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Trial record 1 of 5 for:    "tyrosinemia"
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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
University of Washington
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: October 18, 1999
Last updated: June 23, 2005
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
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.


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
  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 identifier: NCT00004443

United States, Oregon
Oregon Health Sciences University Recruiting
Portland, Oregon, United States, 97201-3098
Contact: Markus Grompe    503-494-6888      
United States, Washington
University of Washington School of Medicine Recruiting
Seattle, Washington, United States, 98195
Contact: C. Ronald Scott    206-543-3370      
Canada, Quebec
Hopital Sainte Justine Recruiting
Montreal, Quebec, Canada, H3T 1C5
Contact: Grant Mitchell    514-345-4931      
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: June 23, 2005
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 February 27, 2015