Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I

Tyrosinemia, Type I Clinical Trial

Status Completed

Clinical Trial Summary

OBJECTIVES:

Assess whether 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione (NTBC) at 0.6 mg/kg per day prevents liver failure in at least 1 patient with tyrosinemia type I.

Clinical Trial Description

PROTOCOL OUTLINE: Only 2 patients with tyrosinemia type I are known to this research team; others will be treated if found and if clinical conditions permit.

The enzyme inhibitor 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione (NTBC) is administered orally, in 2 divided doses daily with meals.

Patients will be followed closely for side effects attributable to NTBC. ;

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Tyrosinemia, Type I
• Tyrosinemias

• NCT number: NCT00004333
• Study type: Interventional
• Source: Office of Rare Diseases (ORD)
• Status: Completed
• Phase: Phase 2
• Start date: November 1994