Pulmonary Emphysema in Alpha-1 PI DeficiencyPhase 3SPARTANCT01983241
Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)
A Phase 3 study testing Alpha-1 MP and 0.9% Sodium Chloride for Injection, USP in people with Pulmonary Emphysema in Alpha-1 PI Deficiency, run by Grifols Therapeutics LLC at 52 sites in 17 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Pulmonary Emphysema in Alpha-1 PI Deficiency
- What is being tested?
- Alpha-1 MP and 0.9% Sodium Chloride for Injection, USP
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 345 participants
- Where
- 52 sitesin 17 countries
- Study dates
- 1 Apr 2014to 5 Aug 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
Show all 12Show fewer
- 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.
Trial locations and contacts
52 sites in 17 countries.
52 sites in 17 countries
United States10 sites
Grifols Investigative Site
Charleston, South Carolina, 29425
Grifols Investigative Site
Gainesville, Florida, 32610
Grifols Investigative Site
Hershey, Pennsylvania, 17033
Grifols Investigative Site
Leesburg, Florida, 34748
Grifols Investigative Site
Miami, Florida, 33136
Grifols Investigative Site
Phoenix, Arizona, 85013
Grifols Investigative Site
Portland, Oregon, 97239
Grifols Investigative Site
St Louis, Missouri, 63110
Grifols Investigative Site
Tyler, Texas, 75708
Grifols Investigative Site
Wilmington, North Carolina, 28401
Australia5 sites
Grifols Investigative Site
Adelaide, South Australia, 5000
Grifols Investigative Site
Chermside, Queensland, 4032
Grifols Investigative Site
Fitzroy, Victoria, 3065
Grifols Investigative Site
Nedlands, Western Australia, 6009
Grifols Investigative Site
Sydney, New South Wales, 2010
Sweden5 sites
Grifols Investigative Site
Gothenburg, 41345
Grifols Investigative Site
Linköping, 587 58
Grifols Investigative Site
Malmö, 20502
Grifols Investigative Site
Stockholm, 11361
Grifols Investigative Site
Uppsala, 752 37
Germany4 sites
Grifols Investigative Site
Essen, North Rhine-Westphalia, 45239
Grifols Investigative Site
Heidelberg, Baden-Wurttemberg, 69126
Grifols Investigative Site
Homburg Saar, 66421
Grifols Investigative Site
Marburg, Hesse, 35043
Brazil3 sites
Grifols Investigative Site
Santo André, São Paulo, 09060-650
Grifols Investigative Site
São Paulo, São Paulo, 04023-061
Grifols Investigative Site
São Paulo, São Paulo, 05403-000
Canada3 sites
Grifols Investigative Site
Halifax, Nova Scotia, B3H3A8
Grifols Investigative Site
St. John's, Newfoundland and Labrador, A1B 3V6
Grifols Investigative Site
Toronto, Ontario, M5T3A11
New Zealand3 sites
Grifols Investigative Site
Auckland, 1051
Grifols Investigative Site
Christchurch, 8013
Grifols Investigative Site
Hamilton, 3240
Russia3 sites
Grifols Investigative Site
Barnaul, 656038
Grifols Investigative Site
Moscow, 105077
Grifols Investigative Site
Yaroslavl, 150003
Argentina2 sites
Grifols Investigative Site
Córdoba, Córdoba Province, 5000
Grifols Investigative Site
Vicente López, Buenos Aires, B1602DOH
Denmark2 sites
Grifols Investigative Site
Arhus C, 8000
Grifols Investigative Site
Hellerup, 2900
Estonia2 sites
Grifols Investigative Site
Tallinn, 13419
Grifols Investigative Site
Tartu, 51014
France2 sites
Grifols Investigative Site
Bron, Rhone, 69500
Grifols Investigative Site
Pessac, Gironde, 33604
Poland2 sites
Grifols Investigative Site
Krakow, 31-066
Grifols Investigative Site
Warsaw, 01-138
Romania2 sites
Grifols Investigative Site
Oradea, 410176
Grifols Investigative Site
Timișoara, 300312
Spain2 sites
Grifols Investigative Site
Barakaldo, Vizcaya, 48903
Grifols Investigative Site
Oviedo, Principality of Asturias, 33011
About this study
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
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.
