Study of NTBC for Tyrosinemia I

This study has been completed.
Sponsor:
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004443
First received: October 18, 1999
Last updated: March 24, 2015
Last verified: April 2000

October 18, 1999
March 24, 2015
October 1998
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Complete list of historical versions of study NCT00004443 on ClinicalTrials.gov Archive Site
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Study of NTBC for Tyrosinemia I
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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.

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

Interventional
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Endpoint Classification: Safety/Efficacy Study
Primary Purpose: Treatment
Tyrosinemia I
Drug: NTBC
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
20
September 2006
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  • 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
Both
up to 17 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
United States,   Canada
 
NCT00004443
199/13443, UWASH-FDR001445
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University of Washington
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Study Chair: C. Ronald Scott University of Washington
FDA Office of Orphan Products Development
April 2000

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP