CompletedNot Applicable

Study of NTBC for Tyrosinemia I

United States, Canada20 participantsStarted 1998-10

Plain-language 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.

Who can participate

Age range17 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

* 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

Trial details

NCT IDNCT00004443

SponsorUniversity of Washington

Sponsor typeOTHER

Study typeINTERVENTIONAL

View on ClinicalTrials.gov More Tyrosinemia I trials