Clinical Trial: Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency
Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional
Official Title:
Brief 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 Summary:
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
Sponsor: University of Pennsylvania
Current Primary Outcome:
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: FDA Office of Orphan Products Development
Dates:
Date Received: October 18, 1999
Date Started: July 1998
Date Completion: September 2000
Last Updated: March 24, 2015
Last Verified: February 2000