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

Alpha 1-Antitrypsin Deficiency Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00067756
• Other study ID #: 87-2001
• Status: Completed
• Phase: Phase 2
• First received: August 26, 2003
• Last updated: January 2, 2014
• Start date: November 2001
• Est. completion date: October 2003

Study information

• Verified date: April 2013
• Source: University of Florida
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: October 2003
• Est. primary completion date: October 2003
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

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

Study Design

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

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency

Intervention

Drug:
• 4-PBA
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

Locations

Country	Name	City	State
United States	University of Florida	Gainesville	Florida

Sponsors (3)

Lead Sponsor	Collaborator
University of Florida	Alpha-1 Foundation, Brantly, Mark L., M.D.

Country where clinical trial is conducted

United States, 

References & 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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease.		10 days	Yes
Secondary	To determine the pharmacokinetics of 4-PBA		10 days	Yes

External Links

•   Alpha-1 Foundation Alpha-1 Research Program