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

The recruitment status of this study is unknown because the information has not been verified recently.

Verified February 2000 by FDA Office of Orphan Products Development. Recruitment status was Active, not recruiting

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

Sponsor:	FDA Office of Orphan Products Development
Collaborator:	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	Genetic: Adenoviral Vector-Mediated Gene Transfer	Phase I

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety Study Primary Purpose: Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: argininosuccinic aciduria ataxia neuropathy spectrum carbamoyl phosphate synthetase I deficiency childhood myocerebrohepatopathy spectrum citrullinemia deoxyguanosine kinase deficiency mitochondrial neurogastrointestinal encephalopathy disease myoclonic epilepsy myopathy sensory ataxia N-acetylglutamate synthase deficiency ornithine transcarbamylase deficiency ornithine translocase deficiency succinic semialdehyde dehydrogenase deficiency Help Me Understand Genetics

Drug Information available for: Ornithine

U.S. FDA Resources

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

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

Additional relevant MeSH terms:

Deficiency Diseases
Carbamoyl-Phosphate Synthase I Deficiency Disease
Ornithine Carbamoyltransferase Deficiency Disease
Malnutrition
Nutrition Disorders
Urea Cycle Disorders, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases

Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Mitochondrial Diseases
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on February 12, 2012