Study of NTBC for Tyrosinemia I

University of Washington20 enrolled

Overview

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.

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

Purpose

TREATMENT

Enrollment

Conditions

Tyrosinemia I

Interventions

NTBCDRUG

Eligibility

Sex: ALLMax age: 17 Years

Medical Language ↔ Plain English

* Confirmed diagnosis of tyrosinemia Detected through newborn screening before the onset of symptoms OR Diagnosed on the basis of symptoms (liver disease, neurological crises, growth failure) and succinylacetone in urine or blood and/or fumarylacetoacetate dehydratase deficiency in cultured fibroblasts or liver biopsy

Locations (3)

Oregon Health Sciences University

Portland, Oregon, United States

University of Washington School of Medicine

Seattle, Washington, United States

Hopital Sainte Justine

Montreal, Quebec, Canada

Data from ClinicalTrials.gov

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