Study of NTBC for Tyrosinemia I

• Status: Completed
• Trial end date: 2006-09-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Washington

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