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.

Condition	Treatment or Intervention	Phase
Ornithine Transcarbamylase Deficiency Disease	Gene Transfer: Adenoviral Vector-Mediated Gene Transfer	Phase I

Further Study Details: 

Expected Total Enrollment:  21

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.

Ages Eligible for Study:  18 Years   -   69 Years,  Genders Eligible for Study:  Both

Criteria

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 chairs or principal investigators

Steven E. Raper,  Study Chair,  University of Pennsylvania   

Study ID Numbers:  199/14290; UPSM-FDR001529
Record last reviewed:  February 2000
Last Updated:  October 13, 2004
Record first received:  October 18, 1999
ClinicalTrials.gov Identifier:  NCT00004498
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08