Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder (NCT00345605) | Clinical Trial Compass
CompletedPhase 2
Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
United States12 participantsStarted 2008-02
Plain-language 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.
Who can participate
Age range
5 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Measures of Liver Function: AST and ALT
Timeframe: Measured after each 1-week treatment period
2
Measures of Liver Function: PT and PTT
Timeframe: Measured after each 1-week treatment period
3
Measures of Liver Function: Coagulation Factors
Timeframe: Measured after each 1-week treatment period
4
Measures of Liver Function: INR
Timeframe: Measured after each 1-week treatment period