4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?

• Status: Completed
• Trial end date: 2003-10-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to find out whether 4-PBA will increase the level of AAT in persons with AAT deficiency whether or not they have liver disease.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Florida
• Collaborators: Alpha-1 Foundation; Brantly, Mark L., M.D.
• Treatments: Alpha 1-Antitrypsin; Butyric Acid; Protein C Inhibitor

Criteria

Inclusion Criteria:

• Age 18-65

• Serum A1-PI levels <11uM an appropriate genetic phenotype/genotype

• 5 of 10 subjects must have documented laboratory evidence of liver disease

• Willingness to withhold Prolastin therapy for 6 weeks prior to screening and throughout the 4-PBA dosing period (up to 3 months)

Exclusion Criteria:

• Any cause of liver disease other than Alpha-1 Antitrypsin deficiency

• Evidence of advanced liver disease

• HIV positive

• Use of systemic steroids, ursodeoxycholic acid (Actigall, Urso), or herbs in the prior 6 months