Trials Sponsors Conditions Drugs Pricing

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

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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

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Sponsors & Collaborators

University of Pennsylvania
Otherlead

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 Diseases

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 1998

Date type not reported

This date has not been verified as actual or estimated.

Study Completion

September 2000

Date type not reported

This date has not been verified as actual or estimated.

Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Study Completion

Conditions

Keywords

Interventions

Eligibility Criteria

Sponsors & Collaborators

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

Source record

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Study Completion

Conditions

Keywords

Interventions

Eligibility Criteria

Sponsors & Collaborators

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

Source record