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Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder

This study has been completed.
Sponsor:
Collaborators:
Rare Diseases Clinical Research Network
Information provided by (Responsible Party):
Children's Research Institute
ClinicalTrials.gov Identifier:
NCT00345605
First received: June 26, 2006
Last updated: November 3, 2014
Last verified: November 2014
Results First Received: May 15, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Double Blind (Subject, Investigator);   Primary Purpose: Treatment
Conditions: Argininosuccinic Aciduria
Amino Acid Metabolism, Inborn Errors
Urea Cycle Disorders
Interventions: Drug: Sodium Phenylbutyrate
Drug: Arginine

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
12 subjects with argininosuccinic aciduria were recruited at Baylor College of Medicine from May 2008 to December 2010.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
patients were randomly assigned to one of the two arms HDA or LDA after treatment and an interval the same patients returned and received the crossover treatment LD or HDA depending.

Reporting Groups
  Description
High Dose First High dose arm 3 day wash-out followed by 7 days of: Arg 500 mg/kg/d or 10 g/m2 BSA Placebo instead of NaPBA interval then: Arg 100 mg/kg/d or 2 g/m2 BSA NaPBA 500 mg/kg/d or 10 g/m2 BSA
Low Dose First low dose arm 3 day wash-out followed by 7 days of: Arg 100 mg/kg/d or 2 g/m2 BSA NaPBA 500 mg/kg/d or 10 g/m2 BSA interval then: Arg 500 mg/kg/d or 10 g/m2 BSA Placebo instead of NaPBA

Participant Flow for 5 periods

Period 1:   Initial Washout
    High Dose First     Low Dose First  
STARTED     6     6  
COMPLETED     6     6  
NOT COMPLETED     0     0  

Period 2:   Arm 1
    High Dose First     Low Dose First  
STARTED     6     6  
COMPLETED     6     5  
NOT COMPLETED     0     1  
Adverse Event                 0                 1  

Period 3:   Interval
    High Dose First     Low Dose First  
STARTED     6     6  
COMPLETED     6     6  
NOT COMPLETED     0     0  

Period 4:   Crossover Wash-out
    High Dose First     Low Dose First  
STARTED     6     6  
COMPLETED     6     6  
NOT COMPLETED     0     0  

Period 5:   Arm 2, Opposite Treatment
    High Dose First     Low Dose First  
STARTED     6     6  
COMPLETED     5     6  
NOT COMPLETED     1     0  
Adverse Event                 1                 0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
High-dose Arginine vs Low-dose Arginine Plus Buphenyl No text entered.

Baseline Measures
    High-dose Arginine vs Low-dose Arginine Plus Buphenyl  
Number of Participants  
[units: participants]
  12  
Age  
[units: participants]
 
<=18 years     8  
Between 18 and 65 years     4  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  15.4  ± 7.2  
Gender  
[units: participants]
 
Female     7  
Male     5  
Region of Enrollment  
[units: participants]
 
United States     12  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Measures of Liver Function: AST and ALT   [ Time Frame: Measured after each 1-week treatment period ]

2.  Primary:   Measures of Liver Function: PT and PTT   [ Time Frame: Measured after each 1-week treatment period ]

3.  Primary:   Measures of Liver Function: Coagulation Factors   [ Time Frame: Measured after each 1-week treatment period ]

4.  Primary:   Measures of Liver Function: INR   [ Time Frame: Measured after each 1-week treatment period ]

5.  Secondary:   Argininosuccinic Acid Levels   [ Time Frame: Measured after each 1-week treatment period ]

6.  Secondary:   Arginine Levels   [ Time Frame: Measured after each 1-week treatment period ]

7.  Secondary:   Urea Production Rate   [ Time Frame: Measured after each 1-week treatment period ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Sandesh Nagamani
Organization: Baylor College of Medicine
e-mail: nagamani@bcm.edu


Publications:

Responsible Party: Children's Research Institute
ClinicalTrials.gov Identifier: NCT00345605     History of Changes
Other Study ID Numbers: RDCRN 5102, U54HD061221
Study First Received: June 26, 2006
Results First Received: May 15, 2014
Last Updated: November 3, 2014
Health Authority: United States: Federal Government