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ACTIVE NOT RECRUITING

NCT01983241

PHASE3

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

Sponsor: Grifols Therapeutics LLC

View on ClinicalTrials.gov

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.

Official title: A Randomized, Double-Blind, Placebo Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alpha1 Proteinase Inhibitor (Human) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency

Key Details

Gender

All

Age Range

18 Years - 70 Years

Study Type

INTERVENTIONAL

Enrollment

345

Start Date

2013-11

Completion Date

2027-01

Last Updated

2026-03-04

Healthy Volunteers

Conditions

Pulmonary Emphysema in Alpha-1 PI Deficiency

Interventions

BIOLOGICAL

Alpha-1 MP

OTHER

0.9% Sodium Chloride for Injection, USP

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.

Locations (52)

Grifols Investigative Site

Phoenix, Arizona, United States

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

Tallinn, Estonia

Grifols Investigative Site

Tartu, Estonia

Grifols Investigative Site

Turku, Finland

Grifols Investigative Site

Pessac, Gironde, France

Grifols Investigative Site

Bron, Rhone, France

Grifols Investigative Site

Heidelberg, Baden-Wurttemberg, Germany

Grifols Investigative Site

Marburg, Hesse, Germany

Grifols Investigative Site

Essen, North Rhine-Westphalia, Germany

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

Grifols Investigative Site

Oradea, Romania

Grifols Investigative Site

Timișoara, Romania

Grifols Investigative Site

Barnaul, Russia

Grifols Investigative Site

Moscow, Russia

Grifols Investigative Site

Yaroslavl, Russia

Grifols Investigative Site

Oviedo, Principality of Asturias, Spain

Grifols Investigative Site

Barakaldo, Vizcaya, Spain

Grifols Investigative Site

Gothenburg, Sweden

Grifols Investigative Site

Linköping, Sweden

Grifols Investigative Site

Malmo, Sweden

Grifols Investigative Site

Stockholm, Sweden

Grifols Investigative Site

Uppsala, Sweden

Clinical Research Directory

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

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (52)