NCT00004307

Brief Summary

RATIONALE: The urea cycle is the process in which nitrogen is removed from the blood and converted into urea, a waste product found in urine . Urea cycle disorders are inherited disorders caused by the lack of an enzyme that removes ammonia from the bloodstream. Gene therapy is treatment given to change a gene so that it functions normally. Studying the treatment and metabolism of patients with urea cycle disorders may be helpful in developing new treatments for these disorders. PURPOSE: Two-part clinical trial to study the treatment and metabolism of patients who have urea cycle disorders.

Trial Health

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Trial Health Score

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

Enrollment
66

participants targeted

Target at P75+ for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

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

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
1 month until next milestone

Study Start

First participant enrolled

December 1, 1999

Completed
Last Updated

June 24, 2005

Status Verified

December 1, 2003

First QC Date

October 18, 1999

Last Update Submit

June 23, 2005

Conditions

Amino Acid Metabolism, Inborn Errors

Keywords

inborn errors of metabolismrare diseaseurea cycle disorder

Interventions

Protein and calorie controlled dietBEHAVIORAL
Ornithine transcarbamylase vectorGENETIC

Eligibility Criteria

Age6 Months - 64 Years
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Baylor College of Medicine

Houston, Texas, 77030, United States

RECRUITING

MeSH Terms

Conditions

Amino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsRare DiseasesUrea Cycle Disorders, Inborn

Interventions

ProteinsCaloric Restriction

Condition Hierarchy (Ancestors)

Genetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System Diseases

Intervention Hierarchy (Ancestors)

Amino Acids, Peptides, and ProteinsDiet TherapyNutrition TherapyTherapeuticsEnergy IntakeDietNutritional Physiological PhenomenaDiet, Food, and NutritionPhysiological Phenomena

Study Officials

  • Brendan Lee

    Baylor College of Medicine

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Purpose
TREATMENT
Sponsor Type
NIH

Study Record Dates

First Submitted

October 18, 1999

First Posted

October 19, 1999

Study Start

December 1, 1999

Last Updated

June 24, 2005

Record last verified: 2003-12

Locations

US(1)