Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00004333|
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : June 24, 2005
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 or disease||Intervention/treatment||Phase|
|Tyrosinemia, Type I||Drug: NTBC||Phase 2|
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 Type :||Interventional (Clinical Trial)|
|Study Start Date :||November 1994|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004333
|Study Chair:||Jess G. Thoene||University of Michigan|