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

University of Florida

Status and phase: Completed; Phase 2

Conditions: Alpha 1-Antitrypsin Deficiency

Treatments: Drug: 4-PBA

Study type: Interventional

Funder types: Other

Identifiers

NCT00067756

87-2001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 12 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

4-PBA

Experimental group

Description:

The 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.

Treatment:

Drug: 4-PBA