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

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Brief Title

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

Official Title

"4 Phenyl Butyrate Mediated Secretion Rescue in Alpha 1-Antitrypsin Deficient Individuals"

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 Phase

Phase 2

Study Type

Interventional


Primary Outcome

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

Secondary Outcome

 To determine the pharmacokinetics of 4-PBA

Condition

Alpha 1-Antitrypsin Deficiency

Intervention

4-PBA

Study Arms / Comparison Groups

 4-PBA
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

12

Start Date

November 2001

Completion Date

October 2003

Primary Completion Date

October 2003

Eligibility Criteria

        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
      

Gender

All

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Mark L Brantly, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00067756

Organization ID

87-2001


Responsible Party

Sponsor

Study Sponsor

University of Florida

Collaborators

 Alpha-1 Foundation

Study Sponsor

Mark L Brantly, MD, Principal Investigator, University of Florida


Verification Date

April 2013