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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 is not designed to be a substitute for certified medical advice, travels to or professional assistance by using a genuine doctor. We aren't mDs. Always consult your physician about 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 committed 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 health trials occur in a lot of of cities throughout the US. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally evaluate the potency of new does drugs. The role of the studies / undertakings is to figure out specific human healthcare questions. Clinical trials are a popular manner for mDs, government agencies, and private sector companies to locate treatments for all sorts 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 permit volunteers to get medical treatment choices before they are available to the general public. Many times the test subjects get professional assistance for free of charge, and occasionally they are compensated for their time. Sometimes 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. Human subjects often get the best 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, nevertheless, and could include additional or frequent dr. calls, medical hazards (perhaps life-threatening), and/or the treatment being ineffectual. Trials are federally governed with exacting guidelines to protect clinical trials patients.

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

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

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