NCT00004498

Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 21 (planned)
• Sponsor: University of Pennsylvania · Academic / Other
• Sex: All
• Age: 18 Years – 69 Years
• Healthy volunteers: Not accepted

Summary

OBJECTIVES: I. Determine the safety, feasibility, and potential efficacy of intravascular adenoviral vector mediated gene transfer in the liver in adults with partial ornithine transcarbamylase deficiency.

Detailed description

PROTOCOL OUTLINE: This is a dose escalation study. Patients undergo a femoral arterial placement of a hepatic intraarterial catheter. Patients then receive adenoviral vector mediated gene transfer intravascularly over 30 minutes. Cohorts of 3 patients each receive escalating doses of adenoviral vector until the maximum tolerated dose is determined. Patients are followed at 3, 5, 7, 8, 15, and 29 days, at 2 months, and then every 3 months thereafter. Completion date provided represents the completion date of the grant per OOPD records

Conditions

• Ornithine Transcarbamylase Deficiency Disease

Interventions

Type	Name	Description
GENETIC	Adenoviral Vector-Mediated Gene Transfer

Timeline

• Start date: 1998-07-01
• Completion: 2000-09-01
• First posted: 1999-10-19
• Last updated: 2015-03-25