Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

1994-11-30

Brief Summary

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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

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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

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Tyrosinemia, Type I

Study Design

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Primary Study Purpose

TREATMENT

Interventions

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NTBC

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

PROTOCOL ENTRY CRITERIA:

Tyrosinemia type I No cirrhosis No hepatocellular carcinoma

Minimum Eligible Age

0 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Principal Investigators

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Jess G. Thoene

Role: STUDY_CHAIR

University of Michigan

Other Identifiers

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UMMC-1319

Identifier Type: -

Identifier Source: secondary_id

199/11892