NCT00004498

Brief Summary

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.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jul 1998

Typical duration for phase_1

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 1998

Completed
1.3 years until next milestone

First Submitted

Initial submission to the registry

October 18, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 19, 1999

Completed
11 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2000

Completed
Last Updated

March 25, 2015

Status Verified

February 1, 2000

First QC Date

October 18, 1999

Last Update Submit

March 24, 2015

Conditions

Ornithine Transcarbamylase Deficiency Disease

Keywords

genetic diseases and dysmorphic syndromesinborn errors of metabolismornithine transcarbamylase deficiencyrare diseaseurea cycle disorder

Interventions

Adenoviral Vector-Mediated Gene TransferGENETIC

Eligibility Criteria

Age18 Years - 69 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

MeSH Terms

Conditions

Ornithine Carbamoyltransferase Deficiency DiseaseGenetic Diseases, InbornMetabolism, Inborn ErrorsRare DiseasesUrea Cycle Disorders, Inborn

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesGenetic Diseases, X-LinkedCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesAmino Acid Metabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Steven E. Raper

    University of Pennsylvania

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Purpose
TREATMENT
Sponsor Type
OTHER

Study Record Dates

First Submitted

October 18, 1999

First Posted

October 19, 1999

Study Start

July 1, 1998

Study Completion

September 1, 2000

Last Updated

March 25, 2015

Record last verified: 2000-02