Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
NCT00004386 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL
Last updated 2005-06-24
Summary
OBJECTIVES:
Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.
Conditions
- Ornithine Transcarbamylase Deficiency Disease
Interventions
- GENETIC
-
recombinant adenovirus containing the ornithine transcarbamylase gene
Sponsors & Collaborators
-
Children's National Research Institute
collaborator OTHER -
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
lead NIH
Principal Investigators
-
Mark Batshaw · Children's National Research Institute
Study Design
- Purpose
- TREATMENT
Eligibility
- Min Age
- 18 Years
- Max Age
- 65 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 1995-10-31
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