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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00004386
Recruitment Status : Terminated
First Posted : October 19, 1999
Last Update Posted : June 24, 2005
Children's Research Institute
Information provided by:
Office of Rare Diseases (ORD)

Brief Summary:


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

Condition or disease Intervention/treatment Phase
Ornithine Transcarbamylase Deficiency Disease Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene Phase 1

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.

Study Type : Interventional  (Clinical Trial)
Primary Purpose: Treatment
Study Start Date : October 1995

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Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00004386

Sponsors and Collaborators
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Children's Research Institute
Study Chair: Mark Batshaw Children's Research Institute Identifier: NCT00004386     History of Changes
Other Study ID Numbers: 199/12054
First Posted: October 19, 1999    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: January 2000

Keywords provided by Office of Rare Diseases (ORD):
inborn errors of metabolism
rare disease
urea cycle disorder

Additional relevant MeSH terms:
Deficiency Diseases
Ornithine Carbamoyltransferase Deficiency Disease
Nutrition Disorders
Urea Cycle Disorders, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases