Abstract
Introduction: Alpha1-Antitrypsin Deficiency (AATD) is a hereditary condition characterized by low serum levels of alpha1-proteinase inhibitor (alpha1-PI) and an increased risk of emphysema. The approved dose for augmentation therapy is 60 mg/kg/wk, associated with raising trough levels to approximately 11 µM. However, the normal lower limit in the nondeficient population is 20 µM. To date, no prospective randomized controlled trial has conclusively proven the clinical efficacy of augmentation therapy.
Goal: To assess the effect of augmentation therapy on emphysema progression in AATD assessed by lung computed tomography (CT) densitometry, dosed to approximate trough levels of 11 µM or 20 µM, vs placebo. SPARTA will be the largest randomized trial with the longest duration of exposure, and the first to compare two active augmentation arms.
Design: Prospective, double-blind, randomized, placebo-controlled
Discussion: SPARTA will be the first prospective, randomized controlled trial of augmentation to assess two active doses, including one designed to achieve population based normal trough levels, vs placebo.Enrollment is anticipated to commence in September 2013 and should allow analyses of a number of secondary endpoints and subject subpopulations.
- © 2013 ERS
