Back to resultsPhase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
Primary Purpose
Ornithine Transcarbamylase Deficiency Disease
Status
Terminated
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
recombinant adenovirus containing the ornithine transcarbamylase gene
Sponsored by
About this trial
This is an interventional treatment trial for Ornithine Transcarbamylase Deficiency Disease focused on measuring inborn errors of metabolism, rare disease, urea cycle disorder
Eligibility 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
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT00004386
First Posted
October 18, 1999
Last Updated
June 23, 2005
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborators
Children's National Research Institute
1. Study Identification
Unique Protocol Identification Number
NCT00004386
Brief Title
Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
Study Type
Interventional
2. Study Status
Record Verification Date
January 2000
Overall Recruitment Status
Terminated
Study Start Date
October 1995 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborators
Children's National Research Institute
4. Oversight
5. Study Description
Brief Summary
OBJECTIVES:
Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Ornithine Transcarbamylase Deficiency Disease
Keywords
inborn errors of metabolism, rare disease, urea cycle disorder
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
8. Arms, Groups, and Interventions
Intervention Type
Genetic
Intervention Name(s)
recombinant adenovirus containing the ornithine transcarbamylase gene
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mark Batshaw
Organizational Affiliation
Children's National Research Institute
Official's Role
Study Chair
12. IPD Sharing Statement
Learn more about this trial
Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
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