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

National Center for Research Resources (NCRR)

Status and phase: Completed; Phase 2

Conditions: Tyrosinemia, Type I

Treatments: Drug: NTBC

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT00004333

199/11892

UMMC-1319

Details and patient eligibility

About

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.

Full 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.

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

PROTOCOL ENTRY CRITERIA:

Tyrosinemia type I No cirrhosis No hepatocellular carcinoma