Trial | NCT00004386  Report issue

Title

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

  • Phase

    Phase 1

  • Study Type

    Interventional

  • Status

    Terminated

  • Study Participants

    None
OBJECTIVES:

Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.
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 Started
Oct 31
1995
Last Update
Jun 24
2005
Estimate

Genetic recombinant adenovirus containing the ornithine transcarbamylase gene

Criteria 

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Confirmed partial ornithine transcarbamylase deficiency Asymptomatic, i.e., at least 1 month since hyperammonemia --Prior/Concurrent Therapy-- Not specified --Patient Characteristics-- No pregnant or nursing women Negative pregnancy test required of fertile women
No Results Posted