ClinicalTrials.gov
ClinicalTrials.gov Menu

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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01983241
Recruitment Status : Recruiting
First Posted : November 13, 2013
Last Update Posted : October 2, 2018
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics LLC

October 28, 2013
November 13, 2013
October 2, 2018
November 2013
November 2023   (Final data collection date for 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 ]
Whole lung PD15 measured by CT scan
Same as current
Complete list of historical versions of study NCT01983241 on ClinicalTrials.gov Archive Site
  • Adverse Events (AEs) [ Time Frame: Week -3 through Week 160 ]
    Monitoring of AEs
  • Serious Adverse Events (SAEs) [ Time Frame: Week -3 through Week 160 ]
    Monitoring of SAEs
  • Discontinuations from the study due to AEs [ Time Frame: Week -3 through Week 160 ]
    Monitoring of discontinuations due to AEs
  • 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)
  • 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
  • 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 ]
    Severe COPD exacerbations as defined by American Thoracic Society/European Respiratory Society (ATS/ERS) criteria (i.e., COPD exacerbations requiring hospitalization)
  • 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
Not Provided
Not Provided
 
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 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 a Screening Phase, a 156-week Treatment Phase, and an End of Study Visit at Week 160.
Not Provided
Interventional
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Pulmonary Emphysema in Alpha-1 PI Deficiency
  • Biological: Alpha-1 MP
    Other Name: Prolastin-C
  • Other: 0.9% Sodium Chloride for Injection, USP
    Other Name: Saline
  • Experimental: Alpha-1 MP 60 mg/kg
    Alpha-1 MP 60 mg/kg administered weekly by IV infusion for 156 weeks
    Intervention: Biological: Alpha-1 MP
  • Experimental: Alpha-1 MP 120 mg/kg
    Alpha-1 MP 120 mg/kg administered weekly by IV infusion for 156 weeks
    Intervention: Biological: Alpha-1 MP
  • Placebo Comparator: Placebo
    0.9% Sodium Chloride for Injection, USP, administered weekly by IV infusion for 156 weeks
    Intervention: Other: 0.9% Sodium Chloride for Injection, USP
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
339
Same as current
November 2023
November 2023   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
18 Years to 70 Years   (Adult, Older Adult)
No
Contact: Susan Sorrells Susan.Sorrells@grifols.com
Brazil,   Argentina,   Australia,   Canada,   Denmark,   Estonia,   France,   Germany,   New Zealand,   Poland,   Romania,   Russian Federation,   Spain,   Sweden,   United States
 
 
NCT01983241
GTi1201
No
Not Provided
Not Provided
Grifols Therapeutics LLC
Grifols Therapeutics LLC
Not Provided
Not Provided
Grifols Therapeutics LLC
September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP