Table 1 Comparison of study characteristics
Danish-Dutch trial | EXACTLE trial | |
|---|---|---|
Genotype/phenotype | PiZZ on IEF | PiZZ or severe deficiency with AAT concentrations <11 μM |
Lung function, FEV1 | 30-80% | 25-80% and FEV1/VC ≤70% or |
Kco | NA | ≤80% if spirometry normal |
Exacerbations | NA | ≥1 exacerbation in the past 2 years |
Smoking history | Never or ex-smokers for >6 months Cotinine checked every 4 weeks | Never or ex-smokers for >6 months Cotinine checked at 1, 6, 24 and 30 months |
Previous augmentation therapy | NA | Never or ≤1 month in past 2 years |
Study design | Randomised, double-blind, placebo-controlled | Randomised, double-blind, placebo-controlled |
AAT dosing | 250 mg•kg-1 body weight AAT | 60 mg•kg-1 body weight AAT |
Treatment interval | Every 4 weeks | Every week |
Placebo | 625 mg•kg-1 body weight albumin | 2% albumin |
Centres | 2 (Copenhagen, Leiden) | 3 (Copenhagen, Birmingham, Malmö) |
Duration of study | Minimum 3 years | 24 months (optional 6-months extension) |
Study period | January 1991 to August 1997 | November 2003 to December 2006 |
Primary endpoints | FEV1 measured by home spirometry twice daily | Change in PD15 measured by CT |
Other endpoints | Change in PD15 measured by CT | Exacerbations Lung function (FEV1, KCO) Quality of life (SGRQ) |