Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Grifols Therapeutics LLC
Age
18–70
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Have a documented total alpha1-PI serum level \< 11 µM.
Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles.
At the Screening (Week -3) Visit, have a post-bronchodilator forced expiratory volume in 1 second (FEV1) ≥ 30% and \< 80% of predicted and FEV1/forced vital capacity (FVC) \< 70% (Global Initiative for Chronic Obstructive Lung Disease \[GOLD\] stage II or III).
Have a carbon monoxide diffusing capacity (DLCO) ≤ 60% of predicted (corrected for HgB) within the past 2 years OR evidence of pulmonary emphysema on CT scan within the past 2 years per the Investigator's judgment.
Have clinical evidence of pulmonary emphysema per the Investigator's judgment.
You may not be if
Has received alpha1-PI augmentation therapy for more than 1 month within the six months prior to the Screening Visit.
Has received alpha1-PI augmentation therapy within one month of the Screening Visit.
Has had a chronic obstructive pulmonary disease (COPD) exacerbation within the 5 weeks prior to the Screening Visit or during the Screening Phase.
Unable to physically (e.g., unable to fit inside the CT scanner) or mentally (e.g., claustrophobic) undergo a CT scan.
History of lung or liver transplant.
Any lung surgery during the past 2 years (excluding lung biopsy).
On the waiting list for lung surgery, including lung transplant.
Smoking during the past 12 months or a positive urine cotinine test at screening that is due to smoking. Maybe on Nicotine replacement, including vapor cigarettes.
History of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s).
Use of systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e., 10 mg every 2 days) within the 5 weeks prior to the Screening Visit (inhaled steroids are not considered systemic steroids) or during the Screening Phase.
Use of systemic or aerosolized antibiotics for a COPD exacerbation within the 5 weeks prior to the Screening Visit or during the Screening Phase.
Known selective or severe Immunoglobulin A (IgA) deficiency.
Clareo Health | Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)
