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.
The purpose of this study is to determine whether 4-PBA will significantly increase serum Z
AAT levels in AAT-deficient individuals with and without evidence of hepatocellular injury
and to assess its effects on liver injury.
- 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)
- 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