Study of NTBC for Tyrosinemia 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: NCT00004443|
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : March 25, 2015
OBJECTIVES: I. Assess the safety and efficacy of NTBC in children with tyrosinemia I.
II. Evaluate the effects of NTBC on survival, rate of neurologic crises, improvement in renal tubular damage, reduction in the need for liver transplantation, and reduction in the development of hepatocarcinoma in these patients.
|Condition or disease||Intervention/treatment||Phase|
|Tyrosinemia I||Drug: NTBC||Not Applicable|
PROTOCOL OUTLINE: Patients are stratified according to age at onset of symptoms (0-2 months vs 2-6 months vs greater than 6 months).
Patients receive oral NTBC twice a day. Treatment continues in the absence of unacceptable toxicity.
Patients are followed once a month for 6 months, then every 3 months thereafter.
Completion date provided represents the completion date of the grant per OOPD records
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Study Start Date :||October 1998|
|Study Completion Date :||September 2006|
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): NCT00004443
|United States, Oregon|
|Oregon Health Sciences University|
|Portland, Oregon, United States, 97201-3098|
|United States, Washington|
|University of Washington School of Medicine|
|Seattle, Washington, United States, 98195|
|Hopital Sainte Justine|
|Montreal, Quebec, Canada, H3T 1C5|
|Study Chair:||C. Ronald Scott||University of Washington|