Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency

Ornithine Transcarbamylase Deficiency Disease Clinical Trial

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00004498
• Other study ID #: 199/14290
• Secondary ID: UPSM-FDR001529
• Status: Terminated
• Phase: Phase 1
• First received: October 18, 1999
• Last updated: March 24, 2015
• Start date: July 1998
• Est. completion date: September 2000

Study information

• Verified date: February 2000
• Source: FDA Office of Orphan Products Development
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

OBJECTIVES:

I. Determine the safety, feasibility, and potential efficacy of intravascular adenoviral vector mediated gene transfer in the liver in adults with partial ornithine transcarbamylase deficiency.

Description:

PROTOCOL OUTLINE: This is a dose escalation study. Patients undergo a femoral arterial placement of a hepatic intraarterial catheter. Patients then receive adenoviral vector mediated gene transfer intravascularly over 30 minutes.

Cohorts of 3 patients each receive escalating doses of adenoviral vector until the maximum tolerated dose is determined.

Patients are followed at 3, 5, 7, 8, 15, and 29 days, at 2 months, and then every 3 months thereafter.

Completion date provided represents the completion date of the grant per OOPD records

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 21
• Est. completion date: September 2000
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 69 Years

Eligibility

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

• Diagnosis of partial ornithine transcarbamylase deficiency Female heterozygote with abnormal allopurinol challenge or underlying defect in either N15 urea or N15 glutamine OR Male with childhood/adulthood onset OR Family history of 2 affected children

• Stable for at least 1 month prior to study

• Plasma ammonium levels less than 50 micromoles

--Prior/Concurrent Therapy--

• Concurrent alternate pathway therapy to control hyperammonemia allowed

--Patient Characteristics--

• Hepatic: No history of liver disease

• Other: Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception No high level of neutralizing antibodies to the adenovirus

Study Design

Endpoint Classification: Safety Study, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Deficiency Diseases
• Ornithine Transcarbamylase Deficiency Disease

Intervention

Genetic:
• Adenoviral Vector-Mediated Gene Transfer

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
University of Pennsylvania