drugset / Trial / NCT00004386
Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
Phase 1
Terminated
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Children's National Research Institute · collab
Treatment
Summary
OBJECTIVES: Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.
Timeline
- Start
- 1995-10
- Primary completion
- —
- Completion
- —
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | recombinant adenovirus | Unknown | — | Other |