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

Completed · Phase 2

Conditions studied: Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders

In brief

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 levels can lead to brain damage and death. Argininosuccinic aciduria (ASA) is a type of urea cycle disorder that is characterized specifically by high levels of argininosuccinic acid, a chemical involved in the urea cycle. People with ASA are at risk for serious liver damage, which may be due to the elevated levels of argininosuccinic acid. Sodium phenylbutyrate (Buphenyl-TM) is a drug that has been used to treat other types of urea cycle disorders. This study will evaluate whether Buphenyl-TM in conjunction with decreased arginine dose (in addition to a normal regimen of protein) will improve short-term liver function and decrease plasma citrulline and ASA levels in people with ASA.

Key facts

Study ID

NCT00345605

Run by

Brendan Lee

People needed

12

Starts

2008-02-01

Expected to finish

2012-11-01

Last updated by the study team

2018-01-31

Who can join

Age: 5 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Baylor College of Medicine — Houston, Texas, United States

Full record on ClinicalTrials.gov

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