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Study of NTBC for Tyrosinemia I

This study has been completed.
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: October 18, 1999
Last updated: March 24, 2015
Last verified: April 2000

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:

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


Ages Eligible for Study:   up to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
  • 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
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Please refer to this study by its identifier: NCT00004443

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
Study Chair: C. Ronald Scott University of Washington
  More Information

No publications provided 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:
Amino Acid Metabolism, Inborn Errors
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Genetic Diseases, Inborn
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases processed this record on November 30, 2015