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).
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
290
Alpha-1 MP 60 mg/kg/week for up to 104 weeks
Grifols Investigative Site
Phoenix, Arizona, United States
Adverse events (AEs)
Monitoring of AEs
Time frame: Week 1 through Week 108
Serious AEs (SAEs)
Monitoring of SAEs
Time frame: Week 1 through Week 108
Discontinuations from the study due to AEs
Monitoring of discontinuations due to AEs
Time frame: Week 1 through Week 108
Change from baseline in whole lung PD15 (15th percentile point)
Whole lung PD15 measured by computed tomography scans
Time frame: Week 1 through Week 104
Change from baseline in carbon monoxide diffusing capacity (DLco)
DLco performed according to American Thoracic Society/European Respiratory Society (ATS/ERS) guidelines
Time frame: Week 52 and Week 104
Changes from baseline in forced expiratory volume in 1 second (FEV1)
FEV1 performed according to ATS/ERS guidelines
Time frame: Week 52 and Week 104
Change from baseline in Saint George's Respiratory Questionnaire
Health-related quality of life assessment tool
Time frame: Week 52 and Week 104
Incidence and severity of Chronic Obstructive Pulmonary Disease (COPD)exacerbations
Severe COPD exacerbations as defined by ATS/ERS guidelines
Time frame: Week 2 through Week 108
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Grifols Investigative Site
Miami, Florida, United States
Grifols Investigative Site
Wilmington, North Carolina, United States
Grifols Investigative Site
Portland, Oregon, United States
Grifols Investigative Site
Charleston, South Carolina, United States
Grifols Investigative Site
Tyler, Texas, United States
Grifols Investigative Site
Darlinghurst, New South Wales, Australia
Grifols Investigative Site
Adelaide, Australia
Grifols Investigative Site
Chermside, Australia
Grifols Investigative Site
Fitzroy, Australia
...and 18 more locations
Change from baseline in the EQ-5D-5L Questionnaire
Heath-related quality of life assessment tool
Time frame: Week 52 and Week 104