Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)
Running, not enrolling · Phase 3 · Has a placebo group
Conditions studied: Pulmonary Emphysema in Alpha-1 PI Deficiency
In brief
This is a multi-center, randomized, placebo-controlled, double blind clinical study to assess the efficacy and safety of two separate dose regimens of Alpha-1 MP versus placebo for 156 weeks (i.e., 3 years) using computed tomography (CT) of the lungs as the main measure of efficacy. The two Alpha-1 MP doses to be tested are 60 mg/kg and 120 mg/kg administered weekly by IV infusion for 156 weeks. The study consists of an optional pre-screening phase, Screening Phase, a 156-week Treatment Phase, and an End of Study Visit at Week 160.
Key facts
- Study ID
- NCT01983241
- Run by
- Grifols Therapeutics LLC
- People needed
- 345
- Starts
- 2013-11-01
- Expected to finish
- 2027-01-01
- Last updated by the study team
- 2026-06-23
Who can join
Age: 18 and older, up to 70. Sex: any. Healthy volunteers: not accepted.
You may qualify 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 qualify 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.
Where it is running
- Grifols Investigative Site — Gainesville, Florida, United States
- Grifols Investigative Site — Leesburg, Florida, United States
- Grifols Investigative Site — Miami, Florida, United States
- Grifols Investigative Site — St Louis, Missouri, United States
- Grifols Investigative Site — Wilmington, North Carolina, United States
- Grifols Investigative Site — Portland, Oregon, United States
- Grifols Investigative Site — Hershey, Pennsylvania, United States
- Grifols Investigative Site — Charleston, South Carolina, United States
- Grifols Investigative Site — Tyler, Texas, United States
- Grifols Investigative Site — Vicente López, Buenos Aires, Argentina
- Grifols Investigative Site — Córdoba, Córdoba Province, Argentina
- Grifols Investigative Site — Sydney, New South Wales, Australia
- Grifols Investigative Site — Chermside, Queensland, Australia
- Grifols Investigative Site — Adelaide, South Australia, Australia
- Grifols Investigative Site — Fitzroy, Victoria, Australia
- Grifols Investigative Site — Nedlands, Western Australia, Australia
- Grifols Investigative Site — Santo André, São Paulo, Brazil
- Grifols Investigative Site — São Paulo, São Paulo, Brazil
- Grifols Investigative Site — São Paulo, São Paulo, Brazil
- Grifols Investigative Site — St. John's, Newfoundland and Labrador, Canada
- Grifols Investigative Site — Halifax, Nova Scotia, Canada
- Grifols Investigative Site — Toronto, Ontario, Canada
- Grifols Investigative Site — Arhus C, Denmark
- Grifols Investigative Site — Hellerup, Denmark
- Grifols Investigative Site — Phoenix, Arizona, United States
Full record on ClinicalTrials.gov
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