Ignite Creation Date:
2024-05-05 @ 4:54 PM
Last Modification Date:
2024-10-26 @ 9:25 AM
Study NCT ID:
NCT00345605
Status:
COMPLETED
Last Update Posted:
2018-01-31
First Post:
2006-06-26
Brief Title:
Arginine and Buphenyl in Patients With Argininosuccinic Aciduria ASA a Urea Cycle Disorder
Sponsor:
Brendan Lee
Organization:
Baylor College of Medicine
Study Overview
Official Title:
A Randomized Double-Blind Crossover Study of Sodium Phenylbutyrate and Low-Dose Arginine Compared to High-Dose Arginine Alone on Liver Function Ureagenesis and Subsequent Nitric Oxide Production in Patients With Argininosuccinic Aciduria
Status:
COMPLETED
Status Verified Date:
2015-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 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
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 researchers believe that the elevated acid levels cause the liver damage Common treatments for urea cycle disorders include a low-protein diet and arginine supplementation which when combined help to decrease ammonia levels in the blood Buphenyl-TM may aid in lowering ammonia and argininosuccinic acid levels Although Buphenyl-TM has been FDA-approved for use in people with some types of urea cycle disorders there is little information on the effectiveness of the drug in children with ASA This study will evaluate whether treatment of ASA patients with Buphenyl-TM in conjunction with lowered doses of arginine improves liver function as measured by short-term assessment of synthetic activity and the use of stable isotope tracers to assess ureagenesis and nitric oxide production
Initially participants in this double-blind placebo-controlled crossover study will undergo a 3-day washout period during which no Buphenyl-TM will be given They will then be randomly assigned to one of two groups either Buphenyl-TM 500 mgkgday or 10 gramsm2 and arginine 100 mgkgday or 2 gramsm2 or arginine alone 500 mgkgday or 10 gramsm2 Participants will remain on this initial treatment arm for 1 week at the conclusion of which an assessment of hepatic synthetic function ureagenesis and nitric oxide production will be performed After this assessment participants will undergo a second 3-day washout and then crossover to the other treatment arm for 1 week At the end of the 1-week treatment period a second assessment will be performed During the washout period before each treatment period no Buphenyl-TM will be administered and arginine will be administered at the standard therapeutic dose of 500 mgkgday or 10 grams2
Initially participants in this double-blind placebo-controlled crossover study will undergo a 3-day washout period during which no Buphenyl-TM will be given They will then be randomly assigned to one of two groups either Buphenyl-TM 500 mgkgday or 10 gramsm2 and arginine 100 mgkgday or 2 gramsm2 or arginine alone 500 mgkgday or 10 gramsm2 Participants will remain on this initial treatment arm for 1 week at the conclusion of which an assessment of hepatic synthetic function ureagenesis and nitric oxide production will be performed After this assessment participants will undergo a second 3-day washout and then crossover to the other treatment arm for 1 week At the end of the 1-week treatment period a second assessment will be performed During the washout period before each treatment period no Buphenyl-TM will be administered and arginine will be administered at the standard therapeutic dose of 500 mgkgday or 10 grams2
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| U54HD061221 | NIH | None | httpsreporternihgovquickSearchU54HD061221 |