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

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title:

Brief Summary:

OBJECTIVES:

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

Detailed Summary:

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.

Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Current Primary Outcome:

Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Office of Rare Diseases (ORD)

Dates:
Date Received: October 18, 1999
Date Started: October 1995
Date Completion:
Last Updated: June 23, 2005
Last Verified: January 2000