NCT00004443

Brief 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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Oct 1998

Longer than P75 for not_applicable

Geographic Reach
2 countries

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 1998

Completed
1 year until next milestone

First Submitted

Initial submission to the registry

October 18, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 19, 1999

Completed
6.9 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2006

Completed
Last Updated

March 25, 2015

Status Verified

April 1, 2000

First QC Date

October 18, 1999

Last Update Submit

March 24, 2015

Conditions

Tyrosinemia I

Keywords

inborn errors of metabolismrare diseasetyrosinemia I

Interventions

NTBCDRUG

Eligibility Criteria

AgeUp to 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
* 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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (3)

Oregon Health Sciences University

Portland, Oregon, 97201-3098, United States

Location

University of Washington School of Medicine

Seattle, Washington, 98195, United States

Location

Hopital Sainte Justine

Montreal, Quebec, H3T 1C5, Canada

Location

MeSH Terms

Conditions

Metabolism, Inborn ErrorsRare Diseases

Condition Hierarchy (Ancestors)

Genetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • C. Ronald Scott

    University of Washington

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
not applicable
Purpose
TREATMENT
Sponsor Type
OTHER

Study Record Dates

First Submitted

October 18, 1999

First Posted

October 19, 1999

Study Start

October 1, 1998

Study Completion

September 1, 2006

Last Updated

March 25, 2015

Record last verified: 2000-04

Locations

US(2)CA(1)