The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 180 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC) infusion and subsequently as weekly SC infusions over 8 weeks in participants with Alpha1-Antitrypsin Deficiency (AATD).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
17
Alpha1-Proteinase Inhibitor (Human), 15%, Subcutaneous infusion
Intravenous infusion
UCLA Medical Center
Los Angeles, California, United States
University of Florida
Gainesville, Florida, United States
University of Miami
Miami, Florida, United States
Cleveland Clinic
Cleveland, Ohio, United States
Number of Participants With Adverse Events (AEs)
Time frame: Up to 668 days
Number of Participants With Suspected Adverse Drug Reactions (ADRs)
Time frame: Up to 668 days
Number of Participants With Infusion Site Reactions
Time frame: Up to 668 days
Number of Participants With Serious Adverse Events (SAEs)
Time frame: Up to 668 days
Number of Participants With AEs and SAEs Leading to Discontinuation
Time frame: Up to 668 days
Number of Participants With Chronic Obstructive Pulmonary Disease (COPD) Exacerbations
Time frame: Up to 668 days
Number of Participants With Clinically Significant Abnormalities in Vital Signs (Heart Rate, Blood Pressure, Respiratory Rate, and Temperature)
Time frame: Up to 668 days
Change from Baseline in Forced Expiratory Volume in 1 Second (FEV1)
Time frame: Up to 668 days
Change from Baseline in Forced Vital Capacity (FVC)
Time frame: Up to 668 days
Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters (Chemistry, Hematology, Urinalysis)
Time frame: Up to 668 days
Immunogenicity: Number of Participants With Alpha1-PI Antibodies
Time frame: Treatment Period 1- Single-Dose Week 1; Treatment Period 2- Repeat-Dose Weeks 1 and 9
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Medical University of South Carolina - Children's Hospital
Charleston, South Carolina, United States