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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)

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ClinicalTrials.gov Identifier: NCT01983241
Recruitment Status : Recruiting
First Posted : November 13, 2013
Last Update Posted : October 2, 2018
Sponsor:
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

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



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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
Criteria

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 ClinicalTrials.gov identifier (NCT number): NCT01983241


Contacts
Contact: Susan Sorrells Susan.Sorrells@grifols.com

  Hide Study Locations
Locations
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, 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 Recruiting
Hershey, Pennsylvania, United States, 17033
Principal Investigator: Timothy Craig, DO         
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         
Argentina
Hospital del Torax Dr. Antonio A. Cetrangolo Recruiting
Vicente Lopez, Buenos Aires, Argentina, B1602DOH
Principal Investigator: Mariano Fernandez Acquier         
Centro Dr. Lazaro Langer Recruiting
Cordoba, Argentina, 5000
Principal Investigator: Marcos Langer         
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         
Brazil
Faculdade de Medicina do ABC Recruiting
Santo André, Sao Paulo, Brazil, 09060-650
Principal Investigator: Elie Fiss         
UNIFESP - Universidade Federal de São Paulo Recruiting
São Paulo, Sao Paulo, Brazil, 04023-061
Principal Investigator: José Roberto de Brito Jardim         
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         
Denmark
Århus Universitetshospital Recruiting
Arhus C, Denmark, 8000
Principal Investigator: Tina Skjold, MD         
Gentofte Hospital Recruiting
Gentofte, Denmark, 2900
Principal Investigator: Niels Seersholm, MD         
Estonia
North Estonia Medical Centre Foundation Recruiting
Tallinn, Estonia, 13419
Principal Investigator: Pille Mukk, MD         
Tartu University Hospital Recruiting
Tartu, Estonia, 51014
Principal Investigator: Rain Jogi, MD         
France
Groupe Hospitalier Sud - Hôpital Haut-Lévêque Recruiting
Pessac, Gironde, France, 33604
Principal Investigator: Claire Dromer, MD         
Hôpital Cardio-Vasculaire et Pneumologique Louis Pradel Recruiting
Bron, Rhone, France, 69500
Principal Investigator: Jean-François Mornex, MD         
Germany
Universitaetsklinikum Heidelberg Terminated
Heidelberg, Baden Wuerttemberg, Germany, 69126
Universitaetsklinikum Giessen und Marburg GmbH Terminated
Marburg, Hessen, Germany, 35043
Ruhrlandklinik Terminated
Essen, Nordrhein Westfalen, Germany, 45239
Univer des Saarlandes Innere Terminated
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         
Poland
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         
Romania
Clinic Dr.G Curteanu Oradea Recruiting
Oradea, Romania, 410176
Principal Investigator: Lavinia Davidescu         
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 Recruiting
Moscow, Russian Federation, 105077
Principal Investigator: Andrey Belevskiy         
Spain
Hospital de Cruces Recruiting
Barakaldo, Vizcaya, Spain, 48903
Principal Investigator: Juan Bautista Galdiz Iturri, MD         
Sweden
Sahlgrenska Sjukhuset Recruiting
Göteborg, Sweden, 41345
Principal Investigator: Dan Curiac, MD         
CTC - Clinical Trial Consultants AB Recruiting
Malmo, Sweden, 205 02
Principal Investigator: Folke Sjoberg, MD         
Skånes Universitetssjukhus, Malmö Recruiting
Malmö, Sweden, 20502
Principal Investigator: Eeva Piitulainen, MD         
Karolinska Universitetssjukhuset, Solna Recruiting
Stockholm, Sweden, 11361
Principal Investigator: Michael Runold, MD         
Sponsors and Collaborators
Grifols Therapeutics LLC

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Grifols Therapeutics LLC
ClinicalTrials.gov Identifier: NCT01983241     History of Changes
Other Study ID Numbers: GTi1201
First Posted: November 13, 2013    Key Record Dates
Last Update Posted: October 2, 2018
Last Verified: September 2018

Keywords provided by Grifols Therapeutics LLC:
Pulmonary Emphysema
Alpha-1 Antitrypsin Deficiency
AATD
Alpha-1 PI Deficiency
Alpha-1 Protienase Inhibitor

Additional relevant MeSH terms:
Alpha 1-Antitrypsin Deficiency
Alpha 1-Antitrypsin
Emphysema
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
Protein C Inhibitor
Protease Inhibitors
Trypsin Inhibitors
Serine Proteinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action