Study of NTBC for Tyrosinemia I
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.
|Study Design:||Primary Purpose: Treatment|
|Study Start Date:||October 1998|
|Estimated Study Completion Date:||September 2006|
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
Please refer to this study by its ClinicalTrials.gov identifier: 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|