4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?
"4 Phenyl Butyrate Mediated Secretion Rescue in Alpha 1-Antitrypsin Deficient Individuals"
  • Phase

    Phase 2
  • Study Type

  • Status

    Completed No Results Posted
  • Study Participants

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.
Study Started
Nov 30
Primary Completion
Oct 31
Study Completion
Oct 31
Last Update
Jan 03

Drug 4-PBA

During the first 3 days of this phase baseline serum AAT levels will be determined. The participants will be then given increased amounts of 4-PBA orally in 6 divided doses (day 4-6, 30 g/day and day 7-9, 40/day

  • Other names: 4-phenyl butyric acid

4-PBA Experimental

The study will involve a 4-PBA dose escalation and pharmacokinetics component The study group will be comprised of a total of at least 10 AAT-deficient,(phenotype ZZ referred to as PiZZ) patients. These patients will be divided into two groups: with and without clinical evidence of mild to moderate hepatocellular injury.


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
No Results Posted