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Clinical Trials in the USA / NCT01983241
Active, not recruiting Phase 3

Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)

NCT01983241 · tracked via the Priya Life Science USA tracker
Sponsor
Grifols Therapeutics LLC
Phase
Phase 3
Started
2013-11
Last updated
2026-06-23

Condition(s) studied

Pulmonary Emphysema in Alpha-1 PI Deficiency

Investigational drug(s) / intervention(s)

Alpha-1 MP0.9% Sodium Chloride for Injection, USP

Study summary

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
70 Years
Healthy volunteers
No
Inclusion Criteria: * 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. Exclusion Criteria: * 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.

Primary outcome measure(s)

Trial sites (52)

FacilityCityRegionStatus
Grifols Investigative Site Phoenix Arizona
Grifols Investigative Site Gainesville Florida
Grifols Investigative Site Leesburg Florida
Grifols Investigative Site Miami Florida
Grifols Investigative Site St Louis Missouri
Grifols Investigative Site Wilmington North Carolina
Grifols Investigative Site Portland Oregon
Grifols Investigative Site Hershey Pennsylvania
Grifols Investigative Site Charleston South Carolina
Grifols Investigative Site Tyler Texas
Grifols Investigative Site Vicente López Buenos Aires
Grifols Investigative Site Córdoba Córdoba Province
Grifols Investigative Site Sydney New South Wales
Grifols Investigative Site Chermside Queensland
Grifols Investigative Site Adelaide South Australia
Grifols Investigative Site Fitzroy Victoria
Grifols Investigative Site Nedlands Western Australia
Grifols Investigative Site Santo André São Paulo
Grifols Investigative Site São Paulo São Paulo
Grifols Investigative Site São Paulo São Paulo
Grifols Investigative Site St. John's Newfoundland and Labrador
Grifols Investigative Site Halifax Nova Scotia
Grifols Investigative Site Toronto Ontario
Grifols Investigative Site Arhus C Denmark
Grifols Investigative Site Hellerup Denmark
Grifols Investigative Site Tallinn Estonia
Grifols Investigative Site Tartu Estonia
Grifols Investigative Site Turku Finland
Grifols Investigative Site Pessac Gironde
Grifols Investigative Site Bron Rhone
Grifols Investigative Site Heidelberg Baden-Wurttemberg
Grifols Investigative Site Marburg Hesse
Grifols Investigative Site Essen North Rhine-Westphalia
Grifols Investigative Site Homburg Saar Germany
Grifols Investigative Site Chisinau Moldova
Grifols Investigative Site Auckland New Zealand
Grifols Investigative Site Christchurch New Zealand
Grifols Investigative Site Hamilton New Zealand
Grifols Investigative Site Krakow Poland
Grifols Investigative Site Warsaw Poland

+ 12 more sites — see the full list on the official registry below.

More Grifols Therapeutics LLC trials in the USA

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT01983241 on ClinicalTrials.gov ↗ ← All trials in the USA