Pulmonary Emphysema in Alpha-1 Antitrypsin DeficiencyPhase 3SPARTA-OLENCT02796937
Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency
A Phase 3 study testing Alpha-1 MP in people with Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency, run by Grifols Therapeutics LLC at 28 sites in 12 countries.
Trial summary
This is a 2-year open-label, multicenter extension of the double-blind, placebo-controlled GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha1-MP 60 mg/kg/week in subjects with alpha1-antitrypsin deficiency (AATD). Read more about this study
- Who is it for?
- Ages 20 to 72 years
- Conditions the study is for
- Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency
- What is being tested?
- Alpha-1 MP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 290 participants
- Where
- 28 sitesin 12 countries
- Study dates
- Jul 2016to Feb 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 20 to 72 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Has completed participation in Study GTi1201 (ie, completed Week 156 and Week 160/End-of-Study Visit) OR has experienced a decline in FEV1 at the annualized rate of ≥134.4 mL/year at or after the Week 104 Visit in GTi1201.
- Is willing and able to provide informed consent
Who cannot join
- Is unable to physically or mentally undergo a CT scan (eg, unable to fit inside the CT scanner, claustrophobic).
- Has severe concomitant disease including, but not limited to, congestive heart failure and liver cirrhosis.
- Has primary and/or secondary (metastatic disease) pulmonary malignancy or other current malignancy with <1 year predicted overall survival.
- Has a metal object (newly received since starting GTi1201) that might interfere with chest CT quality and quantification. Metal objects include, but are not limited to, cardiac pacemaker, defibrillator, metal prosthetic heart valve, metal projectile, metal weapon fragments, or metal shoulder prosthesis.
- Is a female who is pregnant, breastfeeding or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (oral, injectable, or implanted hormonal methods of contraception; placement of an intrauterine device or intrauterine system; condom or occlusive cap with spermicidal foam/gel/film/cream/suppository; male sterilization; or abstinence) throughout the study.
- Has clinical signs and symptoms of viral infection requiring virus safety testing at the Week 160/End-of-Study Visit or Early Discontinuation Visit in Study GTi1201, and the virus safety test results are indicative of acute or chronic infection with hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV), human immunodeficiency virus (HIV), or parvovirus B19 (B19V). Note: If the virus safety test results are indicative of acute or chronic infection with HAV, HBV, HCV, HIV, or B19V, the subject will be considered a screen failure and must be withdrawn from the study.
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- Has current evidence of smoking, which includes electronic/vapor cigarettes, or has a positive urine cotinine test at the Week 160/End-of- Study Visit in Study GTi1201 that is due to smoking.
- Has current evidence of chronic alcoholism or illicit drug abuse (addiction).
- Is currently participating in another investigational product (IP) study.
- Has a history of anaphylaxis or severe systemic response to any plasma- derived alpha1-PI preparation or other blood product(s).
- In the opinion of the investigator, is likely to have compliance problems with the protocol and the procedures of the protocol.
- Has any medical condition that the investigator feels might confound the results of the study or pose an additional risk to the subject during study participation.
Trial locations and contacts
28 sites in 12 countries. Contact the study team or a site near you to ask about taking part.
28 sites in 12 countries
United States6 sites
Grifols Investigative Site
Charleston, South Carolina, 29425
Grifols Investigative Site
Miami, Florida, 33136
Grifols Investigative Site
Phoenix, Arizona, 85013
Grifols Investigative Site
Portland, Oregon, 97239
Grifols Investigative Site
Tyler, Texas, 75708
Grifols Investigative Site
Wilmington, North Carolina, 28401
Australia5 sites
Grifols Investigative Site
Adelaide, 5000
Grifols Investigative Site
Chermside, 4032
Grifols Investigative Site
Darlinghurst, New South Wales, 2010
Grifols Investigative Site
Fitzroy, 3065
Grifols Investigative Site
Nedlands, 6009
New Zealand3 sites
Grifols Investigative Site
Auckland, 2025
Grifols Investigative Site
Christchurch, 8011
Grifols Investigative Site
Hamilton, 3200
Sweden3 sites
Grifols Investigative Site
Gothenburg, 413 45
Grifols Investigative Site
Malmö, 20502
Grifols Investigative Site
Stockholm, 11361
Canada2 sites
Grifols Investigative Site
Halifax, B3H 3A7
Grifols Investigative Site
Toronto, M5T 3A9
Denmark2 sites
Grifols Investigative Site
Arhus C, 8000
Grifols Investigative Site
Hellerup, 2900
Poland2 sites
Grifols Investigative Site
Krakow, 31-066
Grifols Investigative Site
Warsaw, 01-138
About this study
Official title: An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency
This is a 2-year open-label, multicenter extension of the double-blind, placebo-controlled GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha1-MP 60 mg/kg/week in subjects with alpha1-antitrypsin deficiency (AATD).
