Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders

This study has been completed.

Sponsor:

Collaborator:

Johns Hopkins University

Information provided by:

Office of Rare Diseases (ORD)

ClinicalTrials.gov Identifier:

NCT00004767

First received: February 24, 2000

Last updated: June 23, 2005

Last verified: December 2001

History of Changes

Tracking Information

First Received Date ^ICMJE

February 24, 2000

Last Updated Date

June 23, 2005

Start Date ^ICMJE

January 1985

Primary Completion Date

Not Provided

Current Primary Outcome Measures ^ICMJE

Not Provided

Original Primary Outcome Measures ^ICMJE

Not Provided

Change History

Complete list of historical versions of study NCT00004767 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Not Provided

Original Secondary Outcome Measures ^ICMJE

Not Provided

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders

Official Title ^ICMJE

Not Provided

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.

Detailed Description

PROTOCOL OUTLINE: This protocol describes several clinical studies of pharmacologic and dietary management in patients with urea cycle disorders.

Patients with carbamyl phosphate synthetase and ornithine transcarbamylase deficiency are treated with a low-protein diet, essential amino acids (for neonatal onset disease), caloric supplementation, oral sodium phenylbutyrate (now approved as a prescription drug 11/97), and citrulline or arginine free base.

Patients with argininosuccinic acid synthetase deficiency are treated with a low-protein diet, caloric supplementation, oral sodium phenylbutyrate (now approved as a prescription drug 11/97), and arginine free base.

Patients with argininosuccinic aciduria (AA) are treated with a low-protein diet, caloric supplementation, and arginine free base. (Discontinued 11/97) Any patient who develops hyperammonemia is treated with intravenous sodium benzoate, sodium phenylbutyrate, and arginine hydrochloride; benzoate and phenylbutyrate are not given to patients with AA.

If ammonium stabilizes at normal or near normal levels, intravenous medications are gradually replaced by oral medications. If there is no significant decrease in ammonium within 8 hours, patients begin hemodialysis.

Concurrent therapy with ondansetron, high caloric intake, and mannitol for elevated intracranial pressure is allowed. Dietary and intravenous nitrogen is prohibited. (Discontinued 11/97)

Study Type ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Primary Purpose: Treatment

Condition ^ICMJE

Amino Acid Metabolism, Inborn Errors

Intervention ^ICMJE

Drug: Sodium Benzoate
Drug: Sodium Phenylacetate
Drug: Sodium Phenylbutyrate
Behavioral: Dietary Intervention

Study Arm (s)

Not Provided

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Completed

Enrollment ^ICMJE

Completion Date

Not Provided

Primary Completion Date

Not Provided

Eligibility Criteria ^ICMJE

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)

Gender

Both

Ages

18 Years to 65 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

Not Provided

Administrative Information

NCT Number ^ICMJE

NCT00004767

Other Study ID Numbers ^ICMJE

199/11753, JHUSM-11753

Has Data Monitoring Committee

Not Provided

Responsible Party

Not Provided

Study Sponsor ^ICMJE

National Center for Research Resources (NCRR)

Collaborators ^ICMJE

Johns Hopkins University

Investigators ^ICMJE

Study Chair:

Saul W. Brusilow

Johns Hopkins University

Information Provided By

Office of Rare Diseases (ORD)

Verification Date

December 2001

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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