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

This study has been terminated.

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

Sponsors and Collaborators:	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Children's Research Institute
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00004386

Purpose

OBJECTIVES:

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

Condition	Intervention	Phase
Ornithine Transcarbamylase Deficiency Disease	Gene Transfer: recombinant adenovirus containing the ornithine transcarbamylase gene	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

Further study details as provided by Office of Rare Diseases (ORD):

Study Start Date:

October 1995

Detailed 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.

Eligibility

Ages Eligible for Study:	18 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004386

Sponsors and Collaborators

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Children's Research Institute

Investigators

Study Chair:

Mark Batshaw

Children's Research Institute

More Information

No publications provided

Study ID Numbers:	199/12054, CSH-3660
Study First Received:	October 18, 1999
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004386 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):

inborn errors of metabolism
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