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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01983241
Recruitment Status : Recruiting
First Posted : November 13, 2013
Last Update Posted : August 27, 2019
Information provided by (Responsible Party):
Grifols Therapeutics LLC

Brief 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 a Screening Phase, a 156-week Treatment Phase, and an End of Study Visit at Week 160.

Condition or disease Intervention/treatment Phase
Pulmonary Emphysema in Alpha-1 PI Deficiency Biological: Alpha-1 MP Other: 0.9% Sodium Chloride for Injection, USP Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 339 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Study Start Date : November 2013
Estimated Primary Completion Date : November 2023
Estimated Study Completion Date : November 2023

Arm Intervention/treatment
Experimental: Alpha-1 MP 60 mg/kg
Alpha-1 MP 60 mg/kg administered weekly by IV infusion for 156 weeks
Biological: Alpha-1 MP
Other Name: Prolastin-C

Experimental: Alpha-1 MP 120 mg/kg
Alpha-1 MP 120 mg/kg administered weekly by IV infusion for 156 weeks
Biological: Alpha-1 MP
Other Name: Prolastin-C

Placebo Comparator: Placebo
0.9% Sodium Chloride for Injection, USP, administered weekly by IV infusion for 156 weeks
Other: 0.9% Sodium Chloride for Injection, USP
Other Name: Saline

Primary Outcome Measures :
  1. Change from Baseline in Whole lung PD15 (15th percentile point) [ Time Frame: Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156 ]
    Whole lung PD15 measured by CT scan

Secondary Outcome Measures :
  1. Adverse Events (AEs) [ Time Frame: Week -3 through Week 160 ]
    Monitoring of AEs

  2. Serious Adverse Events (SAEs) [ Time Frame: Week -3 through Week 160 ]
    Monitoring of SAEs

  3. Discontinuations from the study due to AEs [ Time Frame: Week -3 through Week 160 ]
    Monitoring of discontinuations due to AEs

  4. Severe COPD Exacerbations [ Time Frame: Week -3 through Week 160 ]
    Severe COPD exacerbations as defined by American Thoracic Society/European Respiratory Society (ATS/ERS) criteria (i.e., COPD exacerbations requiring hospitalization)

  5. Change from Baseline in PD15 of the basal lung region [ Time Frame: Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156 ]
    PD15 of the basal lung region measure by CT scan

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01983241

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Contact: Susan Sorrells

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United States, Arizona
St. Joseph's Hospital and Medical Center Recruiting
Phoenix, Arizona, United States, 85013
Principal Investigator: Rajat Walia, MD         
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Principal Investigator: Mark Brantly, MD         
University of Miami Miller School of Medicine Recruiting
Miami, Florida, United States, 33136
Principal Investigator: Michael Campos, MD         
United States, Missouri
Washington University School of Medicine Withdrawn
Saint Louis, Missouri, United States, 63110
United States, North Carolina
PMG Research Recruiting
Wilmington, North Carolina, United States, 28401
Principal Investigator: Mitchell D Lee, MD         
United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239
Principal Investigator: Alan Barker, MD         
United States, Pennsylvania
Penn State Univ. Milton S. Hershey Medical Center Withdrawn
Hershey, Pennsylvania, United States, 17033
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29425
Principal Investigator: Tatsiana Beiko, MD         
United States, Texas
University of Texas Health Center at Tyler Recruiting
Tyler, Texas, United States, 75708
Principal Investigator: James Stocks, MD         
Hospital del Torax Dr. Antonio A. Cetrangolo Withdrawn
Vicente Lopez, Buenos Aires, Argentina, B1602DOH
Centro Dr. Lazaro Langer Withdrawn
Cordoba, Argentina, 5000
Australia, New South Wales
St Vincent's Hospital Sydney Recruiting
Sydney, New South Wales, Australia, 2010
Principal Investigator: Allan Glanville, MD         
Australia, Queensland
The Prince Charles Hospital Recruiting
Chermside, Queensland, Australia, 4032
Principal Investigator: Daniel Chambers, MD         
Australia, South Australia
Royal Adelaide Hospital Recruiting
Adelaide, South Australia, Australia, 5000
Principal Investigator: Mark Holmes, MD         
Australia, Victoria
St Vincent's Hospital Melbourne Recruiting
Fitzroy, Victoria, Australia, 3065
Principal Investigator: Chong Ong, MD         
Australia, Western Australia
Institute for Respiratory Health Inc Recruiting
Nedlands, Western Australia, Australia, 6009
Principal Investigator: Fiona Lake, MD         
Faculdade de Medicina do ABC Recruiting
Santo André, Sao Paulo, Brazil, 09060-650
Principal Investigator: Elie Fiss         
UNIFESP - Universidade Federal de São Paulo Active, not recruiting
São Paulo, Sao Paulo, Brazil, 04023-061
Instituto do Coração - Incor- HCFMUSP Recruiting
São Paulo, Brazil, 05403-000
Principal Investigator: Alberto Cukier         
Canada, Nova Scotia
Queen Elizabeth II Health Sciences Centre Recruiting
Halifax, Nova Scotia, Canada, B3H3A8
Principal Investigator: Paul Hernandez, MD         
Canada, Ontario
Inspiration Research Limited Recruiting
Toronto, Ontario, Canada, M5T3A11
Principal Investigator: Kenneth Chapman, MD         
Århus Universitetshospital Recruiting
Arhus C, Denmark, 8000
Principal Investigator: Tina Skjold, MD         
Gentofte Hospital Recruiting
Gentofte, Denmark, 2900
Principal Investigator: Niels Seersholm, MD         
North Estonia Medical Centre Foundation Recruiting
Tallinn, Estonia, 13419
Principal Investigator: Pille Mukk, MD         
Tartu University Hospital Active, not recruiting
Tartu, Estonia, 51014
Groupe Hospitalier Sud - Hôpital Haut-Lévêque Withdrawn
Pessac, Gironde, France, 33604
Hôpital Cardio-Vasculaire et Pneumologique Louis Pradel Recruiting
Bron, Rhone, France, 69500
Principal Investigator: Jean-François Mornex, MD         
Universitaetsklinikum Heidelberg Withdrawn
Heidelberg, Baden Wuerttemberg, Germany, 69126
Universitaetsklinikum Giessen und Marburg GmbH Withdrawn
Marburg, Hessen, Germany, 35043
Ruhrlandklinik Withdrawn
Essen, Nordrhein Westfalen, Germany, 45239
Univer des Saarlandes Innere Withdrawn
Homburg Saar, Germany, 66421
New Zealand
New Zealand Respiratory and Sleep institute Recruiting
Auckland, New Zealand, 1051
Principal Investigator: Jeff Garrett, MD         
Christchurch Hospital NZ Recruiting
Christchurch, New Zealand, 8013
Principal Investigator: Michael Epton, MD         
Waikato Hospital Recruiting
Hamilton, New Zealand, 3240
Principal Investigator: Catherina Chang, MD         
SPZOZ Szpital Uniwersytecki w Krakowie Recruiting
Krakow, Poland, 31-066
Principal Investigator: Krzysztof Sladek, MD         
Instytut Gruzlicy i Chorob Pluc w Warszawie Recruiting
Warszawa, Poland, 01-138
Principal Investigator: Joanna Chorostowska-Wynimko, MD         
Clinic Dr.G Curteanu Oradea Withdrawn
Oradea, Romania, 410176
Spitalul Clinic de Boli Infectioase si Pneumoftiziologie Withdrawn
Timisoara, Romania, 300312
Russian Federation
SBEI HPE Altai State Medical University of MoH and SD Recruiting
Barnaul, Russian Federation, 656038
Principal Investigator: Irina Tseimakh         
FSI Scientific Research Institute of Pulmonology Withdrawn
Moscow, Russian Federation, 105077
Hospital Universitario Central de Asturias Withdrawn
Oviedo, Asturias, Spain, 33011
Hospital de Cruces Withdrawn
Barakaldo, Vizcaya, Spain, 48903
Sahlgrenska Sjukhuset Recruiting
Göteborg, Sweden, 41345
Sub-Investigator: Dan Curiac, MD         
CTC - Clinical Trial Consultants AB Recruiting
Linköping, Sweden, 587 58
Sub-Investigator: Folke Sjoberg, MD         
Skånes Universitetssjukhus, Malmö Recruiting
Malmö, Sweden, 20502
Principal Investigator: Eeva Piitulainen, MD         
Karolinska Universitetssjukhuset, Solna Recruiting
Stockholm, Sweden, 11361
Sub-Investigator: Michael Runold, MD         
CTC Clinical Trial Consultants AB Recruiting
Uppsala, Sweden, 752 37
Sub-Investigator: Johan Månflod, MD         
Sponsors and Collaborators
Grifols Therapeutics LLC

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Grifols Therapeutics LLC Identifier: NCT01983241     History of Changes
Other Study ID Numbers: GTi1201
First Posted: November 13, 2013    Key Record Dates
Last Update Posted: August 27, 2019
Last Verified: August 2019
Keywords provided by Grifols Therapeutics LLC:
Pulmonary Emphysema
Alpha-1 Antitrypsin Deficiency
Alpha-1 PI Deficiency
Alpha-1 Protienase Inhibitor
Additional relevant MeSH terms:
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Alpha 1-Antitrypsin Deficiency
Pulmonary Emphysema
Pathologic Processes
Pulmonary Disease, Chronic Obstructive
Lung Diseases, Obstructive
Lung Diseases
Respiratory Tract Diseases
Liver Diseases
Digestive System Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Alpha 1-Antitrypsin
Protein C Inhibitor
Protease Inhibitors
Trypsin Inhibitors
Serine Proteinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action