Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
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|ClinicalTrials.gov Identifier: NCT00004386|
Recruitment Status : Terminated
First Posted : October 19, 1999
Last Update Posted : June 24, 2005
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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|
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)|
|Study Start Date :||October 1995|
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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|
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
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
|Study Chair:||Mark Batshaw||Children's National Research Institute|
|Other Study ID Numbers:||
|First Posted:||October 19, 1999 Key Record Dates|
|Last Update Posted:||June 24, 2005|
|Last Verified:||January 2000|
inborn errors of metabolism
urea cycle disorder
Ornithine Carbamoyltransferase Deficiency Disease
Urea Cycle Disorders, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors