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A Study of Oral L-citrulline in Sickle Cell Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02659644
Recruitment Status : Withdrawn (Principal Investigator moved to another institution)
First Posted : January 20, 2016
Last Update Posted : June 21, 2018
Sponsor:
Information provided by (Responsible Party):
Suvankar Majumdar, University of Mississippi Medical Center

Brief 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.


Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Oral L-citrulline Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
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

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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.




Primary Outcome Measures :
  1. Peak plasma citrulline concentration (Cmax) [ Time Frame: 7 days ]
  2. Rate of citrulline appearance (Rapp) [ Time Frame: 7 days ]
  3. Constant of citrulline removal (krem) [ Time Frame: 7 days ]
  4. Volume of distribution [ Time Frame: 7 days ]
  5. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 7 days ]

Secondary Outcome Measures :
  1. Level of nitric oxide [ Time Frame: 7 days ]


Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Sickle cell disease genotypes (HbSS, HbS/β-thalassemia
  2. HbS/β+thalassemia, HbSC)
  3. Patients with sickle cell disease aged 10 to 25 years old
  4. Patients ages 10 through 17 years of age, whose parents have signed permission, and who provide signed patient assent themselves
  5. Patients 18 through 25 years of age who provide signed consent.

Exclusion Criteria:

  1. Presence of any acute illness defined by fever >100.4°F within the past 48 hours
  2. Presence of sickle cell pain crisis defined by the presence of pain requiring oral or parental opioid therapy.
  3. 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.
  4. Severe anemia (hemoglobin < 5g/dL)
  5. History of red blood cell transfusion within the last 14 days
  6. Systemic steroid therapy within the last 48 hours
  7. Pregnant (as confirmed by a negative urine pregnancy test) or lactating female
  8. Alanine/aspartate transferase >2x upper limit of normal laboratory range for age.
  9. Elevated serum creatinine: Age 6 to 13 years > 0.9 mg/dL, Age 14-17 years 1.0 mg/dL, Age >18 years >1.5mg/dL
  10. Patients with an inability to give assent (ages 10 to 17 years) or consent (ages 18 through 25 years) will be excluded
  11. History of diabetes due to risk of electrolyte imbalance

Information from the National Library of Medicine

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


Locations
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United States, Mississippi
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216
Sponsors and Collaborators
University of Mississippi Medical Center
Publications:
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Responsible Party: Suvankar Majumdar, Associate Professor, University of Mississippi Medical Center
ClinicalTrials.gov Identifier: NCT02659644    
Other Study ID Numbers: 2015-0191
First Posted: January 20, 2016    Key Record Dates
Last Update Posted: June 21, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn