Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
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|ClinicalTrials.gov Identifier: NCT00004386|
Recruitment Status : Terminated
First Posted : October 19, 1999
Last Update Posted : June 24, 2005
Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.
|Condition or disease||Intervention/treatment||Phase|
|Ornithine Transcarbamylase Deficiency Disease||Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene||Phase 1|
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.
|Study Type :||Interventional (Clinical Trial)|
|Study Start Date :||October 1995|
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): NCT00004386
|Study Chair:||Mark Batshaw||Children's Research Institute|