NCT01983241

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

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 345 (actual)
• Sponsor: Grifols Therapeutics LLC · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Pulmonary Emphysema in Alpha-1 PI Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	Alpha-1 MP
OTHER	0.9% Sodium Chloride for Injection, USP

Timeline

• Start date: 2013-11-01
• Primary completion: 2026-08-01
• Completion: 2027-01-01
• First posted: 2013-11-13
• Last updated: 2026-03-04

Locations

52 sites across 17 countries: United States, Argentina, Australia, Brazil, Canada, Denmark, Estonia, Finland, France, Germany, Moldova, New Zealand, Poland, Romania, Russia, Spain, Sweden