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A Dose Escalation Study of Intravenous L-citrulline in Steady-state 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: NCT02314689
Recruitment Status : Completed
First Posted : December 11, 2014
Results First Posted : March 7, 2016
Last Update Posted : September 3, 2019
Sponsor:
Information provided by (Responsible Party):
Suvankar Majumdar, University of Mississippi Medical Center

Study Description
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Brief Summary:
The purpose of this study is to assess the maximum tolerated dose, safety and pharmacokinetics of an investigational drug, intravenous (IV) citrulline, in subjects in steady-state sickle cell disease.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Intravenous (IV) citrulline Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Actual Study Start Date : January 2015
Actual Primary Completion Date : December 2015
Actual Study Completion Date : December 2015

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria [ Time Frame: 12 months ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 25 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Sickle cell disease genotypes (HbSS, HbS/β° thalassemia, HbS/β+thalassemia, HbSC)

Exclusion Criteria:

  • 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
Contacts and Locations
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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): NCT02314689


Locations
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United States, Mississippi
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216
Sponsors and Collaborators
Suvankar Majumdar
More Information
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Publications:

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Responsible Party: Suvankar Majumdar, Associate Professor, University of Mississippi Medical Center
ClinicalTrials.gov Identifier: NCT02314689    
Other Study ID Numbers: SCD-124700.
First Posted: December 11, 2014    Key Record Dates
Results First Posted: March 7, 2016
Last Update Posted: September 3, 2019
Last Verified: July 2019
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn