Study of NTBC for Tyrosinemia I
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT00004443 |
Recruitment Status :
Completed
First Posted : October 19, 1999
Last Update Posted : March 25, 2015
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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 or disease | Intervention/treatment | Phase |
---|---|---|
Tyrosinemia I | Drug: NTBC | Not Applicable |
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
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 20 participants |
Primary Purpose: | Treatment |
Study Start Date : | October 1998 |
Study Completion Date : | September 2006 |


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Ages Eligible for Study: | up to 17 Years (Child) |
Sexes Eligible for Study: | All |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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 |
Study Chair: | C. Ronald Scott | University of Washington |
ClinicalTrials.gov Identifier: | NCT00004443 |
Other Study ID Numbers: |
199/13443 UWASH-FDR001445 |
First Posted: | October 19, 1999 Key Record Dates |
Last Update Posted: | March 25, 2015 |
Last Verified: | April 2000 |
inborn errors of metabolism rare disease tyrosinemia I |
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 |