Alpha-1 Antitrypsin Augmentation Therapy
Treatment for Emphysema
Typical Dosage: 60 mg/kg body weight intravenously once weekly
Effectiveness
55%
Safety Score
70%
Clinical Trials
12
Participants
5K
Comparative Safety Scale(Higher is safer)
Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
70
DangerousModerateSafe
Treatment Details
Dosage Range
60 mg/kg body weight intravenously once weekly
Time to Effect
immediate maintenance of AAT levels, years for clinical benefit
Treatment Duration
lifetime
Evidence Quality
MODERATENumber Needed to Treat (NNT)
8(Treat 8 patients to see 1 additional successful outcome)
Number Needed to Harm (NNH)
75(Treat 75 patients to see 1 additional serious adverse event)
Confidence Score
80%confidence in effectiveness data
Health Economics
Annual Cost of Care
Drug Cost:$100,000
Monitoring:$2,000
Side Effect Mgmt:$500
Total Annual:$102,500
Cost-Effectiveness Analysis
Cost-Effectiveness Rating
POORICER
$500,000/QALY
QALYs Gained
0.1
Outcome-Based Costs
Cost per Responder
$227,778
Alpha-1 Antitrypsin Augmentation Therapy Outcomes
for Emphysema
Efficacy Outcomes
Overall Effectiveness
+55%
Response Rate
+45%
Common Side Effects
Headache
+15%
Dizziness
+7%
Nausea
+7%
Infusion reactions (skin rash, chills, fever)
+3%
Active Clinical Trials
1 active trial recruiting for Alpha-1 Antitrypsin Augmentation Therapy in Emphysema
Gene Therapy for Alpha-1 Antitrypsin Deficiency
NCT06996756RECRUITINGPHASE1
16 participants
INTERVENTIONAL
New York, United States
Started: Feb 26, 2025
Completed Clinical Trials
8 completed trials for Alpha-1 Antitrypsin Augmentation Therapy in Emphysema
Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.
NCT01669421COMPLETEDPHASE2
10 participants
INTERVENTIONAL
Miami, United States
Started: Jul 1, 2012
Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema
NCT05856331COMPLETEDPHASE2
99 participants
INTERVENTIONAL
Birmingham, United States +64 more
Started: Oct 12, 2023
Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency
NCT00261833COMPLETEDPHASE4
180 participants
INTERVENTIONAL
Denver, United States +27 more
Started: Mar 1, 2006
Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.
NCT00670007COMPLETEDPHASE4
140 participants
INTERVENTIONAL
Adelaide, Australia +20 more
Started: Apr 1, 2008
Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor)
NCT00396006COMPLETEDPHASE4
21 participants
INTERVENTIONAL
Adelaide, Australia +4 more
Started: Oct 27, 2006
Genomic Research in Alpha-1 Antitrypsin Deficiency
NCT01832220COMPLETED
140 participants
OBSERVATIONAL
Tucson, United States +8 more
Started: May 1, 2013
Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency
NCT02691611COMPLETED
39 participants
OBSERVATIONAL
Denver, United States
Started: Dec 1, 2015
Microbioma in Sputa From COPD With Alpha-1 Antitrypsin Deficiency
NCT02547532COMPLETED
100 participants
OBSERVATIONAL
Started: Sep 1, 2015