Study of NTBC for Tyrosinemia I

Tyrosinemia I Clinical Trial

Status Completed

Clinical Trial 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.

Clinical Trial 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 Design

Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Tyrosinemia I
• Tyrosinemias

• NCT number: NCT00004443
• Study type: Interventional
• Source: FDA Office of Orphan Products Development
• Status: Completed
• Phase: N/A
• Start date: October 1998
• Completion date: September 2006