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Study of NTBC for Tyrosinemia I

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ClinicalTrials.gov Identifier: NCT00004443
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : March 25, 2015
Sponsor:
Information provided by:
FDA Office of Orphan Products Development

Study Description
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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.


Condition or disease Intervention/treatment Phase
Tyrosinemia I Drug: NTBC Not Applicable

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
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Primary Purpose: Treatment
Study Start Date : October 1998
Study Completion Date : September 2006

Resource links provided by the National Library of Medicine


Arms and Interventions
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Outcome Measures
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Eligibility Criteria
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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
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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
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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
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Study Chair: C. Ronald Scott University of Washington
More Information
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ClinicalTrials.gov Identifier: NCT00004443    
Other Study ID Numbers: 199/13443
UWASH-FDR001445
First Posted: October 19, 1999    Key Record Dates
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:
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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