Study of NTBC for Tyrosinemia I

This study has been completed.

Sponsor:

Information provided by:

FDA Office of Orphan Products Development

ClinicalTrials.gov Identifier:

NCT00004443

First received: October 18, 1999

Last updated: March 24, 2015

Last verified: April 2000

History of Changes

Purpose

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.

Condition	Intervention
Tyrosinemia I	Drug: NTBC


Study Type:	Interventional
Study Design:	Endpoint Classification: Safety/Efficacy Study Primary Purpose: Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: N-acetylglutamate synthase deficiency argininosuccinic aciduria citrullinemia ornithine translocase deficiency succinic semialdehyde dehydrogenase deficiency tyrosinemia

Genetic and Rare Diseases Information Center resources: Tyrosinemia Type 1 Tyrosinemia Type 2 Tyrosinemia Type 3 Inborn Amino Acid Metabolism Disorder

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:


Estimated Enrollment:	20
Study Start Date:	October 1998
Estimated Study Completion Date:	September 2006

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.

Completion date provided represents the completion date of the grant per OOPD records

Eligibility


Ages Eligible for Study:	up to 17 Years (Child)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004443

Locations


United States, Oregon
Oregon Health Sciences University
Portland, Oregon, United States, 97201-3098
United States, Washington
University of Washington School of Medicine
Seattle, Washington, United States, 98195
Canada, Quebec
Hopital Sainte Justine
Montreal, Quebec, Canada, H3T 1C5

Sponsors and Collaborators

University of Washington

Investigators


Study Chair:	C. Ronald Scott	University of Washington

More Information


ClinicalTrials.gov Identifier:	NCT00004443 History of Changes
Other Study ID Numbers:	199/13443 UWASH-FDR001445
Study First Received:	October 18, 1999
Last Updated:	March 24, 2015
Health Authority:	United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:


inborn errors of metabolism rare disease tyrosinemia I

Additional relevant MeSH terms:


Tyrosinemias Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases	Nervous System Diseases Amino Acid Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Metabolic Diseases

ClinicalTrials.gov processed this record on September 23, 2016

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