Clinical Trial: Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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 Subjec

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

Detailed Summary:
Sponsor: Grifols Therapeutics Inc.

Current Primary Outcome: 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


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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


Original Secondary Outcome: Same as current

Information By: Grifols Therapeutics Inc.

Dates:
Date Received: October 28, 2013
Date Started: November 2013
Date Completion: August 2021
Last Updated: December 1, 2016
Last Verified: December 2016