Ignite Creation Date:
2024-05-05 @ 11:07 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004767
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
2000-02-24
Brief Title:
Phase II Study of Sodium Phenylbutyrate Sodium Benzoate Sodium Phenylacetate and Dietary Intervention for Urea Cycle Disorders
Sponsor:
National Center for Research Resources NCRR
Organization:
Office of Rare Diseases ORD
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2001-12
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:
OBJECTIVES
I Assess the safety and efficacy of sodium phenylbutyrate sodium benzoate sodium phenylacetate and dietary intervention in patients with urea cycle disorders
I Assess the safety and efficacy of sodium phenylbutyrate sodium benzoate sodium phenylacetate and dietary intervention in patients with urea cycle disorders
Detailed Description:
PROTOCOL OUTLINE This protocol describes several clinical studies of pharmacologic and dietary management in patients with urea cycle disorders
Patients with carbamyl phosphate synthetase and ornithine transcarbamylase deficiency are treated with a low-protein diet essential amino acids for neonatal onset disease caloric supplementation oral sodium phenylbutyrate now approved as a prescription drug 1197 and citrulline or arginine free base
Patients with argininosuccinic acid synthetase deficiency are treated with a low-protein diet caloric supplementation oral sodium phenylbutyrate now approved as a prescription drug 1197 and arginine free base
Patients with argininosuccinic aciduria AA are treated with a low-protein diet caloric supplementation and arginine free base Discontinued 1197 Any patient who develops hyperammonemia is treated with intravenous sodium benzoate sodium phenylbutyrate and arginine hydrochloride benzoate and phenylbutyrate are not given to patients with AA
If ammonium stabilizes at normal or near normal levels intravenous medications are gradually replaced by oral medications If there is no significant decrease in ammonium within 8 hours patients begin hemodialysis
Concurrent therapy with ondansetron high caloric intake and mannitol for elevated intracranial pressure is allowed Dietary and intravenous nitrogen is prohibited Discontinued 1197
Patients with carbamyl phosphate synthetase and ornithine transcarbamylase deficiency are treated with a low-protein diet essential amino acids for neonatal onset disease caloric supplementation oral sodium phenylbutyrate now approved as a prescription drug 1197 and citrulline or arginine free base
Patients with argininosuccinic acid synthetase deficiency are treated with a low-protein diet caloric supplementation oral sodium phenylbutyrate now approved as a prescription drug 1197 and arginine free base
Patients with argininosuccinic aciduria AA are treated with a low-protein diet caloric supplementation and arginine free base Discontinued 1197 Any patient who develops hyperammonemia is treated with intravenous sodium benzoate sodium phenylbutyrate and arginine hydrochloride benzoate and phenylbutyrate are not given to patients with AA
If ammonium stabilizes at normal or near normal levels intravenous medications are gradually replaced by oral medications If there is no significant decrease in ammonium within 8 hours patients begin hemodialysis
Concurrent therapy with ondansetron high caloric intake and mannitol for elevated intracranial pressure is allowed Dietary and intravenous nitrogen is prohibited Discontinued 1197
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| JHUSM-11753 | None | None | None |