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

Tracking Information

First Received Date ^ICMJE

August 26, 2003

Last Updated Date

February 17, 2009

Start Date ^ICMJE

November 2001

Primary Completion Date

October 2003 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease. [ Time Frame: 10 days ] [ Designated as safety issue: Yes ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT00067756 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

To determine the pharmacokinetics of 4-PBA [ Time Frame: 10 days ] [ Designated as safety issue: Yes ]

Original Secondary Outcome Measures ^ICMJE

Same as current

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

"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 II

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment, Non-Randomized, Open Label, Active Control, Single Group Assignment, Safety/Efficacy Study

Condition ^ICMJE

Alpha 1-Antitrypsin Deficiency

Intervention ^ICMJE

Drug: 4 Phenyl Butyrate (4PBA)

Study Arms / Comparison Groups

Experimental: The study will involve a dose escalation and pharmacokinetics component The study group will be comprised of a total of 10 patients equally divided into PiZZ* AAT-deficient individuals with (n=5) and without (n=5) 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 ^ICMJE

Completed

Enrollment ^ICMJE

Completion Date

October 2003

Primary Completion Date

October 2003 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Both

Ages

18 Years to 65 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States

Administrative Information

NCT ID ^ICMJE

NCT00067756

Responsible Party

Mark L. Brantly, MD, University of Florida

Study ID Numbers ^ICMJE

87-2001

Study Sponsor ^ICMJE

University of Florida

Collaborators ^ICMJE

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

Investigators ^ICMJE

Principal Investigator:

Mark L Brantly, MD

University of Florida

Information Provided By

University of Florida

Verification Date

February 2009

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP