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

This study has been terminated.

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

Sponsor:	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborator:	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	Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene	Phase I

Study Type:	Interventional
Study Design:	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 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

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

Keywords provided by Office of Rare Diseases (ORD):

inborn errors of metabolism
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