Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency
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|ClinicalTrials.gov Identifier: NCT00004498|
Recruitment Status : Terminated
First Posted : October 19, 1999
Last Update Posted : March 25, 2015
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.
|Condition or disease||Intervention/treatment||Phase|
|Ornithine Transcarbamylase Deficiency Disease||Genetic: Adenoviral Vector-Mediated Gene Transfer||Phase 1|
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
|Study Type :||Interventional (Clinical Trial)|
|Enrollment :||21 participants|
|Study Start Date :||July 1998|
|Study Completion Date :||September 2000|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004498
|Study Chair:||Steven E. Raper||University of Pennsylvania|