A phase 2, double-blind, randomized, active-control, parallel group study comparing INBRX-101 with plasma-derived alpha1-proteinase inhibitor (A1-PI) augmentation therapy in patients with alpha-1 antitrypsin deficiency (AATD) with emphysema is currently recruiting.
The study, known as ELEVAATE, will enroll approximately 90 adults (18-80 years old), males or females, with a confirmed diagnosis of AATD and evidence of emphysema secondary to AATD. In addition, participants must be nonsmokers and must have a predicted forced expiratory volume in the first second between 30% and 80% (inclusive).
Participants in experimental arms will receive intravenous INBRX-101 every 3 or 4 weeks and placebo. The active comparator group will receive Zemaira®, 60 mg/kg, administered intravenously once weekly, and placebo.
The primary endpoint of ELEVAATE is serum functional alpha-1 antitrypsin (fAAT) levels at steady state as measured by antineutrophil elastase capacity.
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Secondary outcome measures include changes in fAAT concentration, days with fAAT above the lower limit of the normal range, incidence of treatment-emergent adverse events, frequency of antidrug antibodies, population pharmacokinetics (clearance and volume of distribution), and covariate analysis, including assessment of the impact of patient’s weight, height, age, and sex on the pharmacokinetics of INBRX-101.
Patients who received A1-PI augmentation therapy within 5 weeks prior to the first dose of study drug or intravenous immunoglobulins, monoclonal antibodies, and/or other biologic therapies within 30 days are excluded from the study.
Additional exclusion criteria are known or suspected allergy to components of INBRX-101, A1-PI, or human immunoglobulin G; known selective or severe immunoglobulin A deficiency; known or suspected diagnosis of type 1 diabetes or diagnosis of uncontrolled type 2 diabetes; being on a waiting list for a lung or liver transplant; acute respiratory tract infection or chronic obstructive pulmonary disease exacerbation within 4 weeks prior to or during screening; evidence of decompensated cirrhosis; active cancers or history of malignancy within 5 years prior to screening; history of unstable cor pulmonale; and clinically significant congestive heart failure.
The estimated completion date of ELEVAATE is March 2025.
NBRX-101 is an optimized recombinant human AAT-Fc fusion protein.
Study of INBRX-101 compared to plasma-derived A1PI therapy in adults with AATD emphysema (ELEVAATE). ClinicalTrials.gov. May 12, 2023. Updated May 15, 2023. Accessed May 18, 2023.