Ornithine Transcarbamylase Deficiency Disease Clinical Trial
OBJECTIVES:
Evaluate the safety and feasibility of administering recombinant adenovirus containing the
ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase
deficiency.
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.
;
Primary Purpose: Treatment
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Terminated |
NCT00004498 -
Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency
|
Phase 1 | |
| Recruiting |
NCT06255782 -
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency (OTC-HOPE)
|
Phase 1/Phase 2 |