Trials / Completed
CompletedNCT00004333
Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- —
- Sponsor
- National Center for Research Resources (NCRR) · NIH
- Sex
- All
- Age
- 0 Years
- Healthy volunteers
- Not accepted
Summary
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.
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | NTBC |
Timeline
- Start date
- 1994-11-01
- First posted
- 1999-10-19
- Last updated
- 2005-06-24
Source: ClinicalTrials.gov record NCT00004333. Inclusion in this directory is not an endorsement.