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Completed

NCT Number: NCT00067756

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

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.

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Key information

Conditions

Alpha 1-Antitrypsin Deficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Emphysema Genetic Diseases, Inborn Liver Diseases Lung Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Respiratory Tract Diseases Subcutaneous Emphysema

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of Florida

Gainesville, Florida, 32610, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

4-PBA

Drug

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

Other names: 4-phenyl butyric acid

Primary outcomes

  1. To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease.

    Time frame: 10 days

Secondary outcomes

  1. To determine the pharmacokinetics of 4-PBA

    Time frame: 10 days

Sponsors and collaborators

Lead sponsor

University of Florida

Other

Collaborators

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

Registry information

Official study title

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

Important dates

Study start
2001
Primary completion
2003
Study completion
2003
First posted
Aug 27, 2003
Registry last updated
Jan 3, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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