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.

Condition	Treatment or Intervention	Phase
alpha 1-Antitrypsin Deficiency	Drug: 4 Phenyl Butyrate (4PBA)	Phase II

Ages Eligible for Study:  18 Years   -   65 Years,  Genders Eligible for Study:  Both

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 withold 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

Dawn Baker, MSN      (352) 392-8284    dawn@alphaone.ufl.edu
Pam Schreck, MSN      (352)294-0512    pschreck@ufl.edu

Florida
      University of Florida Health Science Center, Gainesville,  Florida,  32610-0225,  United States; Recruiting

Study chairs or principal investigators

Mark L Brantly, MD,  Principal Investigator,  University of Florida   

Study ID Numbers:  87-2001
Record last reviewed:  August 2003
Record first received:  August 26, 2003
ClinicalTrials.gov Identifier:  NCT00067756
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2004-10-22