Ignite Creation Date:
2024-05-05 @ 10:00 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004307
Status:
UNKNOWN
Last Update Posted:
2005-06-24
First Post:
1999-10-18
Brief Title:
Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
Sponsor:
National Center for Research Resources NCRR
Organization:
National Center for Research Resources NCRR
Study Overview
Official Title:
Therapeutic and Metabolic Studies of Urea Cycle Disorders Part A Nitrogen Flux and Ureagenesis Part B Closed Phase I Adenovirus Vector-Mediated Gene Therapy for Ornithine Transcarbamylase Deficiency
Status:
UNKNOWN
Status Verified Date:
2003-12
Last Known Status:
RECRUITING
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:
RATIONALE The urea cycle is the process in which nitrogen is removed from the blood and converted into urea a waste product found in urine Urea cycle disorders are inherited disorders caused by the lack of an enzyme that removes ammonia from the bloodstream Gene therapy is treatment given to change a gene so that it functions normally Studying the treatment and metabolism of patients with urea cycle disorders may be helpful in developing new treatments for these disorders
PURPOSE Two-part clinical trial to study the treatment and metabolism of patients who have urea cycle disorders
PURPOSE Two-part clinical trial to study the treatment and metabolism of patients who have urea cycle disorders
Detailed Description:
PROTOCOL OUTLINE This protocol involves 2 clinical studies Part A is a metabolic study of glutamine conversion to urea at different levels of protein intake while on and off medications Part B is a dose escalation study of a first-generation adenoviral vector with an E1 deletion and an E3 deletion substitution d1309 expressing ornithine transcarbamylase OTC
In Part A diet is controlled for protein and calories Intravenous glutamine and urea are administered Controls are given intravenous arginine phenylacetate and benzoate
In Part B groups of 3 patients are given a single low intermediate or high dose of intravenous OTC vector Allopurinol is administered every 12 hours for 12 days As of 12101999 Part B of the study is closed
In Part A diet is controlled for protein and calories Intravenous glutamine and urea are administered Controls are given intravenous arginine phenylacetate and benzoate
In Part B groups of 3 patients are given a single low intermediate or high dose of intravenous OTC vector Allopurinol is administered every 12 hours for 12 days As of 12101999 Part B of the study is closed
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 |
|---|---|---|---|
| BCM-H4379 | None | None | None |