Study of NTBC for Tyrosinemia I

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.

Timeline

Start

1998-10

Primary completion

—

Completion

2006-09

Drugs

Indications