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

University of Pennsylvania0 sites21 target enrollmentJuly 1998

ConditionsOrnithine Transcarbamylase Deficiency Disease

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Ornithine Transcarbamylase Deficiency Disease
Sponsor: University of Pennsylvania
Enrollment: 21
Status: Terminated
Last Updated: 11 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

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 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

Registry

clinicaltrials.gov

Start Date

July 1998

End Date

September 2000

Last Updated

11 years ago

Study Type

Interventional

Sex

All