Trial record 1 of 275 for:    "Pulmonary Emphysema"
Previous Study | Return to List | Next Study

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)

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Grifols Therapeutics Inc.
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT01983241
First received: October 28, 2013
Last updated: July 22, 2014
Last verified: July 2014
  Purpose

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 up to 21-day Screening Phase, a 156-week Treatment Phase, and an End of Study Visit at Week 160.


Condition Intervention Phase
Pulmonary Emphysema in Alpha-1 PI Deficiency
Biological: Alpha1-proteinase inhibitor (human), modified process (Alpha-1 MP)
Other: 0.9% Sodium Chloride for Injection, USP
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by Grifols Therapeutics Inc.:

Primary Outcome Measures:
  • Change from Baseline in Whole lung PD15 (15th percentile point) [ Time Frame: Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156 ] [ Designated as safety issue: No ]
    Whole lung PD15 measured by CT scan


Secondary Outcome Measures:
  • Adverse Events (AEs) [ Time Frame: Week -3 through Week 160 ] [ Designated as safety issue: Yes ]
  • Serious Adverse Events (SAEs) [ Time Frame: Week -3 through Week 160 ] [ Designated as safety issue: Yes ]
  • Discontinuations from the study due to AEs [ Time Frame: Week -3 through Week 160 ] [ Designated as safety issue: Yes ]
  • Severe COPD Exacerbations [ Time Frame: Week -3 through Week 160 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    PD15 of the basal lung region measure by CT scan


Estimated Enrollment: 339
Study Start Date: November 2013
Estimated Study Completion Date: August 2021
Estimated Primary Completion Date: August 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Alpha-1 MP 60 mg/kg
Alpha-1 MP 60 mg/kg administered weekly by IV infusion for 156 weeks
Biological: Alpha1-proteinase inhibitor (human), modified process (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: Alpha1-proteinase inhibitor (human), modified process (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

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
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.
  • 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01983241

Contacts
Contact: Rhonda Griffin rhonda.griffin@grifols.com

Locations
United States, Arizona
Recruiting
Phoenix, Arizona, United States, 85013
Principal Investigator: Rajat Walia, MD         
United States, Florida
Recruiting
Gainesville, Florida, United States, 32610
Principal Investigator: Mark Brantly, MD         
Recruiting
Miami, Florida, United States, 33136
Principal Investigator: Michael Campos, MD         
United States, Missouri
Recruiting
St. Louis, Missouri, United States, 63110
Principal Investigator: James Quirk, PhD         
United States, North Carolina
Recruiting
Wilmington, North Carolina, United States, 28401
Principal Investigator: Mitchell D Lee, MD         
United States, Pennsylvania
Recruiting
Hershey, Pennsylvania, United States, 17033
Principal Investigator: Timothy Craig, DO         
United States, Texas
Recruiting
Tyler, Texas, United States, 75708
Principal Investigator: James Stocks, MD         
Sponsors and Collaborators
Grifols Therapeutics Inc.
  More Information

No publications provided

Responsible Party: Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT01983241     History of Changes
Other Study ID Numbers: GTi1201
Study First Received: October 28, 2013
Last Updated: July 22, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Grifols Therapeutics Inc.:
Pulmonary Emphysema
Alpha-1 Antitrypsin Deficiency
AATD
Alpha-1 PI Deficiency
Alpha-1 Protienase Inhibitor

Additional relevant MeSH terms:
Emphysema
Pulmonary Emphysema
Subcutaneous Emphysema
Alpha 1-Antitrypsin Deficiency
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Pathologic Processes
Alpha 1-Antitrypsin
Protein C Inhibitor
Protease Inhibitors
Trypsin Inhibitors
Serine Proteinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 24, 2014