4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?
"4 Phenyl Butyrate Mediated Secretion Rescue in Alpha 1-Antitrypsin Deficient Individuals"
1 other identifier
interventional
12
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Nov 2001
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 1, 2001
CompletedFirst Submitted
Initial submission to the registry
August 26, 2003
CompletedFirst Posted
Study publicly available on registry
August 27, 2003
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2003
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2003
CompletedJanuary 3, 2014
April 1, 2013
1.9 years
August 26, 2003
January 2, 2014
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease.
10 days
Secondary Outcomes (1)
To determine the pharmacokinetics of 4-PBA
10 days
Study Arms (1)
4-PBA
EXPERIMENTALThe 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.
Interventions
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
Eligibility Criteria
You may qualify if:
- Age 18-65
- Serum A1-PI levels \<11uM an appropriate genetic phenotype/genotype
- 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)
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- University of Floridalead
- Alpha-1 Foundationcollaborator
- Brantly, Mark L., M.D.collaborator
Study Sites (1)
University of Florida
Gainesville, Florida, 32610, United States
Related Publications (1)
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
RESULT
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Mark L Brantly, MD
University of Florida
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 26, 2003
First Posted
August 27, 2003
Study Start
November 1, 2001
Primary Completion
October 1, 2003
Study Completion
October 1, 2003
Last Updated
January 3, 2014
Record last verified: 2013-04