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)

March 2, 2026 updated by: Grifols Therapeutics LLC

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

345

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
    • Buenos Aires
      • Vicente López, Buenos Aires, Argentina, B1602DOH
        • Grifols Investigative site
    • Córdoba Province
      • Córdoba, Córdoba Province, Argentina, 5000
        • Grifols Investigative site
  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2010
        • Grifols Investigative site
    • Queensland
      • Chermside, Queensland, Australia, 4032
        • Grifols Investigative site
    • South Australia
      • Adelaide, South Australia, Australia, 5000
        • Grifols Investigative site
    • Victoria
      • Fitzroy, Victoria, Australia, 3065
        • Grifols Investigative site
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Grifols Investigative site
  • Brazil
    • São Paulo
      • Santo André, São Paulo, Brazil, 09060-650
        • Grifols Investigative site
      • São Paulo, São Paulo, Brazil, 04023-061
        • Grifols Investigative site
      • São Paulo, São Paulo, Brazil, 05403-000
        • Grifols Investigative site
  • Canada
    • Newfoundland and Labrador
      • St. John's, Newfoundland and Labrador, Canada, A1B 3V6
        • Grifols Investigative site
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H3A8
        • Grifols Investigative site
    • Ontario
      • Toronto, Ontario, Canada, M5T3A11
        • Grifols Investigative site
  • Denmark
      • Arhus C, Denmark, 8000
        • Grifols Investigative site
      • Hellerup, Denmark, 2900
        • Grifols Investigative site
  • Estonia
      • Tallinn, Estonia, 13419
        • Grifols Investigative site
      • Tartu, Estonia, 51014
        • Grifols Investigative site
  • Finland
      • Turku, Finland, 20520
        • Grifols Investigative site
  • France
    • Gironde
      • Pessac, Gironde, France, 33604
        • Grifols Investigative site
    • Rhone
      • Bron, Rhone, France, 69500
        • Grifols Investigative site
  • Germany
      • Homburg Saar, Germany, 66421
        • Grifols Investigative site
    • Baden-Wurttemberg
      • Heidelberg, Baden-Wurttemberg, Germany, 69126
        • Grifols Investigative site
    • Hesse
      • Marburg, Hesse, Germany, 35043
        • Grifols Investigative site
    • North Rhine-Westphalia
      • Essen, North Rhine-Westphalia, Germany, 45239
        • Grifols Investigative site
  • Moldova
      • Chisinau, Moldova, 2025
        • Grifols Investigative site
  • New Zealand
      • Auckland, New Zealand, 1051
        • Grifols Investigative site
      • Christchurch, New Zealand, 8013
        • Grifols Investigative site
      • Hamilton, New Zealand, 3240
        • Grifols Investigative site
  • Poland
      • Krakow, Poland, 31-066
        • Grifols Investigative site
      • Warsaw, Poland, 01-138
        • Grifols Investigative site
  • Romania
      • Oradea, Romania, 410176
        • Grifols Investigative site
      • Timișoara, Romania, 300312
        • Grifols Investigative site
  • Russia
      • Barnaul, Russia, 656038
        • Grifols Investigative site
      • Moscow, Russia, 105077
        • Grifols Investigative site
      • Yaroslavl, Russia, 150003
        • Grifols Investigative site
  • Spain
    • Principality of Asturias
      • Oviedo, Principality of Asturias, Spain, 33011
        • Grifols Investigative site
    • Vizcaya
      • Barakaldo, Vizcaya, Spain, 48903
        • Grifols Investigative site
  • Sweden
      • Gothenburg, Sweden, 41345
        • Grifols Investigative site
      • Linköping, Sweden, 587 58
        • Grifols Investigative site
      • Malmo, Sweden, 20502
        • Grifols Investigative site
      • Stockholm, Sweden, 11361
        • Grifols Investigative site
      • Uppsala, Sweden, 752 37
        • Grifols Investigative site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • Grifols Investigative site
    • Florida
      • Gainesville, Florida, United States, 32610
        • Grifols Investigative site
      • Leesburg, Florida, United States, 34748
        • Grifols Investigative site
      • Miami, Florida, United States, 33136
        • Grifols Investigative site
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Grifols Investigative site
    • North Carolina
      • Wilmington, North Carolina, United States, 28401
        • Grifols Investigative site
    • Oregon
      • Portland, Oregon, United States, 97239
        • Grifols Investigative site
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Grifols Investigative site
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Grifols Investigative site
    • Texas
      • Tyler, Texas, United States, 75708
        • Grifols Investigative site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 70 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / 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 Names:
  • 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 Names:
  • 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 Names:
  • Saline

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse Events (AEs)
Time Frame: Week -3 through Week 160
Monitoring of AEs
Week -3 through Week 160
Serious Adverse Events (SAEs)
Time Frame: Week -3 through Week 160
Monitoring of SAEs
Week -3 through Week 160
Discontinuations from the study due to AEs
Time Frame: Week -3 through Week 160
Monitoring of discontinuations due to AEs
Week -3 through Week 160
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)
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
PD15 of the basal lung region measure by CT scan
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
DLco performed according to American Thoracic Society/European Respiratory Society (ATS/ERS) guidelines
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
FEV1 performed according to ATS/ERS guidelines
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
Health-related quality of life assessment tool
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
Heath-related quality of life assessment tool
Weeks 26, 52, 78, 104, 130 and 156

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Grifols Therapeutics LLC

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 1, 2013

Primary Completion (Estimated)

August 1, 2026

Study Completion (Estimated)

January 1, 2027

Study Registration Dates

First Submitted

October 28, 2013

First Submitted That Met QC Criteria

November 7, 2013

First Posted (Estimated)

November 13, 2013

Study Record Updates

Last Update Posted (Actual)

March 4, 2026

Last Update Submitted That Met QC Criteria

March 2, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GTi1201

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Pulmonary Emphysema in Alpha-1 PI Deficiency

Clinical Trials on Alpha-1 MP

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Kenya

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe