Clinical Trials Logo
  1. Home
  2. Tyrosinemia I
  3. NCT00004443
  4. Details
NCT00004443 Clinical Trial

Study of NTBC for Tyrosinemia I

Tyrosinemia I Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT00004443
Other study ID # 199/13443
Secondary ID UWASH-FDR001445
Status Completed
Phase N/A
First received October 18, 1999
Last updated March 24, 2015
Start date October 1998
Est. completion date September 2006

Study information
Verified date April 2000
Source FDA Office of Orphan Products Development
Contact n/a
Is FDA regulated No
Health authority United States: Federal Government
Study type Interventional

Clinical Trial Summary

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.

Description:

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

Recruitment information / eligibility
Status Completed
Enrollment 20
Est. completion date September 2006
Est. primary completion date
Accepts healthy volunteers No
Gender Both
Age group N/A to 17 Years
Eligibility - 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

Study Design

Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment

Related Conditions & MeSH terms

Intervention

Drug:
• NTBC

Locations
Country Name City State
Canada Hopital Sainte Justine Montreal Quebec
United States Oregon Health Sciences University Portland Oregon
United States University of Washington School of Medicine Seattle Washington

Sponsors (1)
Lead Sponsor Collaborator
University of Washington

Countries where clinical trial is conducted

United States,  Canada,