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

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

Official Title: 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 Pul

Brief 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.

Detailed Summary:

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 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.

Sponsor: Grifols Therapeutics Inc.

Current Primary Outcome:

• Incidence of adverse events (AEs) [ Time Frame: Week 1 through Week 108 ]
• Incidence of serious adverse events (SAEs) [ Time Frame: Week 1 through Week 108 ]

Original Primary Outcome:

• Incidence of adverse events (AEs) [ Time Frame: Week 1 through Week 108 ]
• Incidence of serious adverse events (SAEs) [ Time Frame: Week 1 through Week 108 ]
• Number of subjects with changes from baseline in vital signs parameters [ Time Frame: Week 26 through Week 108 ]
  To include changes from low, normal, or high at baseline to low, normal, or high post-baseline
• Number of subjects with changes from baseline in hematology parameters [ Time Frame: Week 26 through Week 108 ]
  To include changes from low, normal, or high at baseline to low, normal, or high post-baseline
• Number of subjects with changes from baseline in chemistry parameters [ Time Frame: Week 26 through Week 108 ]
  To include changes from low, normal, or high at baseline to low, normal, or high post-baseline

Current Secondary Outcome:

• Change from baseline in total lung capacity-adjusted lung density [ Time Frame: Week 1 through Week 104 ]
• Change from baseline in carbon monoxide diffusing capacity [ Time Frame: Week 52 and Week 104 ]
• Changes from baseline in forced expiratory volume in 1 second [ Time Frame: Week 52 and Week 104 ]
• Change from baseline in Saint George's Respiratory Questionnaire [ Time Frame: Week 52 and Week 104 ]
• Incidence and severity of Chronic Obstructive Pulmonary Disease exacerbations [ Time Frame: Week 2 through Week 108 ]

Original Secondary Outcome: Same as current

Information By: Grifols Therapeutics Inc.

Dates:
Date Received: June 3, 2016
Date Started: July 2016
Date Completion: July 2023
Last Updated: December 1, 2016
Last Verified: December 2016