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

Completed · Phase 2

Conditions studied: Alpha 1-Antitrypsin Deficiency

In brief

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.

Key facts

Study ID

NCT00067756

Run by

University of Florida

People needed

12

Starts

2001-11-01

Expected to finish

2003-10-01

Last updated by the study team

2014-01-03

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

• 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

Where it is running

• University of Florida — Gainesville, Florida, United States

Full record on ClinicalTrials.gov

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