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

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Disease Information

Descriptive Information
Brief Title † Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I
Official Title †
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
Study Design † Primary Purpose: Treatment
Primary Outcome Measure †
Secondary Outcome Measure †
Condition † Tyrosinemia, Type I
Intervention † DrugNTBC
Study Arms / Comparison Groups
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
Estimated Enrollment †
Start Date † November 1994
Completion Date
Primary Completion Date
Eligibility Criteria † PROTOCOL ENTRY CRITERIA: Tyrosinemia type I No cirrhosis No hepatocellular carcinoma
Gender Both
Ages N/A - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries †
Administrative Information
NCT ID † NCT00004333
Organization ID 199/11892
Secondary IDs †† UMMC-1319
Responsible Party
Study Sponsor † National Center for Research Resources (NCRR)
Collaborators †† University of Michigan
Investigators † Study Chair: Jess G. Thoene, University of Michigan
Information Provided By
Verification Date July 2000
First Received Date † October 18, 1999
Last Updated Date June 23, 2005
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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