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

This study is ongoing, but not recruiting participants.

First Received: October 18, 1999 Last Updated: June 23, 2005 History of Changes

Sponsors and Collaborators:	FDA Office of Orphan Products Development University of Pennsylvania
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00004498

Purpose

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

Genetics Home Reference related topics: argininosuccinic aciduria citrullinemia N-acetylglutamate synthase deficiency ornithine transcarbamylase deficiency ornithine translocase deficiency succinic semialdehyde dehydrogenase deficiency

Drug Information available for: Ornithine

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Safety Study

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment:	21
Study Start Date:	July 1998

Detailed 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.

Eligibility

Ages Eligible for Study:	18 Years to 69 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004498

Sponsors and Collaborators

FDA Office of Orphan Products Development

University of Pennsylvania

Investigators

Study Chair:

Steven E. Raper

University of Pennsylvania

More Information

No publications provided

Study ID Numbers:	199/14290, UPSM-FDR001529
Study First Received:	October 18, 1999
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004498 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:

genetic diseases and dysmorphic syndromes
inborn errors of metabolism
ornithine transcarbamylase deficiency
rare disease
urea cycle disorder

Study placed in the following topic categories:

Metabolic Diseases
Amino Acid Metabolism, Inborn Errors
Rare Diseases
Central Nervous System Diseases
Brain Diseases
Ornithine Carbamoyltransferase Deficiency Disease
Metabolism, Inborn Errors
Malnutrition
Genetic Diseases, Inborn

Inborn Amino Acid Metabolism Disorder
Urea Cycle Disorders
Nutrition Disorders
Brain Diseases, Metabolic, Inborn
Metabolic Disorder
Ornithine Transcarbamylase Deficiency
Deficiency Diseases
Brain Diseases, Metabolic

Additional relevant MeSH terms:

Metabolic Diseases
Amino Acid Metabolism, Inborn Errors
Nervous System Diseases
Central Nervous System Diseases
Brain Diseases
Ornithine Carbamoyltransferase Deficiency Disease
Metabolism, Inborn Errors

Malnutrition
Genetic Diseases, Inborn
Nutrition Disorders
Brain Diseases, Metabolic, Inborn
Deficiency Diseases
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on May 07, 2009