Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.
Status:
Completed
Trial end date:
2016-05-01
Target enrollment:
Participant gender:
Summary
The current treatment of individuals with alpha-1 antitrypsin deficiency (AATD) who develop
lung disease (COPD) is the administration of intravenous purified alpha-1 antitrypsin
(augmentation therapy) at a fixed dose of 60 mg/kg per week. This dose aims at increasing the
deficient AAT serum levels just above a predetermined "safety threshold" of 11 uM. However,
normal levels of AAT are between 25-50 uM.
AAT has shown not only to inhibit lung proteases such as neutrophil elastase, but also to
modulate inflammation. Given that many subjects with AATD who receive augmentation therapy
still have significant lung disease and inflammation, this study will evaluate whether
doubling the dose to 120 mg/kg/week has an effect in decreasing lung inflammation.
Only the dosing of 60 mg/kg /week has received FDA approval. FDA has granted an IND number to
this study to test the higher dose of 120 mg/kg/week.
The study will evaluate systemic (serum) and pulmonary (bronchoscopy samples)markers of
inflammation in 3 phases: standard dose (4 weeks), double dose (4 weeks) and standard dose (4
weeks).
Phase:
Phase 2
Details
Lead Sponsor:
Michael Campos, MD
Collaborator:
CSL Behring
Treatments:
Alpha 1-Antitrypsin Protease Inhibitors Protein C Inhibitor