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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

Tracking Information
First Submitted Date  ICMJE December 5, 2014
First Posted Date  ICMJE December 11, 2014
Results First Submitted Date  ICMJE December 9, 2015
Results First Posted Date  ICMJE March 7, 2016
Last Update Posted Date September 3, 2019
Actual Study Start Date  ICMJE January 2015
Actual Primary Completion Date December 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 30, 2019)
Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria [ Time Frame: 12 months ]
Original Primary Outcome Measures  ICMJE Not Provided
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease
Official Title  ICMJE Not Provided
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE Drug: Intravenous (IV) citrulline
Study Arms  ICMJE 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.
Intervention: Drug: Intravenous (IV) citrulline
Publications * Barr FE, Tirona RG, Taylor MB, Rice G, Arnold J, Cunningham G, Smith HA, Campbell A, Canter JA, Christian KG, Drinkwater DC, Scholl F, Kavanaugh-McHugh A, Summar ML. Pharmacokinetics and safety of intravenously administered citrulline in children undergoing congenital heart surgery: potential therapy for postoperative pulmonary hypertension. J Thorac Cardiovasc Surg. 2007 Aug;134(2):319-26.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 4, 2016)
4
Original Enrollment  ICMJE Not Provided
Actual Study Completion Date  ICMJE December 2015
Actual Primary Completion Date December 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 25 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02314689
Other Study ID Numbers  ICMJE SCD-124700.
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Suvankar Majumdar, University of Mississippi Medical Center
Study Sponsor  ICMJE Suvankar Majumdar
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account University of Mississippi Medical Center
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP