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Active (not recruiting) Phase 3 NCT01983241

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

Phase 3 – large-scale trial before approval
Conditions: Pulmonary Emphysema in Alpha-1 PI Deficiency

Sponsor: Grifols Therapeutics LLC

trial.available_in: БГ
Overview
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.
Who can participate
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 16
Argentina (1)
Grifols Investigative Site
Vicente López , Buenos Aires
Australia (1)
Grifols Investigative Site
Sydney , New South Wales
Brazil (1)
Grifols Investigative Site
Santo André , São Paulo
Canada (1)
Grifols Investigative Site
St. John's , Newfoundland and Labrador
Denmark (1)
Grifols Investigative Site
Arhus C
Estonia (1)
Grifols Investigative Site
Tallinn
Finland (1)
Grifols Investigative Site
Turku
France (1)
Grifols Investigative Site
Pessac , Gironde
Moldova (1)
Grifols Investigative Site
Chisinau
New Zealand (1)
Grifols Investigative Site
Auckland
Poland (1)
Grifols Investigative Site
Krakow
Romania (1)
Grifols Investigative Site
Oradea
Russia (1)
Grifols Investigative Site
Barnaul
Spain (1)
Grifols Investigative Site
Oviedo , Principality of Asturias
Sweden (1)
Grifols Investigative Site
Gothenburg
United States (1)
Grifols Investigative Site
Phoenix , Arizona
Technical details
Status
Active (not recruiting)
Phase
Phase 3
Study type
INTERVENTIONAL
Sex
Male and female
Minimum age
18 Years
Maximum age
70 Years
Healthy volunteers
No
Start date
01.11.2013
Completion date
01.01.2027
Registry ID
NCT01983241
Source
anzctr
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