NCT00004386

Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: —
• Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · NIH
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

OBJECTIVES: Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.

Detailed description

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.

Conditions

• Ornithine Transcarbamylase Deficiency Disease

Interventions

Type	Name	Description
GENETIC	recombinant adenovirus containing the ornithine transcarbamylase gene

Timeline

• Start date: 1995-10-01
• First posted: 1999-10-19
• Last updated: 2005-06-24