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

Phase 2

Completed

• Conditions: Alpha 1-Antitrypsin Deficiency
• Interventions: Drug: 4-PBA

• Registration Number: NCT00067756
• Lead Sponsor: University of Florida

Brief Summary

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.

Detailed Description

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 Timeline

• Study Start: 2001-11
• Study First Submitted: 2003-08-26
• Study First Submitted QC: 2003-08-26
• Study First Posted: 2003-08-27
• Primary Completion: 2003-10
• Study Completion: 2003-10
• Status Verified: 2013-04
• Last Update Submitted: 2014-01-02
• Last Update Posted: 2014-01-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
4-PBA	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.

Primary Outcome Measures

Name	Time	Method
To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease.	10 days

Secondary Outcome Measures

Name	Time	Method
To determine the pharmacokinetics of 4-PBA	10 days

Trial Locations

Locations (1)

University of Florida; 🇺🇸 Gainesville, Florida, United States