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A Phase 1 Study of Continuous Intravenous L-citrulline During Sickle Cell Pain Crisis or Acute Chest Syndrome

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: NCT02697240
Recruitment Status : Completed
First Posted : March 3, 2016
Results First Posted : April 17, 2019
Last Update Posted : June 25, 2019
Sponsor:
Information provided by (Responsible Party):
Suvankar Majumdar, University of Mississippi Medical Center

Study Description
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Brief Summary:
Sickle cell disease is a genetic red blood cell disorder characterized by vaso-occlusion from sickling of red blood cells, that can lead to pain or organ complications such as acute chest syndrome. Sickle cell disease is associated with low amounts of nitric oxide, a compound important for dilating the blood vessel wall. Citrulline is a substance that is known to increase nitric oxide. The goal of this Phase I study are to find the highest safe dose of continuous IV citrulline that can be given to individuals with sickle cell disease experiencing a sickle cell pain crisis or acute chest syndrome without causing severe side effects.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Intravenous 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
Official Title: A Phase 1 Dose Escalation Study of Continuous Intravenous L-citrulline During Sickle Cell Pain Crisis or Acute Chest Syndrome
Study Start Date : February 2016
Actual Primary Completion Date : April 2017
Actual Study Completion Date : April 2017

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Intravenous citrulline
Intravenous citrulline at a bolus dose over 5 minutes and then a continuous rate for the next 23 hours.
 Drug: Intravenous citrulline
Intravenous citrulline at a bolus dose of 20 mg/kg over 5 minutes and then continuous at a rate of 7 mg/kg/hour for the next 23 hours for the first 7 participants, and depending on safety and pharmacokinetic profile, increase or decrease the continuous rate to 9 or 5 mg/kg/hour for the next 7 participants.


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Adverse Events [ Time Frame: 30 days ]
    The number and severity of adverse event will be determined according to the NCI Common Terminology Criteria for Adverse Events (CTCAE)

  2. Plasma Citrulline Level [ Time Frame: Plasma citrulline levels will be evaluated at the following time points: trough level, 10 minutes (peak level), 30 minutes, 1 hour, 2 hours, 3 hours, 4 hours, 6 hours, 8 hours, 12 hours, 24 hours and 48 hours, to evaluate the pharmacokinetic profile ]

Eligibility Criteria
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Ages Eligible for Study:   6 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Sickle cell disease genotypes (HbSS, HbS/β0-thalassemia, HbS/β+-thalassemia, HbSC)
  2. Ages 6 to 50 years old
  3. Patients with sickle cell disease aged 6 to 50 years old Presence of sickle cell pain crisis defined by the presence of pain requiring hospitalization and parental opioid therapy
  4. Presence of acute chest syndrome defined by the presence of a new CXR infiltrate and any one of the following respiratory symptoms of fever, shortness of breath, wheezing, chest pain, cough or new onset hypoxia.

Exclusion Criteria:

  1. 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, etc.
  2. Severe anemia (hemoglobin < 5g/dL)
  3. History of red blood cell transfusion within the last 30 days
  4. Systemic steroid therapy within the last 48 hours
  5. Pregnant (as confirmed by a negative urine pregnancy test) or lactating female
  6. Alanine/aspartate transferase >2x upper limit of normal laboratory range for age.

  7. Subject has the following 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
  8. Patients with an inability to give consent will be excluded
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): NCT02697240


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
  Study Documents (Full-Text)

Documents provided by Suvankar Majumdar, University of Mississippi Medical Center:
Study Protocol and Statistical Analysis Plan  [PDF] January 10, 2019

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: NCT02697240    
Other Study ID Numbers: 2014-0230
First Posted: March 3, 2016    Key Record Dates
Results First Posted: April 17, 2019
Last Update Posted: June 25, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Suvankar Majumdar, University of Mississippi Medical Center:
sickle cell disease
citrulline
Additional relevant MeSH terms:
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Acute Chest Syndrome
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
 Hemoglobinopathies
Genetic Diseases, Inborn
Lung Diseases
Respiratory Tract Diseases
Respiration Disorders