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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.
 
ClinicalTrials.gov Identifier: NCT00004386
Recruitment Status : Terminated
First Posted : October 19, 1999
Last Update Posted : June 24, 2005
Sponsor:
Collaborator:
Children's National Research Institute
Information provided by:
Office of Rare Diseases (ORD)

Study Description
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Brief Summary:

OBJECTIVES:

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

Resource links provided by the National Library of Medicine

Drug Information available for: Ornithine

Arms and Interventions
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Outcome Measures
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Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
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
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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 ClinicalTrials.gov identifier (NCT number): NCT00004386


Sponsors and Collaborators
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Children's National Research Institute
Investigators
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Study Chair: Mark Batshaw Children's National Research Institute
More Information
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ClinicalTrials.gov Identifier: NCT00004386    
Other Study ID Numbers: 199/12054
CSH-3660
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:
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Ornithine Carbamoyltransferase Deficiency Disease
Deficiency Diseases
Malnutrition
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