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

University of Florida12 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Alpha 1-Antitrypsin Deficiency

Interventions

4-PBADRUG

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

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (1)

University of Florida

Gainesville, Florida, United States

Outcomes

Primary Outcomes

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

Time frame: 10 days

Secondary Outcomes

To determine the pharmacokinetics of 4-PBA

Time frame: 10 days

Data from ClinicalTrials.gov

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