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Trial record 1 of 9 for:    "tyrosinemia"
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Study of NTBC for Tyrosinemia I

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00004443
First Posted: October 19, 1999
Last Update Posted: March 25, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
FDA Office of Orphan Products Development
  Purpose

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.


Condition Intervention
Tyrosinemia I Drug: NTBC

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 20
Study Start Date: October 1998
Estimated Study Completion Date: September 2006
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

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004443


Locations
United States, Oregon
Oregon Health Sciences University
Portland, Oregon, United States, 97201-3098
United States, Washington
University of Washington School of Medicine
Seattle, Washington, United States, 98195
Canada, Quebec
Hopital Sainte Justine
Montreal, Quebec, Canada, H3T 1C5
Sponsors and Collaborators
University of Washington
Investigators
Study Chair: C. Ronald Scott University of Washington
  More Information

ClinicalTrials.gov Identifier: NCT00004443     History of Changes
Other Study ID Numbers: 199/13443
UWASH-FDR001445
First Submitted: October 18, 1999
First Posted: October 19, 1999
Last Update Posted: March 25, 2015
Last Verified: April 2000

Keywords provided by FDA Office of Orphan Products Development:
inborn errors of metabolism
rare disease
tyrosinemia I

Additional relevant MeSH terms:
Tyrosinemias
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases