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
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