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

This study has been completed.
Sponsor:
Collaborator:
University of Michigan
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00004333
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: July 2000
History of Changes
  Purpose

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.


Condition Intervention Phase
Tyrosinemia, Type I
 Drug: NTBC
 Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Office of Rare Diseases (ORD):

Study Start Date: November 1994
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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

PROTOCOL ENTRY CRITERIA:

Tyrosinemia type I No cirrhosis No hepatocellular carcinoma

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004333

Sponsors and Collaborators
National Center for Research Resources (NCRR)
University of Michigan
Investigators
Study Chair: Jess G. Thoene University of Michigan
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00004333     History of Changes
Other Study ID Numbers: 199/11892, UMMC-1319
Study First Received: October 18, 1999
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):
inborn errors of metabolism
rare disease
tyrosinemia I

Additional relevant MeSH terms:
Tyrosinemias
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 21, 2013