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

This study has been completed.
Sponsor:
Collaborators:
Alpha-1 Foundation
Brantly, Mark L., M.D.
Information provided by (Responsible Party):
University of Florida
ClinicalTrials.gov Identifier:
NCT00067756
First received: August 26, 2003
Last updated: January 2, 2014
Last verified: April 2013

August 26, 2003
January 2, 2014
November 2001
October 2003   (final data collection date for primary outcome measure)
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 ]
Not Provided
Complete list of historical versions of study NCT00067756 on ClinicalTrials.gov Archive Site
To determine the pharmacokinetics of 4-PBA [ Time Frame: 10 days ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
Not Provided
 
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"

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.

Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Alpha 1-Antitrypsin Deficiency
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 Name: 4-phenyl butyric acid
Experimental: 4-PBA
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.
Intervention: Drug: 4-PBA
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
12
October 2003
October 2003   (final data collection date for primary outcome measure)

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
Both
18 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00067756
87-2001
Yes
University of Florida
University of Florida
  • Alpha-1 Foundation
  • Brantly, Mark L., M.D.
Principal Investigator: Mark L Brantly, MD University of Florida
University of Florida
April 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP