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

This study has been terminated.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00004386
First Posted: October 19, 1999
Last Update Posted: December 9, 2005
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.
Collaborator:
Children's Research Institute
Information provided by:
Office of Rare Diseases (ORD)
October 18, 1999
October 19, 1999
December 9, 2005
October 1995
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No Changes Posted
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Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
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OBJECTIVES:

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

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.

Interventional
Phase 1
Primary Purpose: Treatment
Ornithine Transcarbamylase Deficiency Disease
Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
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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

Sexes Eligible for Study: All
18 Years to 65 Years   (Adult)
No
Contact information is only displayed when the study is recruiting subjects
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NCT00004386
199/12054
CSH-3660
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Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Children's Research Institute
Study Chair: Mark Batshaw Children's Research Institute
Office of Rare Diseases (ORD)
January 2000

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP