NCT02796937

Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency

An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency

• Status: Enrolling By Invitation
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 290 (estimated)
• Sponsor: Grifols Therapeutics LLC · Industry
• Sex: All
• Age: 20 Years – 72 Years
• Healthy volunteers: Not accepted

Summary

This is a 2-year open-label, multicenter extension of the double-blind, placebo-controlled GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha1-MP 60 mg/kg/week in subjects with alpha1-antitrypsin deficiency (AATD).

Detailed description

This is a 2-year open-label extension of the double-blind, placebo-controlled GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha-1 MP 60 mg/kg/week in subjects with AATD. The study consists of a Screening Visit (the same visit as the End-of-Study Visit in the GTi1201 study for subjects who complete the GTi1201 study or is the same visit as the Early Discontinuation Visit for subjects meeting the early withdrawal criterion for forced expiratory volume in 1 second \[FEV1\] decline), a treatment period of 104 weeks (beginning immediately after screening \[on the same day as the Screening Visit\] but no sooner than 1 week after the last infusion of investigational product in the GTi1201 study), and an End-of-Study Visit. Subjects meeting the entrance criteria of the extension study will begin receiving weekly intravenous (IV) infusions of Alpha-1 MP 60 mg/kg on the day of screening and will continue to receive weekly infusions for a total of 104 infusions. Safety assessments will include adverse events, concomitant medications, complete physical examination (excluding breast and genitourinary examination), hematology, chemistry, urine cotinine, and pregnancy test. Efficacy assessments will include whole lung computed tomography density, quality-of-life assessment, carbon monoxide diffusing capacity, and pulmonary function tests. The occurrence of chronic obstructive pulmonary exacerbations, will also be evaluated as a safety and as an efficacy measurement.

Conditions

• Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	Alpha-1 MP	Alpha-1 MP 60 mg/kg/week for up to 104 weeks

Timeline

• Start date: 2016-07-01
• Primary completion: 2028-09-01
• Completion: 2029-02-01
• First posted: 2016-06-13
• Last updated: 2026-03-04

Locations

28 sites across 12 countries: United States, Australia, Canada, Denmark, Estonia, Finland, France, Moldova, New Zealand, Poland, Russia, Sweden