4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?
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.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||"4 Phenyl Butyrate Mediated Secretion Rescue in Alpha 1-Antitrypsin Deficient Individuals"|
- To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease. [ Time Frame: 10 days ] [ Designated as safety issue: Yes ]
- To determine the pharmacokinetics of 4-PBA [ Time Frame: 10 days ] [ Designated as safety issue: Yes ]
|Study Start Date:||November 2001|
|Study Completion Date:||October 2003|
|Primary Completion Date:||October 2003 (Final data collection date for primary outcome measure)|
The study will involve a dose escalation and pharmacokinetics component The study group will be comprised of a total of 10 patients equally divided into PiZZ* AAT-deficient individuals with (n=5) and without (n=5) clinical evidence of mild to moderate hepatocellular injury.
Drug: 4 Phenyl Butyrate (4PBA)
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 Name: 4-phenyl butyric acid
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.
|United States, Florida|
|University of Florida|
|Gainesville, Florida, United States, 32610|
|Principal Investigator:||Mark L Brantly, MD||University of Florida|