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Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency Clinical Trials Facts presented on Clinical Trials Search isn't designed to be a substitute for proven healthcare advice, calls or treatment by using a genuine medical doctor. We aren't mDs. Always confer with your doctor on Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency conditions. Clinical Trials Search.org is a website devoted to listing clinical research studies in human subjects. Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency Clinical research trials and Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency healthcare trials occur in a lot of of places across the United States. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally assess the effectivity of new does drugs. The role of the studies / undertakings is to solve specific human healthcare questions. Clinical trials are a popular way for doctors, government agencies, and private sector companies to find treatments for all kinds of conditions, including Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency. Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency Clinical Trials and other clinical trials allow for volunteers to access health treatment choices before they are available to the general public. Many times the test subjects get treatment for without cost, and sometimes they are compensated for their time. Occasionally there is a cost for a Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency clinical trial. Test subjects typically receive the most effective healthcare possible for their Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency condition. Risks are a reality, nonetheless, and could include extra or frequent dr. calls, health hazards (perhaps life-jeopardizing), and/or the treatment being ineffective. Trials are federally regulated with rigid guidelines to protect clinical trials subjects.

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Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency



Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency

For Condition: Ornithine Transcarbamylase Deficiency Disease
Status: No longer recruiting
Sponsor(s): FDA Office of Orphan Products Development , University of Pennsylvania
Synopsis: OBJECTIVES: I. Determine the safety, feasibility, and potential efficacy of intravascular adenoviral vector mediated gene transfer in the liver in adults with partial ornithine transcarbamylase deficiency.
Details: PROTOCOL OUTLINE: This is a dose escalation study. Patients undergo a femoral arterial placement of a hepatic intraarterial catheter. Patients then receive adenoviral vector mediated gene transfer intravascularly over 30 minutes. Cohorts of 3 patients each receive escalating doses of adenoviral vector until the maximum tolerated dose is determined. Patients are followed at 3, 5, 7, 8, 15, and 29 days, at 2 months, and then every 3 months thereafter.
Eligibility:
Study Type:
  Interventional, Treatment, Safety Study
Minimum Age/Maximum Age: 18 Years/69 Years
Genders: Both
Protocol Entry Criteria: PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- - Diagnosis of partial ornithine transcarbamylase deficiency Female heterozygote with abnormal allopurinol challenge or underlying defect in either N15 urea or N15 glutamine OR Male with childhood/adulthood onset OR Family history of 2 affected children - Stable for at least 1 month prior to study - Plasma ammonium levels less than 50 micromoles --Prior/Concurrent Therapy-- - Concurrent alternate pathway therapy to control hyperammonemia allowed --Patient Characteristics-- - Hepatic: No history of liver disease - Other: Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception No high level of neutralizing antibodies to the adenovirus
Total Enrollment: 21

Location and Contact Information:

Overall Study Official:
StevenRaper,  Study Chair,  University of Pennsylvania


Additional Information:
Study ID Numbers:
  199/14290;  UPSM-FDR001529
Study Start Date: July 1998
Record last reviewed: February 2000
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00004498

Other Ornithine Transcarbamylase Deficiency Disease Studies:
1. Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency

2. Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency

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