Read the detailed description
This is a 2-year open-label extension of the double-blind, placebo-controlled GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha-1 MP 60 mg/kg/week in subjects with AATD.
The study consists of a Screening Visit (the same visit as the End-of-Study Visit in the GTi1201 study for subjects who complete the GTi1201 study or is the same visit as the Early Discontinuation Visit for subjects meeting the early withdrawal criterion for forced expiratory volume in 1 second [FEV1] decline), a treatment period of 104 weeks (beginning immediately after screening [on the same day as the Screening Visit] but no sooner than 1 week after the last infusion of investigational product in the GTi1201 study), and an End-of-Study Visit.
Subjects meeting the entrance criteria of the extension study will begin receiving weekly intravenous (IV) infusions of Alpha-1 MP 60 mg/kg on the day of screening and will continue to receive weekly infusions for a total of 104 infusions.
Safety assessments will include adverse events, concomitant medications, complete physical examination (excluding breast and genitourinary examination), hematology, chemistry, urine cotinine, and pregnancy test. Efficacy assessments will include whole lung computed tomography density, quality-of-life assessment, carbon monoxide diffusing capacity, and pulmonary function tests. The occurrence of chronic obstructive pulmonary exacerbations, will also be evaluated as a safety and as an efficacy measurement.
- Being tested
- Alpha-1 MP
- 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
Alpha-1 MP 60 mg/kg/week for up to 104 weeks
| Study group | Role | What they receive |
|---|---|---|
| Alpha-1 MP | Experimental | Alpha-1 MP 60 mg/kg/week for up to 104 weeks Receives: Biological: Alpha-1 MP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events (AEs)
Monitoring of AEs
Measured: Week 1 through Week 108
Serious AEs (SAEs)
Monitoring of SAEs
Measured: Week 1 through Week 108
Discontinuations from the study due to AEs
Monitoring of discontinuations due to AEs
Measured: Week 1 through Week 108
Secondary outcomes (6)
Change from baseline in whole lung PD15 (15th percentile point)
Whole lung PD15 measured by computed tomography scans
Measured: Week 1 through Week 104
Change from baseline in carbon monoxide diffusing capacity (DLco)
DLco performed according to American Thoracic Society/European Respiratory Society (ATS/ERS) guidelines
Measured: Week 52 and Week 104
Changes from baseline in forced expiratory volume in 1 second (FEV1)
FEV1 performed according to ATS/ERS guidelines
Measured: Week 52 and Week 104
Change from baseline in Saint George's Respiratory Questionnaire
Health-related quality of life assessment tool
Measured: Week 52 and Week 104
Incidence and severity of Chronic Obstructive Pulmonary Disease (COPD)exacerbations
Severe COPD exacerbations as defined by ATS/ERS guidelines
Measured: Week 2 through Week 108
Change from baseline in the EQ-5D-5L Questionnaire
Heath-related quality of life assessment tool
Measured: Week 52 and Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jun 2016 (estimated)
- Study startJul 2016
- Primary completionSep 2028 (estimated)
- Study completionFeb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Emphysema in Alpha-1 Antitrypsin 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 290 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Grifols Therapeutics LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02796937
- Sponsor's protocol ID
- GTi1201-OLE
- Acronym
- SPARTA-OLE
- Status last verified
- Jun 2026
- First submitted
- 3 Jun 2016
- Last updated
- 23 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency
- ClinicalTrials.gov ID
- NCT02796937
- Acronym / sponsor ID
- SPARTA-OLE / GTi1201-OLE
- Keywords
- Pulmonary Emphysema, Alpha-1 Antitrypsin Deficiency, AATD, Alpha-1 PI Deficiency, Alpha-1 Proteinase Inhibitor
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 290 participants (estimated)
- Lead sponsor
- Grifols Therapeutics LLCIndustry (pharma, biotech, devices)
- Study start
- Jul 2016 (actual)
- Primary completion
- Sep 2028 (estimated)
- Study completion
- Feb 2029 (estimated)
- First posted
- 13 Jun 2016 (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 2-year open-label, multicenter extension of the double-blind, placebo-controlled GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha1-MP 60 mg/kg/week in subjects with alpha1-antitrypsin deficiency (AATD).
