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Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency Clinical Trials Information presented on Clinical Trials Search is not intended to be a substitute for qualified health advice, trips or treatment by using a genuine doctor. We aren't doctors. Always consult your mD on Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency conditions. Clinical Trials Search.org is a site committed to listing clinical research studies in human subjects. Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency Clinical research trials and Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency health trials take place in a lot of of cities across the US. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally measure the potency of new drugs. The aim of the studies / projects is to answer specific human medical questions. Clinical trials are a popular manner for physicians, government agencies, and private sector corporations to discover remedies for all kinds of circumstances, like Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency. Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency Clinical Trials and other clinical trials allow for volunteers to have health treatment alternatives before they are available to the general public. Many times the test subjects obtain treatment for without cost, and occasionally they are paid for their time. Sometimes there is a cost for a Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency clinical trial. Subjects oftentimes recieve the most effective healthcare possible for their Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency condition. Hazards are a reality, however, and could include additional or frequent doctor visits, healthcare dangers (perhaps life-threatening), and/or the treatment being ineffective. Trials are federally governed with exacting guidelines to protect clinical trials subjects.

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



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

For Condition: Ornithine Transcarbamylase Deficiency Disease
Status: Terminated
Sponsor(s): National Institute of Child Health and Human Development (NICHD) , Children's National Medical Center
Synopsis: OBJECTIVES: Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.
Details: 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.
Eligibility:
Study Type:
  Interventional, Treatment
Minimum Age/Maximum Age: 18 Years/65 Years
Genders: Both
Protocol Entry 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
Total Enrollment: 

Location and Contact Information:

Overall Study Official:
MarkBatshaw,  Study Chair,  Children's National Medical Center


Additional Information:
Study ID Numbers:
  199/12054;  CSH-3660
Study Start Date: October 1995
Record last reviewed: January 2000
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00004386

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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