Phase 2
N=12
Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
Argininosuccinic Aciduria · Amino Acid Metabolism, Inborn Errors · Urea Cycle Disorders
Bottom Line
View on ClinicalTrials.gov: NCT00345605 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Nov 2014
Primary outcome: Primary: Measures of Liver Function: AST and ALT — 36.2; 52; 31.7; 57.86 IU/L
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Sodium Phenylbutyrate (Drug); Arginine (Drug)
- Age
- Pediatric, Adult, Older Adult · 5+ yrs
- Sex
- All
- Sponsor
- Brendan Lee
- Primary completion
- May 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Measures of Liver Function: AST and ALT |
36.2; 52; 31.7; 57.86 | — |
| PRIMARY Measures of Liver Function: PT and PTT |
14.25; 13.8; 30.91; 30.98 | — |
| PRIMARY Measures of Liver Function: Coagulation Factors |
222.22; 229.77; 105.33; 98.36 | — |
| PRIMARY Measures of Liver Function: INR |
14.2; 13.8 | — |
| SECONDARY Argininosuccinic Acid Levels |
69; 53 | — |
| SECONDARY Arginine Levels |
129; 53 | — |
| SECONDARY Urea Production Rate |
215; 97 | — |
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 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.
Eligibility Criteria
Inclusion Criteria
- 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 Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT00345605). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.