Detailed description
This is a 2-year open-label extension of the double-blind, placebo-controlled GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha-1 MP 60 mg/kg/week in subjects with AATD.
The study consists of a Screening Visit (the same visit as the End-of-Study Visit in the GTi1201 study for subjects who complete the GTi1201 study or is the same visit as the Early Discontinuation Visit for subjects meeting the early withdrawal criterion for forced expiratory volume in 1 second [FEV1] decline), a treatment period of 104 weeks (beginning immediately after screening [on the same day as the Screening Visit] but no sooner than 1 week after the last infusion of investigational product in the GTi1201 study), and an End-of-Study Visit.
Subjects meeting the entrance criteria of the extension study will begin receiving weekly intravenous (IV) infusions of Alpha-1 MP 60 mg/kg on the day of screening and will continue to receive weekly infusions for a total of 104 infusions.
Safety assessments will include adverse events, concomitant medications, complete physical examination (excluding breast and genitourinary examination), hematology, chemistry, urine cotinine, and pregnancy test. Efficacy assessments will include whole lung computed tomography density, quality-of-life assessment, carbon monoxide diffusing capacity, and pulmonary function tests. The occurrence of chronic obstructive pulmonary exacerbations, will also be evaluated as a safety and as an efficacy measurement.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Alpha-1 MP | Experimental | Biological: Alpha-1 MPAlpha-1 MP 60 mg/kg/week for up to 104 weeks |
Who may qualify
- Ages
- 20 to 72 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Has completed participation in Study GTi1201 (ie, completed Week 156 and Week 160/End-of-Study Visit) OR has experienced a decline in FEV1 at the annualized rate of ≥134.4 mL/year at or after the Week 104 Visit in GTi1201.
- Is willing and able to provide informed consent
Key exclusion criteria (12 listed)
- Is unable to physically or mentally undergo a CT scan (eg, unable to fit inside the CT scanner, claustrophobic).
- Has severe concomitant disease including, but not limited to, congestive heart failure and liver cirrhosis.
- Has primary and/or secondary (metastatic disease) pulmonary malignancy or other current malignancy with <1 year predicted overall survival.
- Has a metal object (newly received since starting GTi1201) that might interfere with chest CT quality and quantification. Metal objects include, but are not limited to, cardiac pacemaker, defibrillator, metal prosthetic heart valve, metal projectile, metal weapon fragments, or metal shoulder prosthesis.
- Is a female who is pregnant, breastfeeding or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (oral, injectable, or implanted hormonal methods of contraception; placement of an intrauterine device or intrauterine system; condom or occlusive cap with spermicidal foam/gel/film/cream/suppository; male sterilization; or abstinence) throughout the study.
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-CAlpha-1 MP 60 mg/kg/week for up to 104 weeks | Biological | Alpha-1 MP |
Primary outcome
Adverse events (AEs)
Time frame: Week 1 through Week 108
Monitoring of AEs
Serious AEs (SAEs)
Time frame: Week 1 through Week 108
Monitoring of SAEs
Discontinuations from the study due to AEs
Time frame: Week 1 through Week 108
Monitoring of discontinuations due to AEs
6 secondary outcomes
Change from baseline in whole lung PD15 (15th percentile point)
Time frame: Week 1 through Week 104
Change from baseline in carbon monoxide diffusing capacity (DLco)
Time frame: Week 52 and Week 104
Changes from baseline in forced expiratory volume in 1 second (FEV1)
Time frame: Week 52 and Week 104
Change from baseline in Saint George's Respiratory Questionnaire
Time frame: Week 52 and Week 104
Incidence and severity of Chronic Obstructive Pulmonary Disease (COPD)exacerbations
Time frame: Week 2 through Week 108
Change from baseline in the EQ-5D-5L Questionnaire
Time frame: Week 52 and Week 104
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2026.
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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.
The record lists no contact. Use the official record to reach the sponsor.
28 sites in 12 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 in people with Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency, run by Grifols Therapeutics LLC at 28 sites in 12 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 2 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: enrolling by invitation, as of the sponsor's last update on 23 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States, Australia, New Zealand, Sweden, Canada and 7 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. 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?
This study is still under way. Results are not available in the source record yet.
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?
Adverse events (AEs) (time frame: Week 1 through Week 108). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Jun 2026.