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
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
1 other identifier
interventional
345
16 countries
50
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 an optional pre-screening phase, Screening Phase, a 156-week Treatment Phase, and an End of Study Visit at Week 160.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Nov 2013
Longer than P75 for phase_3
50 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 28, 2013
CompletedStudy Start
First participant enrolled
November 1, 2013
CompletedFirst Posted
Study publicly available on registry
November 13, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
January 1, 2027
March 4, 2026
March 1, 2026
12.8 years
October 28, 2013
March 2, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change from Baseline in Whole lung PD15 (15th percentile point)
Whole lung PD15 measured by CT scan
Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156
Secondary Outcomes (9)
Adverse Events (AEs)
Week -3 through Week 160
Serious Adverse Events (SAEs)
Week -3 through Week 160
Discontinuations from the study due to AEs
Week -3 through Week 160
Severe COPD Exacerbations
Week -3 through Week 160
Change from Baseline in PD15 of the basal lung region
Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156
- +4 more secondary outcomes
Study Arms (3)
Alpha-1 MP 60 mg/kg
EXPERIMENTALAlpha-1 MP 60 mg/kg administered weekly by IV infusion for 156 weeks
Alpha-1 MP 120 mg/kg
EXPERIMENTALAlpha-1 MP 120 mg/kg administered weekly by IV infusion for 156 weeks
Placebo
PLACEBO COMPARATOR0.9% Sodium Chloride for Injection, USP, administered weekly by IV infusion for 156 weeks
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (52)
Grifols Investigative Site
Phoenix, Arizona, 85013, United States
Grifols Investigative Site
Gainesville, Florida, 32610, United States
Grifols Investigative Site
Leesburg, Florida, 34748, United States
Grifols Investigative Site
Miami, Florida, 33136, United States
Grifols Investigative Site
St Louis, Missouri, 63110, United States
Grifols Investigative Site
Wilmington, North Carolina, 28401, United States
Grifols Investigative Site
Portland, Oregon, 97239, United States
Grifols Investigative Site
Hershey, Pennsylvania, 17033, United States
Grifols Investigative Site
Charleston, South Carolina, 29425, United States
Grifols Investigative Site
Tyler, Texas, 75708, United States
Grifols Investigative Site
Vicente López, Buenos Aires, B1602DOH, Argentina
Grifols Investigative Site
Córdoba, Córdoba Province, 5000, Argentina
Grifols Investigative Site
Sydney, New South Wales, 2010, Australia
Grifols Investigative Site
Chermside, Queensland, 4032, Australia
Grifols Investigative Site
Adelaide, South Australia, 5000, Australia
Grifols Investigative Site
Fitzroy, Victoria, 3065, Australia
Grifols Investigative Site
Nedlands, Western Australia, 6009, Australia
Grifols Investigative Site
Santo André, São Paulo, 09060-650, Brazil
Grifols Investigative Site
São Paulo, São Paulo, 04023-061, Brazil
Grifols Investigative Site
São Paulo, São Paulo, 05403-000, Brazil
Grifols Investigative Site
St. John's, Newfoundland and Labrador, A1B 3V6, Canada
Grifols Investigative Site
Halifax, Nova Scotia, B3H3A8, Canada
Grifols Investigative Site
Toronto, Ontario, M5T3A11, Canada
Grifols Investigative Site
Arhus C, 8000, Denmark
Grifols Investigative Site
Hellerup, 2900, Denmark
Grifols Investigative Site
Tallinn, 13419, Estonia
Grifols Investigative Site
Tartu, 51014, Estonia
Grifols Investigative Site
Turku, 20520, Finland
Grifols Investigative Site
Pessac, Gironde, 33604, France
Grifols Investigative Site
Bron, Rhone, 69500, France
Grifols Investigative Site
Heidelberg, Baden-Wurttemberg, 69126, Germany
Grifols Investigative Site
Marburg, Hesse, 35043, Germany
Grifols Investigative Site
Essen, North Rhine-Westphalia, 45239, Germany
Grifols Investigative Site
Homburg Saar, 66421, Germany
Grifols Investigative Site
Chisinau, 2025, Moldova
Grifols Investigative Site
Auckland, 1051, New Zealand
Grifols Investigative Site
Christchurch, 8013, New Zealand
Grifols Investigative Site
Hamilton, 3240, New Zealand
Grifols Investigative Site
Krakow, 31-066, Poland
Grifols Investigative Site
Warsaw, 01-138, Poland
Grifols Investigative Site
Oradea, 410176, Romania
Grifols Investigative Site
Timișoara, 300312, Romania
Grifols Investigative Site
Barnaul, 656038, Russia
Grifols Investigative Site
Moscow, 105077, Russia
Grifols Investigative Site
Yaroslavl, 150003, Russia
Grifols Investigative Site
Oviedo, Principality of Asturias, 33011, Spain
Grifols Investigative Site
Barakaldo, Vizcaya, 48903, Spain
Grifols Investigative Site
Gothenburg, 41345, Sweden
Grifols Investigative Site
Linköping, 587 58, Sweden
Grifols Investigative Site
Malmo, 20502, Sweden
Grifols Investigative Site
Stockholm, 11361, Sweden
Grifols Investigative Site
Uppsala, 752 37, Sweden
Related Publications (1)
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.
PMID: 25727857DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 28, 2013
First Posted
November 13, 2013
Study Start
November 1, 2013
Primary Completion (Estimated)
August 1, 2026
Study Completion (Estimated)
January 1, 2027
Last Updated
March 4, 2026
Record last verified: 2026-03