Study of NTBC for Tyrosinemia I

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Disease Information

Descriptive Information
Brief Title † Study of NTBC for Tyrosinemia I
Official Title †
Brief Summary 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.
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
Study Phase N/A
Study Type † Interventional
Study Design † Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
Primary Outcome Measure †
Secondary Outcome Measure †
Condition † Tyrosinemia I
Intervention † DrugNTBC
Study Arms / Comparison Groups
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 20
Start Date † October 1998
Completion Date September 2006
Primary Completion Date
Eligibility 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
Gender Both
Ages N/A - 17 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT00004443
Organization ID 199/13443
Secondary IDs †† UWASH-FDR001445
Responsible Party
Study Sponsor † University of Washington
Collaborators ††
Investigators † Study Chair: C. Ronald Scott, University of Washington
Information Provided By
Verification Date April 2000
First Received Date † October 18, 1999
Last Updated Date March 24, 2015
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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