Trial record 8 of 18 for:    Open Studies | "alpha 1-Antitrypsin Deficiency"

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.
Verified March 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: March 18, 2014
Last verified: March 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01983241

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

Locations
United States, North Carolina
Recruiting
Wilmington, North Carolina, United States, 28401
Principal Investigator: Mitchell D Lee, MD         
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: March 18, 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:
Alpha 1-Antitrypsin Deficiency
Emphysema
Pulmonary Emphysema
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
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 April 17, 2014