A Phase 2 interventional study of NTBC in Tyrosinemia, Type I, sponsored by National Center for Research Resources (NCRR). Completed. Open to participants aged 0 Years and older. Per ClinicalTrials.gov, last updated 2005-06-24.
Sponsored by National Center for Research Resources (NCRR) · Phase 2, Interventional, and Treatment
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.
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.
21 studies on the registry are indexed under Tyrosinemias; 3 are open to participants now.
Browse Tyrosinemias studies →National Center for Research Resources (NCRR) is the lead sponsor of 265 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
PROTOCOL ENTRY CRITERIA:
Tyrosinemia type I No cirrhosis No hepatocellular carcinoma
No study locations are listed for this record.
This study is completed, as verified in Jul 2000. You cannot join it, but the record below documents what was studied.
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National Center for Research Resources (NCRR)