Status:
COMPLETED
Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
Lead Sponsor:
Brendan Lee
Collaborating Sponsors:
Office of Rare Diseases (ORD)
Rare Diseases Clinical Research Network
Conditions:
Argininosuccinic Aciduria
Amino Acid Metabolism, Inborn Errors
Eligibility:
All Genders
5+ years
Phase:
PHASE2
Brief Summary
Urea cycle disorders are inherited illnesses in which the body does not produce enough of the chemicals that remove ammonia, a byproduct of protein metabolism, from the blood stream. Elevated ammonia ...
Detailed Description
The cause of liver damage in people with ASA is unknown. However, because ASA is the only urea cycle disorder that is characterized by both liver damage and elevated levels of argininosuccinic acid, r...
Eligibility Criteria
Inclusion
- Has confirmed diagnosis of ASA by amino acid or enzyme assay
- Has a history of adequate compliance to the diet and treatment
- Able to take oral or G-tube medication
- Able to perform 24 hour urine collection
- Agrees to travel to Baylor College of Medicine
- If female, of child bearing potential, and sexually active, agrees to use an acceptable method of birth control
- Greater than 5 years of age
Exclusion
- Has a history of congestive heart failure, severe renal insufficiency, or any condition that causes sodium retention or edema
- Currently taking Probenecid, Haloperidol, Valproate or oral corticosteroids
- Pregnant or lactating
- Currently being treated for an acute illness
- Has co-morbid associations causing difficulties in the detection of hyperammonemic episodes, liver damage, or difficulties in the diet compliance
- Has known hypersensitivity to sodium phenylbutyrate
- Has taken any experimental medication within the last 30 days
- Has renal insufficiency with creatinine greater than 1.5 mg/dl at screening
Key Trial Info
Start Date :
February 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 1 2012
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT00345605
Start Date
February 1 2008
End Date
November 1 2012
Last Update
January 31 2018
Active Locations (1)
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1
Baylor College of Medicine
Houston, Texas, United States, 77030