Expired Study
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Portland, Oregon 97201


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.


Study summary:

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


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


NCT ID:

NCT00004443


Primary Contact:

Study Chair
C. Ronald Scott
University of Washington


Backup Contact:

N/A


Location Contact:

Portland, Oregon 97201
United States



There is no listed contact information for this specific location.

Site Status: N/A


Data Source: ClinicalTrials.gov

Date Processed: November 18, 2019

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