NCT00004767

Brief Summary

OBJECTIVES: I. Assess the safety and efficacy of sodium phenylbutyrate, sodium benzoate, sodium phenylacetate, and dietary intervention in patients with urea cycle disorders.

Trial Health

100
On Track

Trial Health Score

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

Enrollment
20

participants targeted

Target at below P25 for phase_2

Status
completed

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

January 1, 1985

Completed
15.2 years until next milestone

First Submitted

Initial submission to the registry

February 24, 2000

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 25, 2000

Completed
Last Updated

June 24, 2005

Status Verified

December 1, 2001

First QC Date

February 24, 2000

Last Update Submit

June 23, 2005

Conditions

Amino Acid Metabolism, Inborn Errors

Keywords

inborn errors of metabolismrare diseaseurea cycle disorder

Interventions

Sodium BenzoateDRUG
Sodium PhenylacetateDRUG
Sodium PhenylbutyrateDRUG
Dietary InterventionBEHAVIORAL

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
PROTOCOL ENTRY CRITERIA: Urea cycle deficiency, i.e.: Carbamyl phosphate synthetase deficiency (CPSD) Ornithine transcarbamylase deficiency (OTCD) Argininosuccinic acid synthetase deficiency (ASD) Argininosuccinic aciduria (AA)

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Sponsors & Collaborators

MeSH Terms

Conditions

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

Interventions

Sodium Benzoatephenylacetic acid4-phenylbutyric acidDiet Therapy

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)

Benzoic AcidBenzoatesAcids, CarbocyclicCarboxylic AcidsOrganic ChemicalsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsNutrition TherapyTherapeutics

Study Officials

  • Saul W. Brusilow

    Johns Hopkins University

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Purpose
TREATMENT
Sponsor Type
NIH

Study Record Dates

First Submitted

February 24, 2000

First Posted

February 25, 2000

Study Start

January 1, 1985

Last Updated

June 24, 2005

Record last verified: 2001-12