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

Ornithine Transcarbamylase Deficiency Disease Clinical Trial

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00004386
• Other study ID #: 199/12054
• Secondary ID: CSH-3660
• Status: Terminated
• Phase: Phase 1
• First received: October 18, 1999
• Last updated: June 23, 2005
• Start date: October 1995

Study information

• Verified date: January 2000
• Source: Office of Rare Diseases (ORD)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

OBJECTIVES:

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

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 0
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

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

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Deficiency Diseases
• Ornithine Transcarbamylase Deficiency Disease

Intervention

Genetic:
• recombinant adenovirus containing the ornithine transcarbamylase gene

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Children's Research Institute