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

January 2, 2014 updated by: University of Florida

"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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

12

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
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 Names:
  • 4-phenyl butyric acid

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
To determine the pharmacokinetics of 4-PBA
Time Frame: 10 days
10 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Florida

Collaborators

Alpha-1 Foundation
Brantly, Mark L., M.D.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

  • Gonzalez-Peralta RP, Leonard S, Harvey R, Schreck P, Vironosvkaya N, Brantly ML. 4-PHENYL BUTYRATE MEDIATED SECRETION RESCUE IN PATIENTS WITH ALPHA 1-ANTITRYPSIN (AAT) DEFICIENCY: A PILOT STUDY HEPATOLOGY, Vol. 44, No. 4, Suppl. 1, 2006 AASLD ABSTRACTS, p. 211A, 61

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2001

Primary Completion (ACTUAL)

October 1, 2003

Study Completion (ACTUAL)

October 1, 2003

Study Registration Dates

First Submitted

August 26, 2003

First Submitted That Met QC Criteria

August 26, 2003

First Posted (ESTIMATE)

August 27, 2003

Study Record Updates

Last Update Posted (ESTIMATE)

January 3, 2014

Last Update Submitted That Met QC Criteria

January 2, 2014

Last Verified

April 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 87-2001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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