Ignite Creation Date:
2024-05-05 @ 11:21 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004386
Status:
TERMINATED
Last Update Posted:
2005-06-24
First Post:
1999-10-18
Brief Title:
Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
Sponsor:
Eunice Kennedy Shriver National Institute of Child Health and Human Development NICHD
Organization:
Office of Rare Diseases ORD
Study Overview
Official Title:
None
Status:
TERMINATED
Status Verified Date:
2000-01
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
Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency
Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency
Detailed Description:
PROTOCOL OUTLINE This a dose escalation study to estimate the maximum tolerated dose of recombinant adenovirus encoded with the ornithine transcarbamylase gene
Patients receive a single dose of virus infused into the liver under fluoroscopic guidance Groups of 3 patients receive successively higher doses of virus each cohort is observed for safety for 3 weeks before entry of the next group
Patients are followed weekly for 1 month then every 3 months until stable
Patients receive a single dose of virus infused into the liver under fluoroscopic guidance Groups of 3 patients receive successively higher doses of virus each cohort is observed for safety for 3 weeks before entry of the next group
Patients are followed weekly for 1 month then every 3 months until stable
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 |
|---|---|---|---|
| CSH-3660 | None | None | None |