Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.
|Ornithine Transcarbamylase Deficiency Disease||Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene||Phase 1|
|Study Design:||Primary Purpose: Treatment|
|Study Start Date:||October 1995|
PROTOCOL OUTLINE: This a dose escalation study to estimate the maximum tolerated dose of recombinant adenovirus encoded with the ornithine transcarbamylase gene.
Patients receive a single dose of virus infused into the liver under fluoroscopic guidance. Groups of 3 patients receive successively higher doses of virus; each cohort is observed for safety for 3 weeks before entry of the next group.
Patients are followed weekly for 1 month, then every 3 months until stable.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004386
|Study Chair:||Mark Batshaw||Children's Research Institute|