Study of NTBC for Tyrosinemia I
The recruitment status of this study is unknown because the information has not been verified recently.
Verified April 2000 by FDA Office of Orphan Products Development.
Recruitment status was Recruiting
Recruitment status was Recruiting
Sponsor:
Collaborator:
University of Washington
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004443
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: April 2000
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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: | Endpoint Classification: Safety/Efficacy Study Primary Purpose: Treatment |
Resource links provided by NLM:
Genetics Home Reference related topics:
argininosuccinic aciduria
citrullinemia
N-acetylglutamate synthase deficiency
ornithine translocase deficiency
succinic semialdehyde dehydrogenase deficiency
tyrosinemia
U.S. FDA Resources
Further study details as provided by FDA Office of Orphan Products Development:
| Estimated Enrollment: | 20 |
| Study Start Date: | October 1998 |
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.
Eligibility| Ages Eligible for Study: | up to 17 Years |
| Genders Eligible for Study: | Both |
| 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004443
Locations
| United States, Oregon | |
| Oregon Health Sciences University | Recruiting |
| Portland, Oregon, United States, 97201-3098 | |
| Contact: Markus Grompe 503-494-6888 | |
| United States, Washington | |
| University of Washington School of Medicine | Recruiting |
| Seattle, Washington, United States, 98195 | |
| Contact: C. Ronald Scott 206-543-3370 | |
| Canada, Quebec | |
| Hopital Sainte Justine | Recruiting |
| Montreal, Quebec, Canada, H3T 1C5 | |
| Contact: Grant Mitchell 514-345-4931 | |
Sponsors and Collaborators
University of Washington
Investigators
| Study Chair: | C. Ronald Scott | University of Washington |
More Information
No publications provided
| ClinicalTrials.gov Identifier: | NCT00004443 History of Changes |
| Other Study ID Numbers: | 199/13443, UWASH-FDR001445 |
| Study First Received: | October 18, 1999 |
| Last Updated: | June 23, 2005 |
| Health Authority: | United States: Federal Government |
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 |
ClinicalTrials.gov processed this record on May 23, 2013