Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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 Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Sickle Cell Disease
Intervention Drug: Intravenous citrulline
Enrollment 4
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Intravenous Citrulline
Hide Arm/Group Description

Intravenous citrulline at a bolus dose over 5 minutes and then a continuous rate for the next 23 hours.

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.

Period Title: Overall Study
Started 4
Completed 4
Not Completed 0
Arm/Group Title Intravenous Citrulline
Hide Arm/Group Description

Intravenous citrulline at a bolus dose over 5 minutes and then a continuous rate for the next 23 hours.

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.

Overall Number of Baseline Participants 4
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
<=18 years
0
   0.0%
Between 18 and 65 years
4
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 4 participants
19.5
(18 to 23)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
Female
3
  75.0%
Male
1
  25.0%
Race and Ethnicity Not Collected   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants
[1]
Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 4 participants
4
 100.0%
1.Primary Outcome
Title Number of Participants With Adverse Events
Hide Description The number and severity of adverse event will be determined according to the NCI Common Terminology Criteria for Adverse Events (CTCAE)
Time Frame 30 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intravenous Citrulline
Hide Arm/Group Description:

Intravenous citrulline at a bolus dose over 5 minutes and then a continuous rate for the next 23 hours.

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.

Overall Number of Participants Analyzed 4
Measure Type: Count of Participants
Unit of Measure: Participants
4
 100.0%
2.Primary Outcome
Title Plasma Citrulline Level
Hide Description [Not Specified]
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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intravenous Citrulline
Hide Arm/Group Description:

Intravenous citrulline at a bolus dose over 5 minutes and then a continuous rate for the next 23 hours.

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.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: micromol/hr/kg
5  (2.2)
Time Frame Adverse events were collected up to 4 weeks from study drug administration
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Intravenous Citrulline
Hide Arm/Group Description

Intravenous citrulline at a bolus dose over 5 minutes and then a continuous rate for the next 23 hours.

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.

All-Cause Mortality
Intravenous Citrulline
Affected / at Risk (%)
Total   0/4 (0.00%)    
Hide Serious Adverse Events
Intravenous Citrulline
Affected / at Risk (%) # Events
Total   1/4 (25.00%)    
Hepatobiliary disorders   
transaminitis   1/4 (25.00%)  1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Intravenous Citrulline
Affected / at Risk (%) # Events
Total   0/4 (0.00%)    
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Suvankar Majumdar
Organization: Children's National Medical Center
Phone: 2024763800
EMail: smajumdar@childrensnational.org
Layout table for additonal information
Responsible Party: Suvankar Majumdar, University of Mississippi Medical Center
ClinicalTrials.gov Identifier: NCT02697240    
Other Study ID Numbers: 2014-0230
First Submitted: February 29, 2016
First Posted: March 3, 2016
Results First Submitted: June 19, 2018
Results First Posted: April 17, 2019
Last Update Posted: June 25, 2019