Study of NTBC for Tyrosinemia I

The recruitment status of this study is unknown because the information has not been verified recently.

Verified April 2000 by FDA Office of Orphan Products Development.
Recruitment status was Recruiting

Sponsor:

Collaborator:

University of Washington

Information provided by:

FDA Office of Orphan Products Development

ClinicalTrials.gov Identifier:

NCT00004443

First received: October 18, 1999

Last updated: June 23, 2005

Last verified: April 2000

History of Changes

Tracking Information

First Received Date ^ICMJE

October 18, 1999

Last Updated Date

June 23, 2005

Start Date ^ICMJE

October 1998

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 NCT00004443 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

Study of NTBC for Tyrosinemia I

Official Title ^ICMJE

Not Provided

Brief Summary

OBJECTIVES: I. Assess the safety and efficacy of NTBC in children with tyrosinemia I.

II. Evaluate the effects of NTBC on survival, rate of neurologic crises, improvement in renal tubular damage, reduction in the need for liver transplantation, and reduction in the development of hepatocarcinoma in these patients.

Detailed Description

PROTOCOL OUTLINE: Patients are stratified according to age at onset of symptoms (0-2 months vs 2-6 months vs greater than 6 months).

Patients receive oral NTBC twice a day. Treatment continues in the absence of unacceptable toxicity.

Patients are followed once a month for 6 months, then every 3 months thereafter.

Study Type ^ICMJE

Interventional

Study Phase

Not Provided

Study Design ^ICMJE

Endpoint Classification: Safety/Efficacy Study
Primary Purpose: Treatment

Condition ^ICMJE

Tyrosinemia I

Intervention ^ICMJE

Drug: NTBC

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

Recruiting

Enrollment ^ICMJE

Completion Date

Not Provided

Primary Completion Date

Not Provided

Eligibility Criteria ^ICMJE

Confirmed diagnosis of tyrosinemia Detected through newborn screening before the onset of symptoms OR Diagnosed on the basis of symptoms (liver disease, neurological crises, growth failure) and succinylacetone in urine or blood and/or fumarylacetoacetate dehydratase deficiency in cultured fibroblasts or liver biopsy

Gender

Both

Ages

up to 17 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Not Provided

Location Countries ^ICMJE

United States, Canada

Administrative Information

NCT Number ^ICMJE

NCT00004443

Other Study ID Numbers ^ICMJE

199/13443, UWASH-FDR001445

Has Data Monitoring Committee

Not Provided

Responsible Party

Not Provided

Study Sponsor ^ICMJE

FDA Office of Orphan Products Development

Collaborators ^ICMJE

University of Washington

Investigators ^ICMJE

Study Chair:

C. Ronald Scott

University of Washington

Information Provided By

FDA Office of Orphan Products Development

Verification Date

April 2000

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

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