Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency
Recruitment status was Active, not recruiting
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.
Ornithine Transcarbamylase Deficiency Disease
Genetic: Adenoviral Vector-Mediated Gene Transfer
|Study Design:||Endpoint Classification: Safety Study
Primary Purpose: Treatment
|Study Start Date:||July 1998|
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.
|Study Chair:||Steven E. Raper||University of Pennsylvania|