- Being tested
- Alpha-1 MP, 0.9% Sodium Chloride for Injection, USP
- Keywords
- Pulmonary Emphysema, Alpha-1 Antitrypsin Deficiency, AATD, Alpha-1 PI Deficiency, Alpha-1 Proteinase Inhibitor
- Medical subject headings
- Pulmonary Emphysema, alpha 1-Antitrypsin Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Alpha-1 MPBiological
Also called Prolastin-C
0.9% Sodium Chloride for Injection, USPOther
Also called Saline
| Study group | Role | What they receive |
|---|---|---|
| Alpha-1 MP 60 mg/kg | Experimental | Alpha-1 MP 60 mg/kg administered weekly by IV infusion for 156 weeks Receives: Biological: Alpha-1 MP |
| Alpha-1 MP 120 mg/kg | Experimental | Alpha-1 MP 120 mg/kg administered weekly by IV infusion for 156 weeks Receives: Biological: Alpha-1 MP |
| Placebo | Placebo comparator | 0.9% Sodium Chloride for Injection, USP, administered weekly by IV infusion for 156 weeks Receives: Other: 0.9% Sodium Chloride for Injection, USP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in Whole lung PD15 (15th percentile point)
Whole lung PD15 measured by CT scan
Measured: Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156
Secondary outcomes (9)
Adverse Events (AEs)
Monitoring of AEs
Measured: Week -3 through Week 160
Serious Adverse Events (SAEs)
Monitoring of SAEs
Measured: Week -3 through Week 160
Discontinuations from the study due to AEs
Monitoring of discontinuations due to AEs
Measured: Week -3 through Week 160
Severe COPD Exacerbations
Severe COPD exacerbations as defined by American Thoracic Society/European Respiratory Society (ATS/ERS) criteria (i.e., COPD exacerbations requiring hospitalization)
Measured: Week -3 through Week 160
Change from Baseline in PD15 of the basal lung region
PD15 of the basal lung region measure by CT scan
Measured: Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156
Change from baseline in carbon monoxide diffusing capacity (DLco)
DLco performed according to American Thoracic Society/European Respiratory Society (ATS/ERS) guidelines
Measured: Weeks 26, 52, 78, 104, 130 and 156
Changes from baseline in forced expiratory volume in 1 second (FEV1)
FEV1 performed according to ATS/ERS guidelines
Measured: Weeks 26, 52, 78, 104, 130 and 156
Change from baseline in Saint George's Respiratory Questionnaire: Minimum value = 0, maximum value = 100, higher scores indicate worse condition
Health-related quality of life assessment tool
Measured: Weeks 26, 52, 78, 104, 130 and 156
Change from baseline in the EuroQoL (Quality of Life)- 5 Dimension- 5 Level (EQ-5D-5L): Minimum value (for each one of the 5 levels) = 1, maximum value (for each one of the 5 levels) = 5, higher scores indicate worse condition
Heath-related quality of life assessment tool
Measured: Weeks 26, 52, 78, 104, 130 and 156
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2013 (estimated)
- Study start1 Apr 2014
- Primary completion7 Jul 2026
- Study completion5 Aug 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pulmonary Emphysema in Alpha-1 PI DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 345 participants (actual)
Sponsor and investigators
- Lead sponsor
- Grifols Therapeutics LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01983241
- Sponsor's protocol ID
- GTi1201
- Acronym
- SPARTA
- Status last verified
- Sep 2026
- First submitted
- 28 Oct 2013
- Last updated
- 1 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 25727857
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01983241
- Acronym / sponsor ID
- SPARTA / GTi1201
- Keywords
- Pulmonary Emphysema, Alpha-1 Antitrypsin Deficiency, AATD, Alpha-1 PI Deficiency, Alpha-1 Proteinase Inhibitor
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 345 participants (actual)
- Lead sponsor
- Grifols Therapeutics LLCIndustry (pharma, biotech, devices)
- Study start
- 1 Apr 2014 (actual)
- Primary completion
- 7 Jul 2026 (actual)
- Study completion
- 5 Aug 2026 (actual)
- First posted
- 13 Nov 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Alpha-1 MP 60 mg/kg | Experimental | Biological: Alpha-1 MPAlpha-1 MP 60 mg/kg administered weekly by IV infusion for 156 weeks |
| Alpha-1 MP 120 mg/kg | Experimental | Biological: Alpha-1 MPAlpha-1 MP 120 mg/kg administered weekly by IV infusion for 156 weeks |
| Placebo | Placebo comparator | Other: 0.9% Sodium Chloride for Injection, USP0.9% Sodium Chloride for Injection, USP, administered weekly by IV infusion for 156 weeks |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Alpha-1 MPAlso: Prolastin-C | Biological | Alpha-1 MP 120 mg/kg, Alpha-1 MP 60 mg/kg |
| 0.9% Sodium Chloride for Injection, USPAlso: Saline | Other | Placebo |
Primary outcome
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
9 secondary outcomes
Adverse Events (AEs)
Time frame: Week -3 through Week 160
Serious Adverse Events (SAEs)
Time frame: Week -3 through Week 160
Discontinuations from the study due to AEs
Time frame: Week -3 through Week 160
Severe COPD Exacerbations
Time frame: Week -3 through Week 160
Change from Baseline in PD15 of the basal lung region
Time frame: Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156
Change from baseline in carbon monoxide diffusing capacity (DLco)
Time frame: Weeks 26, 52, 78, 104, 130 and 156
Changes from baseline in forced expiratory volume in 1 second (FEV1)
Time frame: Weeks 26, 52, 78, 104, 130 and 156
Change from baseline in Saint George's Respiratory Questionnaire: Minimum value = 0, maximum value = 100, higher scores indicate worse condition
Time frame: Weeks 26, 52, 78, 104, 130 and 156
Change from baseline in the EuroQoL (Quality of Life)- 5 Dimension- 5 Level (EQ-5D-5L): Minimum value (for each one of the 5 levels) = 1, maximum value (for each one of the 5 levels) = 5, higher scores indicate worse condition
Time frame: Weeks 26, 52, 78, 104, 130 and 156
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2026.
- Derived from this study: PubMed 25727857Sorrells 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
52 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Alpha-1 MP and 0.9% Sodium Chloride for Injection, USP in people with Pulmonary Emphysema in Alpha-1 PI Deficiency, run by Grifols Therapeutics LLC at 52 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 52 sites in United States, Australia, Sweden, Germany, Brazil and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline in Whole lung PD15 (15th percentile point) (time frame: Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01983241, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2026.