Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)
NCT ID: NCT01983241
Last Updated: 2025-01-15
Study Results
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Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
345 participants
INTERVENTIONAL
2013-11-30
2027-01-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Alpha-1 MP 60 mg/kg
Alpha-1 MP 60 mg/kg administered weekly by IV infusion for 156 weeks
Alpha-1 MP
Alpha-1 MP 120 mg/kg
Alpha-1 MP 120 mg/kg administered weekly by IV infusion for 156 weeks
Alpha-1 MP
Placebo
0.9% Sodium Chloride for Injection, USP, administered weekly by IV infusion for 156 weeks
0.9% Sodium Chloride for Injection, USP
Interventions
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Alpha-1 MP
0.9% Sodium Chloride for Injection, USP
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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.
18 Years
70 Years
ALL
No
Sponsors
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Grifols Therapeutics LLC
INDUSTRY
Responsible Party
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Locations
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St. Joseph's Hospital and Medical Center
Phoenix, Arizona, United States
University of Florida
Gainesville, Florida, United States
Medical Research of Central Florida, LLC
Leesburg, Florida, United States
University of Miami Miller School of Medicine
Miami, Florida, United States
Washington University School of Medicine
St Louis, Missouri, United States
Accellacare
Wilmington, North Carolina, United States
Oregon Health & Science University
Portland, Oregon, United States
Penn State Univ. Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
University of Texas Health Center at Tyler
Tyler, Texas, United States
Hospital del Torax Dr. Antonio A. Cetrangolo
Vicente López, Buenos Aires, Argentina
Centro Dr. Lazaro Langer
Córdoba, , Argentina
St Vincent's Hospital Sydney
Sydney, New South Wales, Australia
The Prince Charles Hospital
Chermside, Queensland, Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia
St Vincent's Hospital Melbourne
Fitzroy, Victoria, Australia
Institute for Respiratory Health Inc
Nedlands, Western Australia, Australia
Faculdade de Medicina do ABC
Santo André, São Paulo, Brazil
UNIFESP - Universidade Federal de São Paulo
São Paulo, São Paulo, Brazil
Instituto do Coração - Incor- HCFMUSP
São Paulo, , Brazil
Health Sciences Centre
St. John's, Newfoundland and Labrador, Canada
Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, Canada
Inspiration Research Limited
Toronto, Ontario, Canada
Århus Universitetshospital
Arhus C, , Denmark
Gentofte Hospital
Hellerup, , Denmark
North Estonia Medical Centre Foundation
Tallinn, , Estonia
Tartu University Hospital
Tartu, , Estonia
Turku University Central Hospital, Department of Pulmonary Diseases
Turku, , Finland
Groupe Hospitalier Sud - Hôpital Haut-Lévêque
Pessac, Gironde, France
Hôpital Cardio-Vasculaire et Pneumologique Louis Pradel
Bron, Rhone, France
Universitaetsklinikum Heidelberg
Heidelberg, Baden-Wurttemberg, Germany
Universitaetsklinikum Giessen und Marburg GmbH
Marburg, Hesse, Germany
Ruhrlandklinik
Essen, North Rhine-Westphalia, Germany
Univer des Saarlandes Innere
Homburg Saar, , Germany
Institutul de Ftiziopneumologie "Chiril Draganiuc"
Chisinau, , Moldova
New Zealand Respiratory and Sleep institute
Auckland, , New Zealand
Christchurch Hospital NZ
Christchurch, , New Zealand
Waikato Hospital
Hamilton, , New Zealand
SPZOZ Szpital Uniwersytecki w Krakowie
Krakow, , Poland
Instytut Gruzlicy i Chorob Pluc w Warszawie
Warsaw, , Poland
Clinic Dr.G Curteanu Oradea
Oradea, , Romania
Spitalul Clinic de Boli Infectioase si Pneumoftiziologie
Timișoara, , Romania
SBEI HPE Altai State Medical University of MoH and SD
Barnaul, , Russia
FSI Scientific Research Institute of Pulmonology
Moscow, , Russia
SAIH of Yaroslavl region "Clinical Hospital of Emergency Medical Care n.a. N. V. Solovyev
Yaroslavl, , Russia
Hospital Universitario Central de Asturias
Oviedo, Principality of Asturias, Spain
Hospital de Cruces
Barakaldo, Vizcaya, Spain
Sahlgrenska Sjukhuset
Gothenburg, , Sweden
CTC - Clinical Trial Consultants AB
Linköping, , Sweden
Skånes Universitetssjukhus, Malmö
Malmo, , Sweden
Karolinska Universitetssjukhuset, Solna
Stockholm, , Sweden
CTC Clinical Trial Consultants AB
Uppsala, , Sweden
Countries
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References
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Sorrells S, Camprubi S, Griffin R, Chen J, Ayguasanosa J. SPARTA clinical trial design: exploring the efficacy and safety of two dose regimens of alpha1-proteinase inhibitor augmentation therapy in alpha1-antitrypsin deficiency. Respir Med. 2015 Apr;109(4):490-9. doi: 10.1016/j.rmed.2015.01.022. Epub 2015 Feb 13.
Other Identifiers
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GTi1201
Identifier Type: -
Identifier Source: org_study_id
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