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

Tyrosinemia, Type I Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004333
• Other study ID #: 199/11892
• Secondary ID: UMMC-1319
• Status: Completed
• Phase: Phase 2
• First received: October 18, 1999
• Last updated: June 23, 2005
• Start date: November 1994

Study information

• Verified date: July 2000
• Source: Office of Rare Diseases (ORD)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

PROTOCOL ENTRY CRITERIA:

Tyrosinemia type I No cirrhosis No hepatocellular carcinoma

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Tyrosinemia, Type I
• Tyrosinemias

Intervention

Drug:
• NTBC

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
National Center for Research Resources (NCRR)	University of Michigan