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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 : March 13, 2023
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics LLC

Study Description
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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.

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 Design
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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
Actual Study Start Date : November 2013
Estimated Primary Completion Date : June 2026
Estimated Study Completion Date : June 2026

Resource links provided by the National Library of Medicine


Arms and Interventions
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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


Outcome Measures
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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

  6. 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

  7. 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

  8. 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

  9. 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


Eligibility Criteria
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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.
Contacts and Locations
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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
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Contact: David Taylor david.taylor@grifols.com
Contact: Gordon McAlester gordon.mcalester@grifols.com

Locations
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Sponsors and Collaborators
Grifols Therapeutics LLC
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Grifols Therapeutics LLC
ClinicalTrials.gov Identifier: NCT01983241    
Other Study ID Numbers: GTi1201
First Posted: November 13, 2013    Key Record Dates
Last Update Posted: March 13, 2023
Last Verified: March 2023
Keywords provided by Grifols Therapeutics LLC:
Pulmonary Emphysema
Alpha-1 Antitrypsin Deficiency
AATD
Alpha-1 PI Deficiency
Alpha-1 Proteinase Inhibitor
Additional relevant MeSH terms:
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Alpha 1-Antitrypsin Deficiency
Pulmonary Emphysema
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
Alpha 1-Antitrypsin
Trypsin Inhibitors
Serine Proteinase Inhibitors
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action