Study of NTBC for Tyrosinemia I
Recruitment status was Recruiting
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:||Endpoint Classification: Safety/Efficacy Study
Primary Purpose: Treatment
|Study Start Date:||October 1998|
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.
|United States, Oregon|
|Oregon Health Sciences University||Recruiting|
|Portland, Oregon, United States, 97201-3098|
|Contact: Markus Grompe 503-494-6888|
|United States, Washington|
|University of Washington School of Medicine||Recruiting|
|Seattle, Washington, United States, 98195|
|Contact: C. Ronald Scott 206-543-3370|
|Hopital Sainte Justine||Recruiting|
|Montreal, Quebec, Canada, H3T 1C5|
|Contact: Grant Mitchell 514-345-4931|
|Study Chair:||C. Ronald Scott||University of Washington|