Expired Study
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Portland, Oregon 97201


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.


Study summary:

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


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 is Available At:


Original ID:

199/13443


NCT ID:

NCT00004443


Secondary ID:

UWASH-FDR001445


Study Acronym:


Brief Title:

Study of NTBC for Tyrosinemia I


Official Title:


Overall Status:

Completed


Study Phase:

N/A


Genders:

Both


Minimum Age:

N/A


Maximum Age:

17 Years


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

FDA Office of Orphan Products Development


Oversight Authority:

United States: Federal Government


Reasons Why Stopped:


Study Type:

Interventional


Study Design:

Endpoint Classification: Safety/Efficacy Study, Pr


Number of Arms:

0


Number of Groups:

0


Total Enrollment:

20


Enrollment Type:

Anticipated


Overall Contact Information

Official Name:C. Ronald Scott
Study Chair
University of Washington

Study Dates

Start Date:October 1998
Completion Date:September 2006
Verification Date:April 2000
Last Changed Date:March 24, 2015
First Received Date:October 18, 1999

Study Outcomes

There are no available Study Outcomes

Study Interventions

Intervention Type:Drug
Name:NTBC

Study Arms

There are no available Study Arms

Study Agencies

Agency Class:Other
Agency Type:Lead Sponsor
Agency Name:University of Washington

Sample and Retention Information

There are no available Sample and Retention Information

Study References

There are no available Study References

Data Source: ClinicalTrials.gov

Date Processed: January 21, 2020

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