Completed

Study of NTBC for Tyrosinemia I

0 criteria met from your profileSee at a glance how your profile meets each eligibility criteria.
What is being tested

NTBC

Drug
Who is being recruted

Metabolic Diseases+6

+ Congenital, Hereditary, and Neonatal Diseases and Abnormalities

+ Nutritional and Metabolic Diseases

Until 17 Years
See all eligibility criteria
How is the trial designed

Treatment Study

Interventional
Study Start: October 1998
See protocol details

Summary

Principal SponsorUniversity of Washington
Last updated: March 25, 2015
Sourced from a government-validated database.Claim as a partner

Study start date: October 1, 1998

Actual date on which the first participant was enrolled.

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

NCT00004443
Principal SponsorUniversity of Washington
Last updated: March 25, 2015
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

20 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Until 17 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Metabolic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNutritional and Metabolic DiseasesPathologic ProcessesPathological Conditions, Signs and SymptomsDisease AttributesGenetic Diseases, InbornMetabolism, Inborn ErrorsRare Diseases

Criteria

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 Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 3 locations

Oregon Health Sciences University

Portland, United StatesOpen Oregon Health Sciences University in Google Maps

University of Washington School of Medicine

Seattle, United States

Hopital Sainte Justine

Montreal, Canada
Completed3 Study Centers
Study of NTBC for Tyrosinemia I | PatLynk