- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00004333
Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I
OBJECTIVES:
Assess whether 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione (NTBC) at 0.6 mg/kg per day prevents liver failure in at least 1 patient with tyrosinemia type I.
Study Overview
Detailed Description
PROTOCOL OUTLINE: Only 2 patients with tyrosinemia type I are known to this research team; others will be treated if found and if clinical conditions permit.
The enzyme inhibitor 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione (NTBC) is administered orally, in 2 divided doses daily with meals.
Patients will be followed closely for side effects attributable to NTBC.
Study Type
Phase
- Phase 2
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
PROTOCOL ENTRY CRITERIA:
Tyrosinemia type I No cirrhosis No hepatocellular carcinoma
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
Collaborators and Investigators
Collaborators
Investigators
- Study Chair: Jess G. Thoene, University of Michigan
Study record dates
Study Major Dates
Study Start
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ESTIMATE)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 199/11892
- UMMC-1319
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Tyrosinemia, Type I
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Vitaflo International, LtdCompletedAlkaptonuria | Tyrosinemias | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type IIIUnited Kingdom
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metaX Institut fuer Diatetik GmbHGreat Ormond Street Hospital for Children NHS Foundation Trust; Birmingham...Not yet recruitingAlkaptonuria | Homocystine; Metabolic Disorder | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type III | MSUD (Maple Syrup Urine Disease)
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Yassin Abdelghaffar Charity Center for Liver Disease...Society of Friends of Liver Patients in the Arab World (SLPAW)RecruitingHereditary Tyrosinemia, Type IEgypt
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Cycle Pharmaceuticals Ltd.ParexelCompletedHereditary Tyrosinemia, Type ISouth Africa
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Swedish Orphan BiovitrumCompletedHereditary Tyrosinemia, Type IGermany, United Kingdom, France
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Cycle Pharmaceuticals Ltd.ParexelCompletedHereditary Tyrosinemia, Type ISouth Africa
-
Swedish Orphan BiovitrumCompletedEfficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 (HT-1)Hereditary Tyrosinemia, Type IBelgium, Denmark, France, Germany, Sweden
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Swedish Orphan BiovitrumCompletedHereditary Tyrosinemia, Type IBelgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
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Cycle Pharmaceuticals Ltd.ParexelCompletedHereditary Tyrosinemia, Type ISouth Africa
-
Swedish Orphan BiovitrumRecruitingHereditary Tyrosinemia, Type IChina
Clinical Trials on NTBC
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University of WashingtonCompleted
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University of California, San DiegoCompleted
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National Human Genome Research Institute (NHGRI)Completed
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University of FloridaCompleted