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

Information source: University of Florida
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Alpha 1-Antitrypsin Deficiency

Intervention: 4-PBA (Drug)

Phase: Phase 2

Status: Completed

Sponsored by: University of Florida

Official(s) and/or principal investigator(s):
Mark L Brantly, MD, Principal Investigator, Affiliation: University of Florida

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.

Clinical Details

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

Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

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

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.

Eligibility

Minimum age: 18 Years. Maximum age: 65 Years. Gender(s): Both.

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

Locations and Contacts

University of Florida, Gainesville, Florida 32610, United States
Additional Information

Alpha-1 Foundation Alpha-1 Research Program

Starting date: November 2001
Last updated: January 2, 2014

Page last updated: August 23, 2015

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