drugset
drugset / Trial / NCT00067756

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

NCT00067756 ↗

Phase 2 Completed 12 enrolled University of Florida Alpha-1 Foundation · collabBrantly, Mark L., M.D. · collab
NaSingle-groupOpen-labelTreatment

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.

Timeline

Start
2001-11
Primary completion
2003-10
Completion
2003-10

Publications

  • Results 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

Drugs

EvaluationDrugModalityDoseRoute
Subject Sodium Phenylbutyrate Small molecule 40 g Oral

Indications

alpha 1-antitrypsin deficiency