Diagnosis of emphysema confirmed by CT scan. If a report of past CT scan is not available at site documenting then a CT scan is to be performed at screening 
Male or female patients at least 18 years of age. 
Able and willing to sign an informed consent. 
Patient with record of congenital AAT deficiency of phenotype PiZZ (homozygote) or other rare phenotypes related to AAT deficiency and with AAT serum level ≤ 11 micromole. For patients receiving IV AAT augmentation therapy the serum AAT level threshold does not apply. 
FEV1/SVC <70% of predicted value post bronchodilator (SVC is slow VC) and FEV1 < 80% of predicted value post-bronchodilator 
History of at least two moderate or severe exacerbations that required change in treatment (antibiotics, systemic steroids, hospitalization) in the last 18 months prior to date of screening , with at least one of these occurring within the last 12 months prior to screening. 
Ability to comply with completion of electronic diary. 
Ability to self-administer inhaled AAT. 
No significant abnormalities in serum hematology, serum chemistry and serum inflammatory / immunogenic markers according to the Principal Investigator's judgment, taking into considerations the potential effects of the AAT deficiency. 
No significant abnormalities in urinalysis according to the Principal Investigator's judgment, taking into considerations the potential effects of the AAT deficiency. 
No significant abnormalities in ECG per investigator judgment. 
Negative for HBsAg and for antibodies to HCV, HIV-1. 
AAT deficient patients who are either naïve (not receiving IV augmentation therapy) or AAT deficient patients (receiving IV augmentation therapy), if they have been stable on regular therapy for at least 3 months prior to the screening visit and are willing to continue the same regime throughout this trial. Note that only sites in Germany can recruit patients who are currently being treated with IV AAT.Patients who stopped IV augmentation treatment 6 months prior to screening date and will not re-start this treatment for the course of the study will be considered Naïve. 
Non-pregnant, non-lactating female patients, whose screening pregnancy test is negative and who are using contraceptive methods deemed reliable by the investigator, or who are at least 2 years post-menopausal or surgically sterilized. 

NCT01217671_inc_T1
Condition
emphysema
NCT01217671_inc_T2
Procedure
CT scan
NCT01217671_inc_T3
Procedure
CT scan
NCT01217671_inc_T4
Temporal
past
NCT01217671_inc_T6
Negation
not available
NCT01217671_inc_T7
Observation
report of past CT scan
NCT01217671_inc_T8
Procedure
CT scan
NCT01217671_inc_T9
Scope
report of past CT scan is not available
NCT01217671_inc_T5
Scope
report of past CT scan is not available at site documenting then a CT scan is to be performed at screening
NCT01217671_inc_T10
Temporal
at screening
NCT01217671_inc_T11
Reference_point
screening
NCT01217671_inc_T12
Person
Male
NCT01217671_inc_T13
Person
female
NCT01217671_inc_T14
Value
at least 18 years
NCT01217671_inc_T15
Person
age
NCT01217671_inc_T17
Condition
congenital AAT deficiency of phenotype PiZZ (homozygote)
NCT01217671_inc_T18
Condition
rare phenotypes related to AAT deficiency
NCT01217671_inc_T19
Measurement
AAT serum level
NCT01217671_inc_T20
Value
≤ 11 micromole
NCT01217671_inc_T21
Procedure
IV AAT augmentation therapy
NCT01217671_inc_T23
Measurement
FEV1/SVC
NCT01217671_inc_T24
Value
<70% of predicted value
NCT01217671_inc_T25
Qualifier
post bronchodilator
NCT01217671_inc_T26
Temporal
post bronchodilator
NCT01217671_inc_T27
Drug
bronchodilator
NCT01217671_inc_T28
Measurement
FEV1
NCT01217671_inc_T29
Value
< 80% of predicted value
NCT01217671_inc_T30
Qualifier
post-bronchodilator
NCT01217671_inc_T31
Temporal
post-bronchodilator
NCT01217671_inc_T32
Drug
bronchodilator
NCT01217671_inc_T33
Reference_point
bronchodilator
NCT01217671_inc_T34
Reference_point
bronchodilator
NCT01217671_inc_T35
Multiplier
at least two
NCT01217671_inc_T36
Qualifier
moderate
NCT01217671_inc_T37
Qualifier
severe
NCT01217671_inc_T38
Condition
exacerbations
NCT01217671_inc_T39
Scope
moderate or severe
NCT01217671_inc_T40
Observation
change in treatment
NCT01217671_inc_T41
Qualifier
required change in treatment
NCT01217671_inc_T42
Drug
antibiotics
NCT01217671_inc_T43
Drug
systemic steroids
NCT01217671_inc_T44
Procedure
hospitalization
NCT01217671_inc_T45
Qualifier
systemic
NCT01217671_inc_T46
Temporal
in the last 18 months prior to date of screening
NCT01217671_inc_T47
Reference_point
date of screening
NCT01217671_inc_T48
Multiplier
at least one
NCT01217671_inc_T49
Temporal
within the last 12 months prior to screening
NCT01217671_inc_T50
Reference_point
screening
NCT01217671_inc_T51
Scope
antibiotics, systemic steroids, hospitalization
NCT01217671_inc_T52
Procedure
treatment
NCT01217671_inc_T53
Observation
self-administer inhaled AAT
NCT01217671_inc_T54
Observation
comply with completion of electronic diary
NCT01217671_inc_T61
Procedure
ECG
NCT01217671_inc_T62
Condition
abnormalities in ECG
NCT01217671_inc_T63
Qualifier
significant
NCT01217671_inc_T64
Negation
No
NCT01217671_inc_T66
Measurement
HBsAg
NCT01217671_inc_T67
Value
Negative
NCT01217671_inc_T68
Measurement
antibodies to HCV
NCT01217671_inc_T69
Measurement
HIV-1
NCT01217671_inc_T70
Condition
AAT deficient
NCT01217671_inc_T71
Condition
naïve
NCT01217671_inc_T72
Procedure
IV augmentation therapy
NCT01217671_inc_T73
Negation
not receiving
NCT01217671_inc_T75
Procedure
IV augmentation therapy
NCT01217671_inc_T74
Procedure
therapy
NCT01217671_inc_T76
Temporal
for at least 3 months prior to the screening
NCT01217671_inc_T77
Reference_point
the screening
NCT01217671_inc_T78
Observation
willing to continue
NCT01217671_inc_T79
Temporal
throughout this trial
NCT01217671_inc_T80
Reference_point
this trial
NCT01217671_inc_T81
Qualifier
stable
NCT01217671_inc_T82
Condition
pregnant
NCT01217671_inc_T83
Negation
Non
NCT01217671_inc_T84
Condition
lactating
NCT01217671_inc_T85
Negation
non
NCT01217671_inc_T86
Person
female
NCT01217671_inc_T87
Measurement
pregnancy test
NCT01217671_inc_T88
Value
negative
NCT01217671_inc_T89
Device
contraceptive methods
NCT01217671_inc_T91
Temporal
at least 2 years
NCT01217671_inc_T92
Condition
post-menopausal
NCT01217671_inc_T93
Condition
surgically sterilized
NCT01217671_inc_T94
Procedure
surgically
NCT01217671_inc_T95
Qualifier
deemed reliable by the investigator
NCT01217671_inc_T96
Scope
post-menopausal or surgically sterilized
NCT01217671_inc_T97
Scope
Non-pregnant, non-lactating female
NCT01217671_inc_T98
Scope
pregnancy test is negative and who are using contraceptive methods deemed reliable by the investigator, or who are at least 2 years post-menopausal or surgically sterilized