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

Brief Title

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


Brief 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.
    

Detailed 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 Phase

Phase 2

Study Type

Interventional




Condition

Tyrosinemia, Type I

Intervention

NTBC


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug


Start Date

November 1994



Eligibility Criteria

        PROTOCOL ENTRY CRITERIA:

        Tyrosinemia type I No cirrhosis No hepatocellular carcinoma
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Jess G. Thoene, , 



Administrative Informations


NCT ID

NCT00004333

Organization ID

199/11892

Secondary IDs

UMMC-1319


Study Sponsor

National Center for Research Resources (NCRR)

Collaborators

 University of Michigan

Study Sponsor

Jess G. Thoene, Study Chair, University of Michigan


Verification Date

July 2000