute illness defined by fever >100.4° F within the past 48 hours
- Presence of sickle cell pain crisis defined by the presence of pain requiring oral or parental opioid therapy.
- Presence of acute chest syndrome or presence of any other complication related to sickle cell disease requiring hospitalization such as splenic sequestration, hepatic sequestration, stroke, avascular necrosis of the hip/shoulder, acute priapism, and patients with diabetes etc.
- Severe anemia (hemoglobin < 5g/dL)
- History of red blood cell transfusion within the last 14 days
- Systemic steroid therapy within the last 48 hours
- Pregnant (as confirmed by a negative urine pregnancy test) or lactating female
- Alanine/aspartate transferase >2x upper limit of normal laboratory range for age.
- Elevated serum creatinine >1.5mg/dL
- Patients with an inability to give consent will be excluded
- Medications that are known to be contra-indicated with use of L-citrulline (concurrent use of hydroxyurea will be allowed).
- History of diabetes due to risk of electrolyte imbalance
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): NCT02314689
|United States, Mississippi|
|University of Mississippi Medical Center|
|Jackson, Mississippi, United States, 39216|
|Responsible Party:||Suvankar Majumdar, Associate Professor, University of Mississippi Medical Center|
|Other Study ID Numbers:||
|First Posted:||December 11, 2014 Key Record Dates|
|Results First Posted:||March 7, 2016|
|Last Update Posted:||September 3, 2019|
|Last Verified:||July 2019|
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn