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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00067756

Enrollment

Registered

2003-08-27

Start date

2001-11-30

Completion date

2003-10-31

Last updated

2014-01-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Alpha 1-Antitrypsin Deficiency

Keywords

Pulmonary Disease, Liver Disease

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.

Interventions

DRUG4-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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 65 Years

Healthy volunteers

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

Design outcomes

Primary

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

Secondary

Measure	Time frame
To determine the pharmacokinetics of 4-PBA	10 days

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026