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Study of NTBC for Tyrosinemia I

Sponsor
University of Washington (Other)
Overall Status
Completed
CT.gov ID
NCT00004443
Collaborator
(none)
20
Enrollment
3
Locations
95
Duration (Months)
6.7
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

OBJECTIVES: I. Assess the safety and efficacy of NTBC in children with tyrosinemia I.

  1. 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.
Condition or Disease Intervention/Treatment Phase
N/A

Detailed 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

Study Design

Study Type:
Interventional
Anticipated Enrollment :
20 participants
Primary Purpose:
Treatment
Study Start Date :
Oct 1, 1998
Study Completion Date :
Sep 1, 2006

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A to 17 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    • 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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Oregon Health Sciences University Portland Oregon United States 97201-3098
    2 University of Washington School of Medicine Seattle Washington United States 98195
    3 Hopital Sainte Justine Montreal Quebec Canada H3T 1C5

    Sponsors and Collaborators

    • University of Washington

    Investigators

    • Study Chair: C. Ronald Scott, University of Washington

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    , ,
    ClinicalTrials.gov Identifier:
    NCT00004443
    Other Study ID Numbers:
    • 199/13443
    • UWASH-FDR001445
    First Posted:
    Oct 19, 1999
    Last Update Posted:
    Mar 25, 2015
    Last Verified:
    Apr 1, 2000
    Keywords provided by , ,
    inborn errors of metabolism
    rare disease
    tyrosinemia I
    Additional relevant MeSH terms:
    Tyrosinemias

    Study Results

    No Results Posted as of Mar 25, 2015