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Phase I Pilot Study of Liver-Directed Gene Therapy for 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 treatment with a real dr.. 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 website dedicated 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 medical trials occur in many of places across the U.S.A.. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally assess the effectiveness of new does drugs. The role of the studies / undertakings is to figure out certain human healthcare questions. Clinical trials are a popular means for doctors, government agencies, and private sector corporations to locate treatments for all forms of circumstances, including 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 permit volunteers to get medical treatment options before they are available to the masses. Most times the human subjects acquire treatment for free of charge, and sometimes they are paid for their time. Occasionally there is a cost for a Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency clinical trial. Participants oftentimes recieve the finest healthcare available for their Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency condition. Dangers are a reality, nonetheless, and might include extra or frequent physician calls, health hazards (potentially life-endangering), and/or the treatment being ineffectual. Trials are federally regulated with strict 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 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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